drTarget portfolio target-disease associations for nervous system

OpenTargets offers > 40k target-disease associations from PChem targets suitable for virtual screening based in OpenTargets scores for nervous system diseases. See here the best scored pairs.

Target-disease associations for nervous system diseases.

Check best scored target-disease associations in table by selecting scores, genes or diseases:

PubChemAssay	program	testedComopunds	activeCompounds	diseaseName	associationScore	numberOfEvidences
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ1 potassium channels	KCNQ1	305610	3878	epilepsy	0.601110466899752	32
Primary cell-based high-throughput screening assay for identification of compounds that potentiate/activate KCNQ1 potassium channels	KCNQ1	305610	1082	epilepsy	0.601110466899752	32
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	epilepsy	0.694388489028926	1078
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	epilepsy	0.694388489028926	1078
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	epilepsy	0.700209149187544	307
Inhibitors of Cav3 T-type Calcium Channels: Primary Screen	CACNA1H_inhibitors	104728	4230	epilepsy	0.708489341786052	1893
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	epilepsy	0.56770916893568	14
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	stroke	0.539927483209128	7
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	stroke	0.539927483209128	7
qHTS of D3 Dopamine Receptor Agonist: qHTS	D3_agonists	407539	2380	stroke	0.563827088782784	12
qHTS of D3 Dopamine Receptor Antagonist: qHTS	D3_antagonists	364051	9106	stroke	0.563827088782784	12
qHTS of D3 Dopamine Receptor Potentiators: qHTS	D3_PAMs	407539	2380	stroke	0.563827088782784	12
HTS for Inhibition of CaV1.3 ICDI/IQ interaction using a live-cell FRET assay Measured in Cell-Based System Using Plate Reader - 7081-01_Inhibitor_SinglePoint_HTS_Activity	CACNA1D_inhibitors	335531	328	stroke	0.646209969421255	32
HTS for Beta-2AR agonists via FAP method	ADRB2_activators	339297	1446	stroke	0.628120473874919	23
Thrombin 1536 HTS	F2_modulation	217233	557	stroke	0.745819195337535	29
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	stroke	0.51828395256274	6
qHTS of TDP-43 Inhibitors	TARDBP	403703	7150	familial amyotrophic lateral sclerosis	0.91464415824384	482
qHTS of TDP-43 Inhibitors: NCGC Sytravon Library Screen	TARDBP	45163	203	familial amyotrophic lateral sclerosis	0.91464415824384	482
Primary biochemical high-throughput screening assay to measure P97 ATPase inhibition	VCP	217959	923	familial amyotrophic lateral sclerosis	0.699636907731946	319
qHTS for Suppressors of FUS Proteinopathy: Primary screen using FUS-linked yeast assay	FUS_asupressors	385746	932	sporadic amyotrophic lateral sclerosis	0.892054957027048	663
High-Throughput Screening for Modulators of Cytosolic Chaperonin Activity	MARVELD2	362274	1085	deafness	0.737707430803668	111
Primary Cell-Based High-Throughput Screening to Identify Agonists of the Sphingosine 1-phosphate receptor 2 (S1P2)	S1PR2	96879	447	deafness	0.581946589576287	24
Primary Cell-Based High-Throughput Screening to Identify Antagonists of the Sphingosine 1-phosphate receptor 2 (S1P2)	S1PR2	96879	207	deafness	0.581946589576287	24
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	peripheral neuropathy	0.511377243461816	809
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	peripheral neuropathy	0.554662029590942	19
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	peripheral neuropathy	0.554662029590942	19
qHTS of TDP-43 Inhibitors	TARDBP	403703	7150	motor neuron disease	0.636159681085823	613
qHTS of TDP-43 Inhibitors: NCGC Sytravon Library Screen	TARDBP	45163	203	motor neuron disease	0.636159681085823	613
uHTS identification of small molecule antagonists of the kappa opioid receptor via a luminescent beta-arrestin assay	OPRK1	290355	265	pain	0.66739120894794	1664
HTS of Estrogen Receptor- alpha Coactivator Binding inhibitors	ESR1_inhibitors	86095	1442	pain	0.502874234984747	10
HTS of Estrogen Receptor- alpha Coactivator Binding Potentiators	ESR1_modulators	86095	1151	pain	0.502874234984747	10
Luminescence-based cell-based primary high throughput screening assay to identify agonists of the mouse 5-hydroxytryptamine (serotonin) receptor 2A (HTR2A)	HTR2A	363803	2412	pain	0.602176088428136	20
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	pain	0.669376737588158	4676
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	pain	0.669376737588158	4676
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	pain	0.575594093745969	11
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Agonists	D2_agonists	359518	300	pain	0.583819867656698	15
HTS Assay for Allosteric Agonists of the Human D2 Dopamine Receptor: Primary Screen for Agonists	D2_agonists	335652	1779	pain	0.583819867656698	15
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Antagonists	D2_antagonists	362274	1056	pain	0.583819867656698	15
HTS Assay for Allosteric Antagonists of the Human D2 Dopamine Receptor: Primary Screen for Antagonists	D2_antagonists	336308	6862	pain	0.583819867656698	15
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Potentiators	D2_PAMs	357537	806	pain	0.583819867656698	15
HTS Assay for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Primary Screen for Potentiators	D2_PAMs	339887	1178	pain	0.583819867656698	15
qHTS of D3 Dopamine Receptor Agonist: qHTS	D3_agonists	407539	2380	pain	0.565948160307315	14
qHTS of D3 Dopamine Receptor Antagonist: qHTS	D3_antagonists	364051	9106	pain	0.565948160307315	14
qHTS of D3 Dopamine Receptor Potentiators: qHTS	D3_PAMs	407539	2380	pain	0.565948160307315	14
HTS for Inhibition of CaV1.3 ICDI/IQ interaction using a live-cell FRET assay Measured in Cell-Based System Using Plate Reader - 7081-01_Inhibitor_SinglePoint_HTS_Activity	CACNA1D_inhibitors	335531	328	pain	0.520819643474188	6
Fluorescence-based cell-based primary high throughput screening assay to identify agonists of the human cholinergic receptor, muscarinic 1 (CHRM1)	CHRM1_agonists	359207	1189	pain	0.608284957389344	18
Discovery of Novel Allosteric Modulators of the M1 Muscarinic Receptor: Agonist Primary Screen	CHRM1_allosteric_activators	63676	1938	pain	0.608284957389344	18
Fluorescence-based cell-based primary high throughput screening assay to identify positive allosteric modulators (PAMs) of the human M1 muscarinic receptor (CHRM1).	CHRM1_PAMs	359207	316	pain	0.608284957389344	18
Discovery of novel allosteric modulators of the M1 muscarinic receptor: Antagonist Primary Screen	CHRM1_allosteric_antagonists	63656	2179	pain	0.608284957389344	18
Fluorescence-based cell-based primary high throughput screening assay to identify antagonists of the human M1 muscarinic receptor (CHRM1)	CHRM1_antgonists	359207	4555	pain	0.608284957389344	18
HTS for Beta-2AR agonists via FAP method	ADRB2_activators	339297	1446	pain	0.659538183102147	110
Primary cell-based screen for identification of compounds that inhibit the two-pore domain potassium channel KCNK9	KCNK9_blockers	305610	3794	pain	0.593212449856628	10
Primary cell-based screen for identification of compounds that inhibit the two-pore domain potassium channel KCNK3	KCNK3	339674	2841	pain	0.593212449856628	10
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	pain	0.675089080796179	1370
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	pain	0.662857415411265	216
qHTS Assay for Inhibitors of the Six1/Eya2 Interaction	SIX1	364053	2026	hearing loss	0.563348198535835	93
High-Throughput Screening for Modulators of Cytosolic Chaperonin Activity	MARVELD2	362274	1085	hearing loss	0.54644243570006	75
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	partial epilepsy	0.592949719312303	9
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of the liver receptor homolog-1 (LRH-1; NR5A2)	NR5A2	363803	458	vital capacity	0.511751699353967	12
Fluorescence polarization-based primary biochemical high throughput screening assay to identify inhibitors of the HTRA serine peptidase 1 (HTRA1)	HTRA1	343467	1710	vital capacity	0.64677440372713	18
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	restless legs syndrome	0.516582222217723	9
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	restless legs syndrome	0.516582222217723	9
