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 ..
Target
| Sequence: ras-related protein Rab-9A [Homo sapiens] Protein Family: Rab GTPase family 9 (Rab9) Gene:RAB9A Related Protein 3D Structures |
Depositor Specified Assays
| AID | Name | Type | Comment |
|---|---|---|---|
| 1379 | Counterscreen for Luciferase (Kinase-Glo TM) Inhibition | confirmatory | Luciferase counterscreen for primary assay. |
| 485297 | qHTS Assay for Rab9 Promoter Activators | confirmatory | Primary screen assay. |
| 624501 | qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Firefly Luciferase Read-Out for Rab9 | confirmatory | |
| 624116 | qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Renilla Luciferase Read-Out for Rab9 | confirmatory | |
| 624117 | qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Renilla Luciferase Read-Out | confirmatory | |
| 485315 | qHTS Assay for NPC1 Promoter Activators: Summary | summary | |
| 493200 | qHTS Assay for Rab9 Promoter Activators: Initial hit validation from the primary screen | confirmatory | |
| 624493 | qHTS Assay for Rab9 Promoter Activators: Hit Validation Using Firefly Luciferase Read-Out for NPC1 | confirmatory |
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.
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.
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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