Clinical progress in gene therapy for RPGR-associated X-linked retinitis pigmentosa.
INTRODUCTION X-linked retinitis pigmentosa (XLRP) caused by pathogenic variants in RPGR is among the most severe inherited retinal dystrophies, with childhood-onset nyctalopia followed by progressive visual-field constriction and central visual loss. The purine-rich ORF15 terminal exon has driven sustained development...