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Agonists	D2_agonists	359518	300	restless legs syndrome	0.658301784622236	117
HTS Assay for Allosteric Agonists of the Human D2 Dopamine Receptor: Primary Screen for Agonists	D2_agonists	335652	1779	restless legs syndrome	0.658301784622236	117
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Antagonists	D2_antagonists	362274	1056	restless legs syndrome	0.658301784622236	117
HTS Assay for Allosteric Antagonists of the Human D2 Dopamine Receptor: Primary Screen for Antagonists	D2_antagonists	336308	6862	restless legs syndrome	0.658301784622236	117
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Potentiators	D2_PAMs	357537	806	restless legs syndrome	0.658301784622236	117
HTS Assay for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Primary Screen for Potentiators	D2_PAMs	339887	1178	restless legs syndrome	0.658301784622236	117
qHTS of D3 Dopamine Receptor Agonist: qHTS	D3_agonists	407539	2380	restless legs syndrome	0.658022198908649	108
qHTS of D3 Dopamine Receptor Antagonist: qHTS	D3_antagonists	364051	9106	restless legs syndrome	0.658022198908649	108
qHTS of D3 Dopamine Receptor Potentiators: qHTS	D3_PAMs	407539	2380	restless legs syndrome	0.658022198908649	108
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	smoking cessation	0.640378804205756	36
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	suicidal ideation	0.574864567642163	18
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	attempted suicide	0.556037572799865	6
qHTS Assay for Identification of Novel General Anesthetics	FTL	341499	255	movement disorder	0.549353067496944	104
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS4-Galphao.	GNAO1	218528	711	movement disorder	0.514885375373638	44
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS7-Galphao.	GNAO1	218528	770	movement disorder	0.514885375373638	44
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS8-Galphao.	GNAO1	218528	750	movement disorder	0.514885375373638	44
Luminescence-based cell-based primary high throughput screening assay to identify agonists of the mouse 5-hydroxytryptamine (serotonin) receptor 2A (HTR2A)	HTR2A	363803	2412	movement disorder	0.608815855986794	37
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Agonists	D2_agonists	359518	300	movement disorder	0.653671582289342	258
HTS Assay for Allosteric Agonists of the Human D2 Dopamine Receptor: Primary Screen for Agonists	D2_agonists	335652	1779	movement disorder	0.653671582289342	258
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Antagonists	D2_antagonists	362274	1056	movement disorder	0.653671582289342	258
HTS Assay for Allosteric Antagonists of the Human D2 Dopamine Receptor: Primary Screen for Antagonists	D2_antagonists	336308	6862	movement disorder	0.653671582289342	258
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Potentiators	D2_PAMs	357537	806	movement disorder	0.653671582289342	258
HTS Assay for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Primary Screen for Potentiators	D2_PAMs	339887	1178	movement disorder	0.653671582289342	258
qHTS of D3 Dopamine Receptor Agonist: qHTS	D3_agonists	407539	2380	movement disorder	0.647165212846683	81
qHTS of D3 Dopamine Receptor Antagonist: qHTS	D3_antagonists	364051	9106	movement disorder	0.647165212846683	81
qHTS of D3 Dopamine Receptor Potentiators: qHTS	D3_PAMs	407539	2380	movement disorder	0.647165212846683	81
Primary HTS assay for 5-Hydroxytryptamine (Serotonin) Receptor Subtype 1a (5HT1a) agonists	HTR1A	64908	366	movement disorder	0.555655515890195	27
Primary HTS assay for 5-Hydroxytryptamine (Serotonin) Receptor Subtype 1a (5HT1a) antagonists	HTR1A	61606	416	movement disorder	0.555655515890195	27
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	alcohol drinking	0.51828395256274	6
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	alcohol drinking	0.51828395256274	6
qHTS Assay to Identify Small Molecule Activators of BRCA1 Expression	BRCA1 activation	376029	3978	neurodegenerative disease	0.534883338324039	90
qHTS Assay for Activators of Human Muscle isoform 2 Pyruvate Kinase	PKM	263662	892	neurodegenerative disease	0.50081897368671	10
uHTS fluorescence polarization assay for the identification of translation initiation inhibitors (PABP)	PABPC1	290915	2982	neurodegenerative disease	0.502663178367153	8
uHTS identification of microRNA-mediated mRNA deadenylation inhibitors by fluoresence polarization assay	PABPC1	359244	1307	neurodegenerative disease	0.502663178367153	8
qHTS Assay for Inhibitors of HADH2 (Hydroxyacyl-Coenzyme A Dehydrogenase, Type II)	HSD17B10	72072	2464	neurodegenerative disease	0.608987326393621	21
qHTS Assay for Inhibitors of HSD17B4, hydroxysteroid (17-beta) dehydrogenase 4	HSD17B10	73912	5649	neurodegenerative disease	0.608987326393621	21
HTS using DiI-HDL to assay lipid transfer in ldlA[SR-BI] cells Measured in Cell-Based System Using Plate Reader - 2085-01_Inhibitor_SinglePoint_HTS_Activity	SCARB1	319204	3065	neurodegenerative disease	0.564018684854049	11
HTS using DiI-HDL to assay lipid transfer in ldlA[SR-BI] cells Measured in Cell-Based System Using Plate Reader - 2085-01_Activator_SinglePoint_HTS_Activity	SCARB1	316970	306	neurodegenerative disease	0.564018684854049	11
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	neurodegenerative disease	0.592672623557014	34
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	neurodegenerative disease	0.592672623557014	34
uHTS identification of small molecule inhibitors of the catalytic domain of the SUMO protease, SENP1 in a FRET assay	SENP1	363840	774	neurodegenerative disease	0.575703329789328	7
Fluorescence polarization to screen for inhibitors that disrupt the protein-protein interaction between Keap1 and Nrf2 Measured in Biochemical System Using Plate Reader - 2119-01_Inhibitor_SinglePoint_HTS_Activity	KEAP1	336894	489	neurodegenerative disease	0.589190058569869	85
Luminescence-based cell-based primary high throughput screening assay to identify inhibitors of the Steroid Receptor Coactivator 1 (SRC1; NCOA1)	NCOA1	359206	428	neurodegenerative disease	0.532226285183309	8
HTS Assay for Peg3 Promoter Inhibitors	PPP1R15A	359244	6145	neurodegenerative disease	0.511414146696408	9
uHTS Identification of Diaphorase Inhibitors and Chemcical Oxidizers: Counter Screen for Diaphorase-based Primary Assays	DiaphoraseInhibitors	194152	1342	neurodegenerative disease	0.537758733599517	21
TRFRET-based biochemical primary high throughput screening assay to identify inhibitors of the interaction of the Ras and Rab interactor 1 protein (Rin1) and the c-abl oncogene 1, non-receptor tyrosine kinase (Abl)	ABL1_interaction	359207	1432	neurodegenerative disease	0.577328189160672	45
Dicer-mediated maturation of pre-microRNA	Dicer_inhibitors	46715	2829	neurodegenerative disease	0.595242557991075	21
qHTS Assay for Inhibitors of the Human Apurinic/apyrimidinic Endonuclease 1 (APE1)	APEX1_inhibitors	345898	1172	neurodegenerative disease	0.508986414703378	19
Primary cell-based high throughput screening assay to identify inhibitors of kruppel-like factor 5 (KLF5)	KLF5	290726	671	neurodegenerative disease	0.574191290410045	9
uHTS HTRF assay for identification of inhibitors of SUMOylation	UBE2I	290915	1039	neurodegenerative disease	0.515732094857419	8
EZH2/PRC2 methyltransferase inhibitors Measured in Biochemical System Using Plate Reader - 2125-01_Inhibitor_SinglePoint_HTS_Activity	EZH2_inhibitors	57013	201	neurodegenerative disease	0.582665511204253	28
Luminescence-based primary cell-based high throughput screening assay to identify activators of the Aryl Hydrocarbon Receptor (AHR)	AHR_activators	324747	7988	neurodegenerative disease	0.552731722062662	59
Epi Absorbance-based biochemical primary high throughput screening assay to identify inhibitors of human tyrosyl-DNA phosphodiesterase 2 (TDP2)	TDP2	369953	1241	neurodegenerative disease	0.671502953160591	26
uHTS Luminescent assay for identification of inhibitors of Sentrin-specific protease 6 (SENP6)	SENP6	330392	5817	neurodegenerative disease	0.554494817370566	9
HTS for Identification of VLA-4 Allosteric Modulators from MLPCN library	ITGA4	326888	645	neurodegenerative disease	0.59512464250193	58
A screen for small molecule inhibitors of the human deubiquitinating enzyme, UCH37	UCHL5	330707	1075	neurodegenerative disease	0.599029121048609	10
