Evaluation of CRISPR/Cas9 exon-skipping vector for choroideremia using human induced pluripotent stem cell-derived RPE.
Toshiro IwagawaHiroki MasumotoHitoshi TabuchiKenzaburo TaniBruce R ConklinSumiko WatanabePublished in: The journal of gene medicine (2022)
CHM exon 6-skipping contributed to RPE phagocytosis probably by increasing RAB38 prenylation under oxidative stress.