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Target intelligence / Profile preview
Retinitis Pigmentosa GTPase Regulator Protein (RPGR)
- Target
- RPGR
- Molecular classification
- GTPase regulator
01
Overview
RPGR (Retinitis pigmentosa GTPase regulator) is a protein encoded by the *RPGR* gene, which is essential for normal vision. The protein plays a critical role in the function of cilia and is particularly important for photoreceptor cells, regulating ciliary function necessary for sensory perception. Mutations in RPGR are linked to various retinal diseases, most notably X-linked retinitis pigmentosa.
02
Mechanism of action
N/A
03
Biological functions
CiliogenesisCargo traffickingPhototransduction supportIntracellular transport regulation
04
Disease associations
X-linked retinitis pigmentosaCone-rod dystrophyMacular degenerationCiliopathy
05
Safety considerations
Potential for off-target effects in gene therapy approachesChallenges in delivering gene therapies effectively to retinal cells
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