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of the liver receptor homolog-1 (LRH-1; NR5A2)	NR5A2	363803	458	neurodegenerative disease	0.538369387010613	9
uHTS Fluorescent assay for identification of activators of Apaf-1	APAF1_activators	331671	1041	neurodegenerative disease	0.599107081143662	29
uHTS Fluorescent assay for identification of inhibitors of Apaf-1	APAF1_inhibitors	331671	2353	neurodegenerative disease	0.599107081143662	29
qHTS of TDP-43 Inhibitors	TARDBP	403703	7150	neurodegenerative disease	0.642288414311277	2279
qHTS of TDP-43 Inhibitors: NCGC Sytravon Library Screen	TARDBP	45163	203	neurodegenerative disease	0.642288414311277	2279
HTS for PAX8 inhibitors using PAX8 luciferase reporter gene assay in RMG-I cells Measured in Cell-Based System Using Plate Reader - 7054-01_Inhibitor_SinglePoint_HTS_Activity	PAX8	353950	4145	neurodegenerative disease	0.559746831113451	37
Primary biochemical fluorescence polarization-based high throughput screening assay to identify inhibitors of protein arginine methyltransferase 1 (PRMT1)	PRMT1	369953	4757	neurodegenerative disease	0.502599783695389	6
HTS-Luminescent assay for inhibitors of ALR by detection of hydrogen peroxide production Measured in Biochemical System Using Plate Reader - 2036-02_Inhibitor_SinglePoint_HTS	ALR inhibitors	288728	10857	neurodegenerative disease	0.608729374700095	14
HTS-Luminescent assay for inhibitors of ALR by detection of hydrogen peroxide production Measured in Biochemical System Using Plate Reader - 2036-02_Inhibitor_SinglePoint_HTS	ALR activators	288728	10857	neurodegenerative disease	0.608729374700095	14
Screen for inhibitors of the SWI/SNF chromatin remodeling complex (esBAF) in mouse embryonic stem cells with Luciferase reporter assay Measured in Cell-Based System Using Plate Reader - 2141-01_Inhibitor_SinglePoint_HTS_Activity	SMARCA4	344733	7043	neurodegenerative disease	0.601022749475411	13
uHTS identification of DNMT1 inhibitors in a Fluorescent Molecular Beacon assay	DNMT1	359244	2975	neurodegenerative disease	0.618369114146208	709
Fluorescence polarization-based primary biochemical high throughput screening assay to identify inhibitors of tRNA 2'-phosphotransferase (TPT1).	TPT1	302663	2452	neurodegenerative disease	0.583074611929383	9
qHTS assay for re-activators of p53 using a Luc reporter	TP53	321427	201	neurodegenerative disease	0.652444219623287	429
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54509	528	neurodegenerative disease	0.652444219623287	429
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54513	338	neurodegenerative disease	0.652444219623287	429
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	125394	1890	neurodegenerative disease	0.652444219623287	429
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	124022	1156	neurodegenerative disease	0.652444219623287	429
qHTS Assay for Inhibitors Targeting the Menin-MLL Interaction in MLL Related Leukemias: Competition With Texas Red Labeled MLL-derived Mutant Peptide	Menin-MLL_inhibitors	263421	615	neurodegenerative disease	0.562884079664889	9
Primary biochemical high-throughput screening assay for inhibitors of Rho kinase 2 (Rhok2)	ROCK2	59788	212	neurodegenerative disease	0.611849024230215	21
Luminescence cell-based high throughput primary assay to identify inhibitors of TEAD-YAP interaction.	YAP1	639428	9218	neurodegenerative disease	0.606205598209275	33
SSB-PriA antibiotic resistant target AlphaScreen	Klebsiella pneumonia SSB-PriA interaction	431236	2568	neurodegenerative disease	0.60023710564504	20
USP8 deubiquitinase inhibition: Primary qHTS	USP8	47480	2010	neurodegenerative disease	0.628428729911184	75
HTS Assay for Inhibitors of Akt Phophorylation: Primary Screen	AKT1_inhibitors	356517	1139	neurodegenerative disease	0.529943602416502	353
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	neurodegenerative disease	0.611164613433357	317
Fluorescence polarization-based primary biochemical high throughput screening assay to identify inhibitors of myeloid cell leukemia sequence 1 (MCL1) interactions with BIM-BH3 peptide.	MCL1	314998	2139	neurodegenerative disease	0.539248910974092	13
uHTS of Mcl-1/Bid interaction inhibitors	MCL1	218602	2129	neurodegenerative disease	0.539248910974092	13
uHTS of Mcl-1/Noxa interaction inhibitors	MCL1	217330	3334	neurodegenerative disease	0.539248910974092	13
Inhibitors of CDC25B-CDK2/CyclinA interaction	Inhibitors of CDC25B-CDK2/CyclinA interaction	93798	679	neurodegenerative disease	0.599474308138305	16
qHTS Assay for Identifying a Potential Treatment of Ataxia-Telangiectasia	ATM_modulators	322361	619	neurodegenerative disease	0.593816422964424	6952
qHTS for Inhibitors of phosphatidylinositol 5-phosphate 4-kinase (PI5P4K)	PIP4K2A	328860	4078	neurodegenerative disease	0.559303232996058	6
uHTS for Small Molecule Inhibitors of Eukaryotic Translation Initiation	EIF4E_inhibition	217332	799	neurodegenerative disease	0.579549606498111	10
Luminescence Cell-Based Primary HTS to Identify Inhibitors of A1 Apoptosis.	BCL2L11_inhibitors	325630	216	neurodegenerative disease	0.568226999632248	24
Identification of Novel Modulators of Cl- dependent Transport Process via HTS: Primary Screen	SLC12A5	189132	4127	generalised epilepsy	0.726615041643907	463
uHTS identification of compounds inhibiting the binding between the RUNX1 Runt domain and CBFb via a fluorescence resonance energy transfer (FRET) assay.	RUNX1	215667	993	neurodegenerative disease	0.51129791900956	25
uHTS identification of compounds inhibiting the binding between the RUNX1 Runt domain and CBFb-SMMHC via a fluorescence resonance energy transfer (FRET) assay.	RUNX1	218234	1620	neurodegenerative disease	0.51129791900956	25
Absorbance-based primary biochemical high throughput screening assay to identify activators of procaspase-3	procaspase3Activators	326024	350	neurodegenerative disease	0.59508041112244	192
Luminescence-based cell-based primary high throughput screening assay to identify activators of the GAA850 frataxin (FXN) promoter	FXN	356160	1985	neurodegenerative disease	0.701945822331272	694
Primary biochemical high-throughput screening assay to measure P97 ATPase inhibition	VCP	217959	923	neurodegenerative disease	0.690582730159146	1125
Primary qHTS assay for inhibitors of Glutamate pyruvate transaminase 2 (GPT2)	GPT2	98912	236	neurodegenerative disease	0.614812938066516	23
Primary Cell-based High Throughput Screening Assay for Inhibitors of Wee1 Degradation	WEE1	217959	2616	neurodegenerative disease	0.565035496135817	92
Fluorescence polarization assay for PLK1 inhibitors	PLK1	97099	518	neurodegenerative disease	0.611292905266571	18
qHTS for Inhibitors of PLK1-PDB (polo-like kinase 1 - polo-box domain): Primary Screen	PLK1	364065	10181	neurodegenerative disease	0.611292905266571	18
High Throughput Imaging Assay for Beta-Catenin	betaCateninTranslocation	193542	587	neurodegenerative disease	0.588306145605832	37
qHTS Assay for the Inhibitors of Human Flap endonuclease 1 (FEN1).	FEN1_inhibitors	386270	1331	neurodegenerative disease	0.544693329001652	12
Primary cell-based high throughput screening assay to measure STAT3 activation	STAT3	194666	1772	neurodegenerative disease	0.576017409602427	101
Primary cell-based high throughput screening assay to measure STAT3 inhibition	STAT3	194666	1722	neurodegenerative disease	0.576017409602427	101
uHTS identification of small molecule antagonists of the kappa opioid receptor via a luminescent beta-arrestin assay	OPRK1	290355	265	neuropathic pain	0.529686109361221	14
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	neuropathic pain	0.654073840918953	89
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	neuropathic pain	0.654073840918953	89
Allosteric Modulators of D1 Receptors: Primary Screen	D1	57705	3413	neurodegenerative disease	0.543335400131825	31
Allosteric Agonists of the Human D1 Dopamine Receptor: qHTS	D1_activators	361330	3713	neurodegenerative disease	0.543335400131825	31
Antagonist of Human D 1 Dopamine Receptor: qHTS	D1_inhibitors	355805	11440	neurodegenerative disease	0.543335400131825	31
Luminescence-based cell-based primary high throughput screening assay to identify inhibitors of COUP-TFII (NR2F2)	NR2F2	369953	2602	neurodegenerative disease	0.59632424455501	11
HTS for Beta-2AR agonists via FAP method	ADRB2_activators	339297	1446	neurodegenerative disease	0.544728007188587	19
