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