CRISPR-Cas9–mediated therapeutic editing of Rpe65ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis


Dong Hyun Jo1*, Dong Woo Song2*, Chang Sik Cho1, Un Gi Kim2, Kyu Jun Lee2, Kihwang Lee3, Sung Wook Park1†, Daesik Kim4, Jin Hyoung Kim1, Jin-Soo Kim4, Seokjoong Kim2, Jeong Hun Kim1,5,6‡, Jung Min Lee2,7‡


https://youtu.be/uCpO-MjcnyY