Article
In vivo genome editing via CRISPR/Cas9-mediated homology-independent targeted integration for Bietti crystalline corneoretinal dystrophy treatment.
Nature communications - 6 May 2024
Meng Xiang, Jia Ruixuan, Zhao Xinping, Zhang Fan, Chen Shaohong, Yu Shicheng, Liu Xiaozhen, Dou Hongliang, Feng Xuefeng, Zhang Jinlu, Wang Ni, Xu Boling, Yang Liping
Abstract excerpt
Bietti crystalline corneoretinal dystrophy (BCD) is an autosomal recessive chorioretinal degenerative disease without approved therapeutic drugs. It is caused by mutations in CYP4V2 gene, and about 80% of BCD patients carry mutations in exon 7 to 11. Here, we apply CRISPR/Cas9 mediated homology-independent targeted integration (HITI)-based gene editing therapy in HEK293T cells, BCD patient derived iPSCs, and...
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