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Article

Molecular Strategies for RPGR Gene Therapy

4 Sept 2019

Abstract excerpt

Mutations affecting the Retinitis Pigmentosa GTPase Regulator (RPGR) gene are the commonest cause of X-linked and recessive retinitis pigmentosa (RP), accounting for 10%–20% of all cases of RP. The phenotype is one of the most severe amongst all causes of RP, characteristic for its early onset and rapid progression to blindness in young people. At present there is no cure for RPGR-related retinal disease....

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PMID: 31487940
DOI: 10.3390/genes10090674

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