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Target intelligence / Profile preview
Rab escort protein-1 expression stimulant
- Molecular classification
- Other
01
Overview
Agents or interventions that increase the expression of the CHM gene, which encodes Rab escort protein-1 (REP-1). REP-1 is essential for intracellular trafficking by facilitating the prenylation and membrane targeting of Rab GTPase proteins. Increasing CHM expression is a therapeutic strategy for choroideremia, an X-linked retinal degenerative disease caused by mutations in the CHM gene. Approaches include inhibiting nonsense-mediated decay (NMD) of mutant CHM transcripts and gene augmentation therapy to deliver functional copies of CHM.
- Other names
- CHM protein expression stimulant
02
Mechanism of action
Inhibition of nonsense-mediated decay, exogenous gene delivery
03
Biological functions
Intracellular traffickingProtein prenylationRab GTPase targetingLipid metabolism regulationOxidative stress response regulation
04
Disease associations
ChoroideremiaMetabolic disorders
05
Safety considerations
Off-target effects of NMD inhibitorsImmune response to gene therapy vectorsPotential systemic effects of altered Rab GTPase activity
06
Interacting drugs
Caffeine
3 more in the full profile.
07
Biomarkers
CHM mRNA levelsREP-1 protein levels
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