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Target intelligence / Profile preview
Retinitis Pigmentosa GTPase Regulator (RPGR)
- Target
- RPGR
- Molecular classification
- GTPase regulator, Ciliary protein
01
Overview
Retinitis pigmentosa GTPase regulator (RPGR) is a protein encoded by the RPGR gene, located on the X chromosome in humans. It is primarily known for its essential role in normal vision and is most notably associated with X-linked retinitis pigmentosa (XLRP), a severe inherited retinal degenerative disease. RPGR plays a critical role in regulating cargo trafficking between inner and outer segments of photoreceptors, maintaining ciliary structure and function, and ciliogenesis. Mutations in the RPGR gene are a major cause of XLRP and cone-rod dystrophy.
- Other names
- COD1CORDX1CRDPCDXRP15RP3XLRP3orf15
02
Biological functions
Cargo trafficking regulationCiliogenesisMaintenance of photoreceptor structureRegulation of actin stress filamentsCell contractility during cilia formationRegulation of rhodopsin traffickingPhosphoinositide metabolism
03
Disease associations
X-linked retinitis pigmentosaCone-rod dystrophy
04
Safety considerations
Potential for off-target effects in gene therapy approachesChallenges in delivering therapies specifically to retinal photoreceptors
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