³í¹®) CRISPR/Cas9-mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis
• Full title: CRISPR/Cas9-mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis
• Short title: Therapeutic Rpe65 editing by CRISPR/Cas9