Luminescence-based cell-based primary high throughput screening assay for inhibitors of the orphan nuclear receptor subfamily 0, group B, member 1 (DAX1; NR0B1): repression of SF-1 (NR5A1) activated StAR promoter by full-length DAX-1	NR0B1	368927	4094	neurodegenerative disease	0.533758386789442	16
Luminescence-based primary cell-based high throughput screening assay to identify inhibitors of the orphan nuclear receptor subfamily 0, group B, member 1 (DAX1; NR0B1)	NR0B1	343468	3417	neurodegenerative disease	0.533758386789442	16
Primary biochemical high-throughput screening assay for inhibitors of Focal Adhesion Kinase (FAK)	PTK2	96879	811	neurodegenerative disease	0.608234150917648	23
HCS assay for microtubule stabilizers	TUBB	195821	1625	neurodegenerative disease	0.61128822057763	13
Alphascreen assay for small molecules abrogating mHTT-CaM Interaction	HTT	189882	6790	neurodegenerative disease	0.637927127163836	2712
Primary qHTS for Identification of Small Molecule Inhibitors of Huntingtin Promoter Activity	HTT	48068	449	neurodegenerative disease	0.637927127163836	2712
qHTS Multiplex Assay to Identify Dual Action Probes in a Cell Model of Huntington: Aggregate Formation (GFP)	HTT	220571	2380	neurodegenerative disease	0.637927127163836	2712
qHTS Multiplex Assay to Identify Dual Action Probes in a Cell Model of Huntington: Cytoprotection (ATP)	HTT	223611	305	neurodegenerative disease	0.637927127163836	2712
Primary biochemical high throughput screening assay to identify inhibitors of BCL2-related protein, long isoform (BCLXL).	BCL2L1_modulators	314998	2199	neurodegenerative disease	0.583916454689616	35
Fluorescence Cell-Free Homogeneous Primary HTS to Identify Inhibitors of Histone Deacetylase 3	HDAC3	318291	483	neurodegenerative disease	0.586931833345308	43
TRFRET-based cell-based primary high throughput screening assay to identify inhibitors of cell surface Prion Protein (PRPC)	PRNP	369953	1596	neurodegenerative disease	0.674892905265714	1044
Primary cell-based high-throughput screening assay to measure PERK inhibition	PERK_inhibitors	217959	370	neurodegenerative disease	0.537983171760528	102
Counterscreen for inhibitors of PP5: fluorescence-based biochemical high throughput primary assay to identify inhibitors of Protein Phosphatase 1 (PP1).	PPP1CA	314999	2841	neurodegenerative disease	0.588329498055209	15
Acumen qHTS Assay for Inhibitors of the mTORC1 Signaling Pathway in MEF (Tsc2-/-, p53-/-) Cells: Sytravon	MTOR	43989	342	neurodegenerative disease	0.624690579486488	367
AlphaScreen-based biochemical high throughput primary assay to identify inhibitors of Unc-51 Like Autophagy Activating Kinase 1 (ULK1).	ULK1	30185	575	neurodegenerative disease	0.603278579256237	26
HTS identification of compounds activating phosphomannose isomerase (PMI) via a fluorescence intensity assay using a near- saturating concentration of mannose 6-phosphat	MPI	194152	656	neurodegenerative disease	0.586380059223403	8
HTS identification of compounds inhibiting phosphomannose isomerase (PMI) via a fluorescence intensity assay using a high concentration of mannose 6-phosphate	MPI	194152	1288	neurodegenerative disease	0.586380059223403	8
HTS identification of compounds inhibiting phosphomannose isomerase (PMI) via a fluorescence intensity assay.	MPI	194152	814	neurodegenerative disease	0.586380059223403	8
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	neuropathic pain	0.609134327145434	84
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	neuropathic pain	0.602628172465082	20
Primary qHTS assay for inhibitors of alpha-synuclein gene (SNCA) expression	SNCA	140118	236	neuroticism measurement	0.529970620572164	14
qHTS of alpha-syn Inhibitors	SNCA	368791	501	neuroticism measurement	0.529970620572164	14
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Agonists	D2_agonists	359518	300	neuroticism measurement	0.506410375043145	23
HTS Assay for Allosteric Agonists of the Human D2 Dopamine Receptor: Primary Screen for Agonists	D2_agonists	335652	1779	neuroticism measurement	0.506410375043145	23
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Antagonists	D2_antagonists	362274	1056	neuroticism measurement	0.506410375043145	23
HTS Assay for Allosteric Antagonists of the Human D2 Dopamine Receptor: Primary Screen for Antagonists	D2_antagonists	336308	6862	neuroticism measurement	0.506410375043145	23
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Potentiators	D2_PAMs	357537	806	neuroticism measurement	0.506410375043145	23
HTS Assay for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Primary Screen for Potentiators	D2_PAMs	339887	1178	neuroticism measurement	0.506410375043145	23
Primary screen for KDM5B Histone Demethylases FDH-coupled qHTS	KDM5B	217819	5697	cognitive function measurement	0.516586227462943	16
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	status epilepticus	0.520086420560293	11
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	neuralgia	0.542711443815767	17
Luminescence-based cell-based primary high throughput screening assay to identify agonists of the mouse 5-hydroxytryptamine (serotonin) receptor 2A (HTR2A)	HTR2A	363803	2412	spinal cord disease	0.580512916716499	9
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	neurodevelopmental disorder with seizures and non-epileptic hyperkinetic movements	0.785713138394098	92
Alphascreen assay for small molecules abrogating mHTT-CaM Interaction	HTT	189882	6790	Lopes-Maciel-Rodan syndrome	0.73275567258744	41
Primary qHTS for Identification of Small Molecule Inhibitors of Huntingtin Promoter Activity	HTT	48068	449	Lopes-Maciel-Rodan syndrome	0.73275567258744	41
qHTS Multiplex Assay to Identify Dual Action Probes in a Cell Model of Huntington: Aggregate Formation (GFP)	HTT	220571	2380	Lopes-Maciel-Rodan syndrome	0.73275567258744	41
qHTS Multiplex Assay to Identify Dual Action Probes in a Cell Model of Huntington: Cytoprotection (ATP)	HTT	223611	305	Lopes-Maciel-Rodan syndrome	0.73275567258744	41
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	Neurodevelopmental disorder	0.569435323605255	923
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	Neurodevelopmental disorder	0.569435323605255	923
qHTS Assay for Inhibitors of BAZ2B	BAZ2B_modulators	356826	15709	Neurodevelopmental disorder	0.583262943892584	18
HTS for BAP1 Enzyme inhibitors	BAP1_inhibitors	71016	346	Neurodevelopmental disorder	0.60292127111447	19
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	cancer pain	0.647690739119118	54
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	cancer pain	0.647690739119118	54
HTS for Inhibition of CaV1.3 ICDI/IQ interaction using a live-cell FRET assay Measured in Cell-Based System Using Plate Reader - 7081-01_Inhibitor_SinglePoint_HTS_Activity	CACNA1D_inhibitors	335531	328	intracranial vasospasm	0.520819643474188	6
HTS for Beta-2AR agonists via FAP method	ADRB2_activators	339297	1446	low tension glaucoma	0.520819643474188	6
Luminescence-based cell-based primary high throughput screening assay to identify agonists of the mouse 5-hydroxytryptamine (serotonin) receptor 2A (HTR2A)	HTR2A	363803	2412	cerebral palsy	0.577168330395272	6
Fluorescence-based cell-based primary high throughput screening assay to identify agonists of the human cholinergic receptor, muscarinic 1 (CHRM1)	CHRM1_agonists	359207	1189	cerebral palsy	0.569561257660925	6
Discovery of Novel Allosteric Modulators of the M1 Muscarinic Receptor: Agonist Primary Screen	CHRM1_allosteric_activators	63676	1938	cerebral palsy	0.569561257660925	6
Fluorescence-based cell-based primary high throughput screening assay to identify positive allosteric modulators (PAMs) of the human M1 muscarinic receptor (CHRM1).	CHRM1_PAMs	359207	316	cerebral palsy	0.569561257660925	6
Discovery of novel allosteric modulators of the M1 muscarinic receptor: Antagonist Primary Screen	CHRM1_allosteric_antagonists	63656	2179	cerebral palsy	0.569561257660925	6
Fluorescence-based cell-based primary high throughput screening assay to identify antagonists of the human M1 muscarinic receptor (CHRM1)	CHRM1_antgonists	359207	4555	cerebral palsy	0.569561257660925	6
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	diabetic neuropathy	0.591755915992814	28
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	diabetic neuropathy	0.591755915992814	28
