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BioAssay: AID 485316

qHTS Assay for Rab9 Promoter Activators: Summary

Niemann Pick Type C (NPC) is a rare neurodegenerative lipidosis that is characterized by lipid storage in the endosomal/lysosomal system. Treatment modalities for this devastating disease are currently non-existent due to the severe obstacles associated with accessing the central nervous system with proteins or genes (Ioannou, 2000). We have developed a new paradigm, termed "Orphan Receptor more ..
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AID: 485316
Data Source: NCGC (RAB9543)
BioAssay Type: Summary, Candidate Probes/Leads with Supporting Evidence
Depositor Category: NIH Molecular Libraries Probe Production Network
BioAssay Version:
Deposit Date: 2010-09-29
Modify Date: 2010-10-04
Target
Depositor Specified Assays
Show more
AIDNameTypeComment
1379Counterscreen for Luciferase (Kinase-Glo TM) InhibitionconfirmatoryLuciferase counterscreen for primary assay.
485297qHTS Assay for Rab9 Promoter ActivatorsconfirmatoryPrimary screen assay.
624117qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Renilla Luciferase Read-Outconfirmatory
720525qHTS Assay for NPC1 Promoter Activators: Hit Validation Using Firefly Luciferase Read-Out for Rab9confirmatory
624501qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Firefly Luciferase Read-Out for Rab9confirmatory
720526qHTS Assay for NPC1 Promoter Activators: Hit Validation Using Renila Luciferase Read-Out for NPC1confirmatory
624493qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Firefly Luciferase Read-Out for NPC1confirmatory
485315qHTS Assay for NPC1 Promoter Activators: Summarysummary
624116qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Renilla Luciferase Read-Out for Rab9confirmatory
720528qHTS Assay for NPC1 Promoter Activators: Hit Validation Using Renila Luciferase Read-Out for Rab9confirmatory
493200qHTS Assay for Rab9 Promoter Activators: Initial hit validation from the primary screenconfirmatory
Description:
NIH Chemical Genomics Center [NCGC]
NIH Molecular Libraries Probe Production centers Network [MLPCN]

MLPCN Grant: MH089537-01
Assay Submitter (PI): Yiannis A. Ioannou

NCGC Assay Overview:
Niemann Pick Type C (NPC) is a rare neurodegenerative lipidosis that is characterized by lipid storage in the endosomal/lysosomal system. Treatment modalities for this devastating disease are currently non-existent due to the severe obstacles associated with accessing the central nervous system with proteins or genes (Ioannou, 2000). We have developed a new paradigm, termed "Orphan Receptor Bypass Therapy" (ORByT), to address these disorders. This paradigm posits the existence of endogenous "suppressor" proteins whose expression can dramatically improve LSD pathogenesis. The goal is the identification/discovery of small chemical compounds that can modulate the expression of these proteins and provide a new treatment modality for these devastating disorders. One such protein whose overexpression reverses aspects of the NPC phenotype is Rab9.
We have developed and optimized a cell based luciferase reporter assay in 1536 well format for the identification of up-regulators of the Rab9 promoter.
Protocol
See related assays for detailed assay protocols.
Comment
Keywords: NIH Roadmap, MLSCN, MLI, MLSMR, qHTS, NCGC

Compound ranking comments will be provided here as small molecule probes are developed and are updated with results in PubChem.
Additional Information
Grant Number: MH089537-01

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