A human-specific RPGR isoform and a clinically approved Rho/ROCK inhibitor ameliorate defects associated with RPGR dysfunction
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Pathogenic variants in the RPGR gene are the primary cause of photoreceptor degeneration in X-linked retinitis pigmentosa (RP). Previous studies have linked RPGR dysfunction to defects in ciliary structure and actin turnover. RPGR encodes three major isoforms-... ...