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	diabetic neuropathy	0.5850204572023	73
Primary Cell-based High Throughput Screening assay for activators of the Retinoic Acid Receptor-related orphan receptor A (RORA)	RORA	64908	979	Intellectual disability	0.579381490814232	14
Primary Cell-based High Throughput Screening assay for inhibitors of the Retinoic Acid Receptor-related orphan receptor A (RORA)	RORA	64908	278	Intellectual disability	0.579381490814232	14
uHTS fluorescence polarization assay for the identification of translation initiation inhibitors (PABP)	PABPC1	290915	2982	Intellectual disability	0.522565290992928	6
uHTS identification of microRNA-mediated mRNA deadenylation inhibitors by fluoresence polarization assay	PABPC1	359244	1307	Intellectual disability	0.522565290992928	6
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	Intellectual disability	0.53550223619396	42
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	Intellectual disability	0.53550223619396	42
Luminescence-based cell-based primary high throughput screening assay to identify activators of the function of SWI/SNF related, matrix associated, actin dependent regulator of chromatin, subfamily a, member 2 (SMARCA2, BRM)	SMARCA2	368927	3838	Intellectual disability	0.548715545063125	50
qHTS Assay for Inhibitors of the ERK Signaling Pathway using a Homogeneous Screening Assay	MAPK1	70898	707	Intellectual disability	0.658125365429922	17
qHTS Assay for Inhibitors of the ERK Signaling Pathway using a Homogeneous Screening Assay; Stimulation with EGF	MAPK1	131324	544	Intellectual disability	0.658125365429922	17
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	Intellectual disability	0.507442465199123	6
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	Intellectual disability	0.547021782892492	9
Dyrk1 A HTS Measured in Biochemical System Using Plate Reader - 2124-01_Inhibitor_SinglePoint_HTS_Activity	DYRK1A_inhibitors	310014	1321	Intellectual disability	0.607223771004651	138
High Throughput Imaging Assay for Beta-Catenin	betaCateninTranslocation	193542	587	Intellectual disability	0.675838335815259	63
Acumen qHTS Assay for Inhibitors of the mTORC1 Signaling Pathway in MEF (Tsc2-/-, p53-/-) Cells: Sytravon	MTOR	43989	342	Intellectual disability	0.533070204890202	76
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ1 potassium channels	KCNQ1	305610	3878	Seizure	0.564151783716501	8
Primary cell-based high-throughput screening assay for identification of compounds that potentiate/activate KCNQ1 potassium channels	KCNQ1	305610	1082	Seizure	0.564151783716501	8
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	Seizure	0.742012328791577	104
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	Seizure	0.742012328791577	104
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	Seizure	0.809233289945136	281
Dyrk1 A HTS Measured in Biochemical System Using Plate Reader - 2124-01_Inhibitor_SinglePoint_HTS_Activity	DYRK1A_inhibitors	310014	1321	Seizure	0.549729739789671	12
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS4-Galphao.	GNAO1	218528	711	Abnormality of the nervous system	0.566476400857916	112
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS7-Galphao.	GNAO1	218528	770	Abnormality of the nervous system	0.566476400857916	112
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS8-Galphao.	GNAO1	218528	750	Abnormality of the nervous system	0.566476400857916	112
Inhibitors of Cav3 T-type Calcium Channels: Primary Screen	CACNA1H_inhibitors	104728	4230	Seizure	0.509373379751668	11
uHTS identification of small molecule antagonists of the kappa opioid receptor via a luminescent beta-arrestin assay	OPRK1	290355	265	Chronic pain	0.648225361607717	53
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	Chronic pain	0.666248934967793	527
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	Chronic pain	0.666248934967793	527
Fluorescence-based primary cell-based high throughput screening assay to identify agonists of the Oxytocin Receptor (OXTR).	OXTR	324747	1043	Chronic pain	0.524065327840843	8
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	Chronic pain	0.583510030116279	12
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	Chronic pain	0.609255866556582	15
uHTS identification of small molecule antagonists of the kappa opioid receptor via a luminescent beta-arrestin assay	OPRK1	290355	265	Back pain	0.552121233804408	21
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	Back pain	0.646964767679243	78
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	Back pain	0.646964767679243	78
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	Back pain	0.662533243340019	73
uHTS identification of small molecule antagonists of the kappa opioid receptor via a luminescent beta-arrestin assay	OPRK1	290355	265	Low back pain	0.620861137391453	42
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	Low back pain	0.658909432708107	138
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	Low back pain	0.658909432708107	138
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	Low back pain	0.647073385018578	35
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	Cognitive impairment	0.509396915955061	14
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	Cognitive impairment	0.509396915955061	14
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Cognitive impairment	0.541999650506393	1697
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Cognitive impairment	0.541999650506393	1697
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Cognitive impairment	0.541999650506393	1697
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	Cognitive impairment	0.59152190496849	179
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	Cognitive impairment	0.544595988971466	25
qHTS Assay for Identifying a Potential Treatment of Ataxia-Telangiectasia	ATM_modulators	322361	619	cerebellar ataxia	0.513266557507496	23
Fluorescence-based cell-based primary high throughput screening assay to identify agonists of the human cholinergic receptor, muscarinic 1 (CHRM1)	CHRM1_agonists	359207	1189	amblyopia	0.555091317350212	12
Discovery of Novel Allosteric Modulators of the M1 Muscarinic Receptor: Agonist Primary Screen	CHRM1_allosteric_activators	63676	1938	amblyopia	0.555091317350212	12
Fluorescence-based cell-based primary high throughput screening assay to identify positive allosteric modulators (PAMs) of the human M1 muscarinic receptor (CHRM1).	CHRM1_PAMs	359207	316	amblyopia	0.555091317350212	12
Discovery of novel allosteric modulators of the M1 muscarinic receptor: Antagonist Primary Screen	CHRM1_allosteric_antagonists	63656	2179	amblyopia	0.555091317350212	12
Fluorescence-based cell-based primary high throughput screening assay to identify antagonists of the human M1 muscarinic receptor (CHRM1)	CHRM1_antgonists	359207	4555	amblyopia	0.555091317350212	12
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	Epileptic encephalopathy	0.587761656536964	194
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	Epileptic encephalopathy	0.587761656536964	194
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS4-Galphao.	GNAO1	218528	711	Epileptic encephalopathy	0.721014842095344	71
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS7-Galphao.	GNAO1	218528	770	Epileptic encephalopathy	0.721014842095344	71
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS8-Galphao.	GNAO1	218528	750	Epileptic encephalopathy	0.721014842095344	71
Luminescence-based cell-based primary high throughput screening assay to identify agonists of the mouse 5-hydroxytryptamine (serotonin) receptor 2A (HTR2A)	HTR2A	363803	2412	Parkinson disease	0.636198525212416	78
qHTS assay for re-activators of p53 using a Luc reporter	TP53	321427	201	Parkinson disease	0.511918072179894	141
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54509	528	Parkinson disease	0.511918072179894	141
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54513	338	Parkinson disease	0.511918072179894	141
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	125394	1890	Parkinson disease	0.511918072179894	141
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	124022	1156	Parkinson disease	0.511918072179894	141
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	Parkinson disease	0.613478296323205	1196
Primary qHTS assay for inhibitors of alpha-synuclein gene (SNCA) expression	SNCA	140118	236	Parkinson disease	0.828171049197643	6136
qHTS of alpha-syn Inhibitors	SNCA	368791	501	Parkinson disease	0.828171049197643	6136
Homogeneous Time-Resolved Fluorescence Resonance Energy Transfer (HTRF) Assay	CACNA1B_modulators	292323	567	Parkinson disease	0.517948677875559	11
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Agonists	D2_agonists	359518	300	Parkinson disease	0.685174950061005	842
HTS Assay for Allosteric Agonists of the Human D2 Dopamine Receptor: Primary Screen for Agonists	D2_agonists	335652	1779	Parkinson disease	0.685174950061005	842
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Antagonists	D2_antagonists	362274	1056	Parkinson disease	0.685174950061005	842
HTS Assay for Allosteric Antagonists of the Human D2 Dopamine Receptor: Primary Screen for Antagonists	D2_antagonists	336308	6862	Parkinson disease	0.685174950061005	842
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Potentiators	D2_PAMs	357537	806	Parkinson disease	0.685174950061005	842
HTS Assay for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Primary Screen for Potentiators	D2_PAMs	339887	1178	Parkinson disease	0.685174950061005	842
qHTS of D3 Dopamine Receptor Agonist: qHTS	D3_agonists	407539	2380	Parkinson disease	0.670877516160389	746
qHTS of D3 Dopamine Receptor Antagonist: qHTS	D3_antagonists	364051	9106	Parkinson disease	0.670877516160389	746
qHTS of D3 Dopamine Receptor Potentiators: qHTS	D3_PAMs	407539	2380	Parkinson disease	0.670877516160389	746
Fluorescence polarization assay for PLK1 inhibitors	PLK1	97099	518	Parkinson disease	0.577878323850478	9
qHTS for Inhibitors of PLK1-PDB (polo-like kinase 1 - polo-box domain): Primary Screen	PLK1	364065	10181	Parkinson disease	0.577878323850478	9
Fluorescence-based cell-based primary high throughput screening assay to identify agonists of the human cholinergic receptor, muscarinic 1 (CHRM1)	CHRM1_agonists	359207	1189	Parkinson disease	0.730757536945714	165
Discovery of Novel Allosteric Modulators of the M1 Muscarinic Receptor: Agonist Primary Screen	CHRM1_allosteric_activators	63676	1938	Parkinson disease	0.730757536945714	165
Fluorescence-based cell-based primary high throughput screening assay to identify positive allosteric modulators (PAMs) of the human M1 muscarinic receptor (CHRM1).	CHRM1_PAMs	359207	316	Parkinson disease	0.730757536945714	165
Discovery of novel allosteric modulators of the M1 muscarinic receptor: Antagonist Primary Screen	CHRM1_allosteric_antagonists	63656	2179	Parkinson disease	0.730757536945714	165
Fluorescence-based cell-based primary high throughput screening assay to identify antagonists of the human M1 muscarinic receptor (CHRM1)	CHRM1_antgonists	359207	4555	Parkinson disease	0.730757536945714	165
Glucocerebrosidase	GBA1	48118	549	Parkinson disease	0.812818197273596	1199
Allosteric Modulators of D1 Receptors: Primary Screen	D1	57705	3413	Parkinson disease	0.644792569331339	158
Allosteric Agonists of the Human D1 Dopamine Receptor: qHTS	D1_activators	361330	3713	Parkinson disease	0.644792569331339	158
Antagonist of Human D 1 Dopamine Receptor: qHTS	D1_inhibitors	355805	11440	Parkinson disease	0.644792569331339	158
qHTS for Inhibitors of Tau Fibril Formation, Fluorescence Polarization	MAPT	271402	1048	Parkinson disease	0.62030182859644	1115
qHTS for Inhibitors of Tau Fibril Formation, Thioflavin T Binding	MAPT	267412	5703	Parkinson disease	0.62030182859644	1115
qHTS Assay for Tau Filament Binding	MAPT	69668	1391	Parkinson disease	0.62030182859644	1115
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	Parkinson disease	0.662457377099708	142
Acumen qHTS Assay for Inhibitors of the mTORC1 Signaling Pathway in MEF (Tsc2-/-, p53-/-) Cells: Sytravon	MTOR	43989	342	Parkinson disease	0.512498148428698	185
uHTS identification of small molecule antagonists of the kappa opioid receptor via a luminescent beta-arrestin assay	OPRK1	290355	265	migraine disorder	0.662916077246258	83
Luminescence-based cell-based primary high throughput screening assay to identify agonists of the mouse 5-hydroxytryptamine (serotonin) receptor 2A (HTR2A)	HTR2A	363803	2412	migraine disorder	0.525317902710603	73
QFRET-based biochemical primary high throughput screening assay to identify exosite inhibitors of ADAM10.	ADAM10_inhibitors	369953	2294	Alzheimer disease	0.511119614331058	17
qHTS assay for re-activators of p53 using a Luc reporter	TP53	321427	201	Alzheimer disease	0.511773771609474	12
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54509	528	Alzheimer disease	0.511773771609474	12
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54513	338	Alzheimer disease	0.511773771609474	12
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	125394	1890	Alzheimer disease	0.511773771609474	12
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	124022	1156	Alzheimer disease	0.511773771609474	12
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Alzheimer disease	0.861771076336328	1120
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Alzheimer disease	0.861771076336328	1120
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Alzheimer disease	0.861771076336328	1120
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	Alzheimer disease	0.596827949801317	14
Fluorescence polarization assay for PLK1 inhibitors	PLK1	97099	518	Alzheimer disease	0.577168330395272	6
qHTS for Inhibitors of PLK1-PDB (polo-like kinase 1 - polo-box domain): Primary Screen	PLK1	364065	10181	Alzheimer disease	0.577168330395272	6
Luminescence-based cell-based primary high throughput screening assay to identify agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	991	migraine disorder	0.664498935640487	95
Luminescence-based cell-based primary high throughput screening assay to identify inverse agonists of heterodimerization of the mu 1 (OPRM1) and delta 1 (OPRD1) opioid receptors	OPRM1	335239	695	migraine disorder	0.664498935640487	95
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Agonists	D2_agonists	359518	300	migraine disorder	0.656348969587944	101
HTS Assay for Allosteric Agonists of the Human D2 Dopamine Receptor: Primary Screen for Agonists	D2_agonists	335652	1779	migraine disorder	0.656348969587944	101
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Antagonists	D2_antagonists	362274	1056	migraine disorder	0.656348969587944	101
HTS Assay for Allosteric Antagonists of the Human D2 Dopamine Receptor: Primary Screen for Antagonists	D2_antagonists	336308	6862	migraine disorder	0.656348969587944	101
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Potentiators	D2_PAMs	357537	806	migraine disorder	0.656348969587944	101
HTS Assay for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Primary Screen for Potentiators	D2_PAMs	339887	1178	migraine disorder	0.656348969587944	101
HTS for Beta-2AR agonists via FAP method	ADRB2_activators	339297	1446	migraine disorder	0.66863024609379	163
HTS Assay for Compounds that Act as Agonists of the Vanilloid Receptor 1	TRPV1	316642	617	migraine disorder	0.687070847503308	335
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	Alzheimer disease	0.657913251023908	81
Acumen qHTS Assay for Inhibitors of the mTORC1 Signaling Pathway in MEF (Tsc2-/-, p53-/-) Cells: Sytravon	MTOR	43989	342	Alzheimer disease	0.509978156110572	6
qHTS for Suppressors of FUS Proteinopathy: Primary screen using FUS-linked yeast assay	FUS_asupressors	385746	932	amyotrophic lateral sclerosis	0.820983415810258	893
qHTS of TDP-43 Inhibitors	TARDBP	403703	7150	amyotrophic lateral sclerosis	0.824971871393985	552
qHTS of TDP-43 Inhibitors: NCGC Sytravon Library Screen	TARDBP	45163	203	amyotrophic lateral sclerosis	0.824971871393985	552
Luminescence-based cell-based primary high throughput screening assay to identify agonists of the mouse 5-hydroxytryptamine (serotonin) receptor 2A (HTR2A)	HTR2A	363803	2412	anorexia nervosa	0.557048668979413	32
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Agonists	D2_agonists	359518	300	anorexia nervosa	0.627716787694477	23
HTS Assay for Allosteric Agonists of the Human D2 Dopamine Receptor: Primary Screen for Agonists	D2_agonists	335652	1779	anorexia nervosa	0.627716787694477	23
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Antagonists	D2_antagonists	362274	1056	anorexia nervosa	0.627716787694477	23
HTS Assay for Allosteric Antagonists of the Human D2 Dopamine Receptor: Primary Screen for Antagonists	D2_antagonists	336308	6862	anorexia nervosa	0.627716787694477	23
Beta-Arrestin HTS for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Potentiators	D2_PAMs	357537	806	anorexia nervosa	0.627716787694477	23
HTS Assay for Positive Allosteric Modulators of the Human D2 Dopamine Receptor: Primary Screen for Potentiators	D2_PAMs	339887	1178	anorexia nervosa	0.627716787694477	23
Primary biochemical high-throughput screening assay to measure P97 ATPase inhibition	VCP	217959	923	amyotrophic lateral sclerosis	0.827061532998182	1066
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	Charcot-Marie-Tooth disease type 1A	0.900951611526078	408
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	Charcot-Marie-Tooth disease type 1E	0.861079375757666	69
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Alzheimer disease type 1	0.863489229240414	90
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Alzheimer disease type 1	0.863489229240414	90
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Alzheimer disease type 1	0.863489229240414	90
Alphascreen assay for small molecules abrogating mHTT-CaM Interaction	HTT	189882	6790	Huntington disease	0.731024441538217	7266
Primary qHTS for Identification of Small Molecule Inhibitors of Huntingtin Promoter Activity	HTT	48068	449	Huntington disease	0.731024441538217	7266
qHTS Multiplex Assay to Identify Dual Action Probes in a Cell Model of Huntington: Aggregate Formation (GFP)	HTT	220571	2380	Huntington disease	0.731024441538217	7266
qHTS Multiplex Assay to Identify Dual Action Probes in a Cell Model of Huntington: Cytoprotection (ATP)	HTT	223611	305	Huntington disease	0.731024441538217	7266
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	Charcot-Marie-Tooth disease type 3	0.851523859619184	102
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	hereditary neuropathy with liability to pressure palsies	0.858608783470146	360
qHTS Assay for Identifying a Potential Treatment of Ataxia-Telangiectasia	ATM_modulators	322361	619	ataxia telangiectasia	0.929721108758776	19098
Luminescence-based cell-based primary high throughput screening assay for inhibitors of the orphan nuclear receptor subfamily 0, group B, member 1 (DAX1; NR0B1): repression of SF-1 (NR5A1) activated StAR promoter by full-length DAX-1	NR0B1	368927	4094	X-linked adrenal hypoplasia congenita	0.895131822908991	639
Luminescence-based primary cell-based high throughput screening assay to identify inhibitors of the orphan nuclear receptor subfamily 0, group B, member 1 (DAX1; NR0B1)	NR0B1	343468	3417	X-linked adrenal hypoplasia congenita	0.895131822908991	639
qHTS Assay for Inhibitors of HADH2 (Hydroxyacyl-Coenzyme A Dehydrogenase, Type II)	HSD17B10	72072	2464	HSD10 mitochondrial disease	0.91026921331801	81
qHTS Assay for Inhibitors of HSD17B4, hydroxysteroid (17-beta) dehydrogenase 4	HSD17B10	73912	5649	HSD10 mitochondrial disease	0.91026921331801	81
qHTS assay for re-activators of p53 using a Luc reporter	TP53	321427	201	choroid plexus papilloma	0.555890050810532	14
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54509	528	choroid plexus papilloma	0.555890050810532	14
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53 Null Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	54513	338	choroid plexus papilloma	0.555890050810532	14
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Nonpermissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	125394	1890	choroid plexus papilloma	0.555890050810532	14
qHTS Screen for Compounds that Selectively Target Cancer Cells with p53 Mutations: Cytotoxicity of p53ts Cells at the Permissive Temperature	nonSmallCellLungCarcinomaWithP53Mutations	124022	1156	choroid plexus papilloma	0.555890050810532	14
Inhibitors of Cav3 T-type Calcium Channels: Primary Screen	CACNA1H_inhibitors	104728	4230	childhood absence epilepsy	0.555369110677203	980
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	developmental and epileptic encephalopathy, 1	0.514339544478264	6
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	developmental and epileptic encephalopathy, 1	0.514339544478264	6
Acumen qHTS Assay for Inhibitors of the mTORC1 Signaling Pathway in MEF (Tsc2-/-, p53-/-) Cells: Sytravon	MTOR	43989	342	isolated focal cortical dysplasia type II	0.901441338803717	153
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	parkinsonism-dystonia, infantile	0.809677753063431	616
Modulation of AMPAR-stargazin complexes	AMPAStargazinComplexModulators	40473	1400	intellectual disability, autosomal dominant 10	0.539916981653843	14
Primary biochemical high-throughput screening assay to measure P97 ATPase inhibition	VCP	217959	923	frontotemporal dementia and/or amyotrophic lateral sclerosis 6	0.823020002027243	783
QFRET-based biochemical primary high throughput screening assay to identify exosite inhibitors of ADAM10.	ADAM10_inhibitors	369953	2294	Alzheimer disease 18	0.640966313727322	12
qHTS Assay for Identifying the Cell-Membrane Permeable IMPase Inhibitors: Potentiation with Lithium	IMPA1	205582	727	intellectual disability, autosomal recessive 59	0.572390863323355	12
Luminescence-based cell-based high throughput primary screening assay to identify agonists of nuclear receptor subfamily 2, group E, member 3 (NR2E3)	NR2E3	362026	1281	Joubert syndrome and related disorders	0.564628920710667	36
TR-FRET-based primary biochemical high throughput screening assay to identify agonists of nuclear receptor subfamily 2, group E, member 3 (NR2E3).	NR2E3	314998	380	Joubert syndrome and related disorders	0.564628920710667	36
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	Charcot-Marie-Tooth disease	0.714642276410934	828
Identification of Novel Modulators of Cl- dependent Transport Process via HTS: Primary Screen	SLC12A5	189132	4127	malignant migrating partial seizures of infancy	0.719347083244386	1206
qHTS for Suppressors of FUS Proteinopathy: Primary screen using FUS-linked yeast assay	FUS_asupressors	385746	932	juvenile amyotrophic lateral sclerosis	0.573170704033966	30
Luminescence-based cell-based primary high throughput screening assay for inhibitors of the orphan nuclear receptor subfamily 0, group B, member 1 (DAX1; NR0B1): repression of SF-1 (NR5A1) activated StAR promoter by full-length DAX-1	NR0B1	368927	4094	alternating hemiplegia of childhood	0.637739119574785	216
Luminescence-based primary cell-based high throughput screening assay to identify inhibitors of the orphan nuclear receptor subfamily 0, group B, member 1 (DAX1; NR0B1)	NR0B1	343468	3417	alternating hemiplegia of childhood	0.637739119574785	216
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	infantile epileptic-dyskinetic encephalopathy	0.514339544478264	6
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	infantile epileptic-dyskinetic encephalopathy	0.514339544478264	6
qHTS Assay for Identifying a Potential Treatment of Ataxia-Telangiectasia	ATM_modulators	322361	619	ataxia - telangiectasia variant	0.544159269596901	9
Acumen qHTS Assay for Inhibitors of the mTORC1 Signaling Pathway in MEF (Tsc2-/-, p53-/-) Cells: Sytravon	MTOR	43989	342	cerebral cortical dysplasia	0.508992815545776	120
uHTS identification of small molecule inhibitors of Csn-mediated Deneddylation of Cullin-Ring Ligases, vis a fluorescence polarization assay	COPS5	363840	1401	Joubert syndrome	0.532318314379688	74
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	Charcot-Marie-Tooth disease type 1	0.721305340964187	609
High-Throughput Screening for Modulators of Cytosolic Chaperonin Activity	MARVELD2	362274	1085	nonsyndromic genetic hearing loss	0.520611792557172	38
qHTS Assay for Inhibitors of the Six1/Eya2 Interaction	SIX1	364053	2026	autosomal dominant nonsyndromic hearing loss	0.737234129322474	225
uHTS Luminescent assay for identification of inhibitors of NALP3 in yeast	NLRP3	330392	1295	autosomal dominant nonsyndromic hearing loss	0.558404046933777	123
High-Throughput Screening for Modulators of Cytosolic Chaperonin Activity	MARVELD2	362274	1085	hearing loss, autosomal recessive	0.704448220309956	103
Primary Cell-Based High-Throughput Screening to Identify Agonists of the Sphingosine 1-phosphate receptor 2 (S1P2)	S1PR2	96879	447	hearing loss, autosomal recessive	0.574957700449752	11
Primary Cell-Based High-Throughput Screening to Identify Antagonists of the Sphingosine 1-phosphate receptor 2 (S1P2)	S1PR2	96879	207	hearing loss, autosomal recessive	0.574957700449752	11
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Hereditary cerebral hemorrhage with amyloidosis, Dutch type	0.678931554588663	12
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Hereditary cerebral hemorrhage with amyloidosis, Dutch type	0.678931554588663	12
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Hereditary cerebral hemorrhage with amyloidosis, Dutch type	0.678931554588663	12
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	developmental and epileptic encephalopathy	0.752819952083254	2611
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	developmental and epileptic encephalopathy	0.752819952083254	2611
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS4-Galphao.	GNAO1	218528	711	developmental and epileptic encephalopathy	0.773169282096942	596
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS7-Galphao.	GNAO1	218528	770	developmental and epileptic encephalopathy	0.773169282096942	596
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS8-Galphao.	GNAO1	218528	750	developmental and epileptic encephalopathy	0.773169282096942	596
qHTS for Inhibitors of Glutaminase (GLS)	GLS	405291	844	developmental and epileptic encephalopathy	0.549037961909993	11
Identification of Novel Modulators of Cl- dependent Transport Process via HTS: Primary Screen	SLC12A5	189132	4127	developmental and epileptic encephalopathy	0.624427192659521	1214
HTS for 14-3-3 protein interaction modulators	YWHAG	157962	312	developmental and epileptic encephalopathy	0.57853134759266	33
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	brain injury	0.561120224360147	12
uHTS identification of small molecule modulators of NR3A	GRIN3A	339772	8480	brain injury	0.595686804534385	17
HTS for Beta-2AR agonists via FAP method	ADRB2_activators	339297	1446	brain injury	0.594910589729757	15
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS4-Galphao.	GNAO1	218528	711	neurodevelopmental disorder with involuntary movements	0.877979009956465	90
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS7-Galphao.	GNAO1	218528	770	neurodevelopmental disorder with involuntary movements	0.877979009956465	90
Multiplexed high-throughput screen for small molecule regulators of RGS family protein interactions, specifically RGS8-Galphao.	GNAO1	218528	750	neurodevelopmental disorder with involuntary movements	0.877979009956465	90
Acumen qHTS Assay for Inhibitors of the mTORC1 Signaling Pathway in MEF (Tsc2-/-, p53-/-) Cells: Sytravon	MTOR	43989	342	overgrowth syndrome and/or cerebral malformations due to abnormalities in MTOR pathway genes	0.786905869129326	133
Primary Cell-based High Throughput Screening assay for activators of the Retinoic Acid Receptor-related orphan receptor A (RORA)	RORA	64908	979	intellectual developmental disorder with or without epilepsy or cerebellar ataxia	0.815362549629963	102
Primary Cell-based High Throughput Screening assay for inhibitors of the Retinoic Acid Receptor-related orphan receptor A (RORA)	RORA	64908	278	intellectual developmental disorder with or without epilepsy or cerebellar ataxia	0.815362549629963	102
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	complex neurodevelopmental disorder	0.540500710871587	898
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	complex neurodevelopmental disorder	0.540500710871587	898
Dyrk1 A HTS Measured in Biochemical System Using Plate Reader - 2124-01_Inhibitor_SinglePoint_HTS_Activity	DYRK1A_inhibitors	310014	1321	complex neurodevelopmental disorder	0.696313309749674	190
High Throughput Screening for Cocaine Antagonists: Primary Screen	SLC6A3	108286	1415	Infantile dystonia-parkinsonism	0.751026462125741	122
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Hereditary cerebral hemorrhage with amyloidosis	0.761314869524214	39
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Hereditary cerebral hemorrhage with amyloidosis	0.761314869524214	39
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Hereditary cerebral hemorrhage with amyloidosis	0.761314869524214	39
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	Dejerine-Sottas syndrome	0.890154648768819	101
Cell-based coincidence reporter assay to measure PMP22 gene transcription in S16 Pmp22-F2sN cells - Primary screen	PMP22	42576	834	Roussy-LÃ©vy syndrome	0.59205178605105	42
Primary cell-based high-throughput screening assay for identification of compounds that inhibit KCNQ2 potassium channels	KCNQ2	305609	3405	Spasticity - intellectual disability - X-linked epilepsy	0.514339544478264	6
Primary cell-based high-throughput screening assay for identification of compounds that potentiate KCNQ2 potassium channels	KCNQ2	305600	1644	Spasticity - intellectual disability - X-linked epilepsy	0.514339544478264	6
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Hereditary cerebral hemorrhage with amyloidosis, Piedmont type	0.678595966853222	8
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Hereditary cerebral hemorrhage with amyloidosis, Piedmont type	0.678595966853222	8
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Hereditary cerebral hemorrhage with amyloidosis, Piedmont type	0.678595966853222	8
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Hereditary cerebral hemorrhage with amyloidosis, Iowa type	0.678595966853222	8
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Hereditary cerebral hemorrhage with amyloidosis, Iowa type	0.678595966853222	8
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Hereditary cerebral hemorrhage with amyloidosis, Iowa type	0.678595966853222	8
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Hereditary cerebral hemorrhage with amyloidosis, Italian type	0.678595966853222	8
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Hereditary cerebral hemorrhage with amyloidosis, Italian type	0.678595966853222	8
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Hereditary cerebral hemorrhage with amyloidosis, Italian type	0.678595966853222	8
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Hereditary cerebral hemorrhage with amyloidosis, Flemish type	0.678595966853222	8
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Hereditary cerebral hemorrhage with amyloidosis, Flemish type	0.678595966853222	8
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Hereditary cerebral hemorrhage with amyloidosis, Flemish type	0.678595966853222	8
Primary screen for compounds that activate Alzheimer's amyloid precursor	APP_inhibitors	193400	1987	Hereditary cerebral hemorrhage with amyloidosis, Arctic type	0.678595966853222	8
Primary screen for compounds that inhibit Alzheimer's amyloid precursor protein (APP) translation	APP_inhibitors	193714	1590	Hereditary cerebral hemorrhage with amyloidosis, Arctic type	0.678595966853222	8
qHTS for compounds that reverse cellular toxicity of Amyloid beta (A-beta) peptide in yeast: Primary Screen	APP_inhibitors	404343	257	Hereditary cerebral hemorrhage with amyloidosis, Arctic type	0.678595966853222	8
High-Throughput Screening for Modulators of Cytosolic Chaperonin Activity	MARVELD2	362274	1085	Rare genetic deafness	0.504264981526539	6
MITF Measured in Cell-Based System Using Plate Reader - 2084-01_Inhibitor_SinglePoint_HTS_Activity	MITF	331360	2760	Rare genetic deafness	0.540264391088458	42
AlphaScreen-based biochemical high throughput primary assay to identify inhibitors of microphthalmia-associated transcription factor (MITF)	MITF inhibitors	642362	5830	Rare genetic deafness	0.540264391088458	42
qHTS Assay for Identifying a Potential Treatment of Ataxia-Telangiectasia	ATM_modulators	322361	619	Ataxia-telangiectasia variant	0.645586906054851	11
Epi Absorbance-based biochemical primary high throughput screening assay to identify inhibitors of human tyrosyl-DNA phosphodiesterase 2 (TDP2)	TDP2	369953	1241	Autosomal recessive cerebellar ataxia-epilepsy-intellectual disability syndrome due to TUD deficiency	0.716707964739166	14
qHTS Assay for Inhibitors of the Six1/Eya2 Interaction	SIX1	364053	2026	Branchio-otic syndrome	0.890802361045508	257

Some of these associations have also gone through clinical trials.

graph

Find clinical trials details in table. Use scroll bar or search funtion to select specific drugs, molecular targets or diseases.

table