Article
Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model.
Proceedings of the National Academy of Sciences of the United States of America - 14 Feb 2017
Wu Wenbo, Lu Zhiwei, Li Fei, Wang Wenjie, Qian Nannan, Duan Jinzhi, Zhang Yu, Wang Fengchao, Chen Ting
Abstract excerpt
The prokaryotic CRISPR/Cas9 system has recently emerged as a powerful tool for genome editing in mammalian cells with the potential to bring curative therapies to patients with genetic diseases. However, efficient in vivo delivery of this genome editing machinery and indeed the very feasibility of using these techniques in vivo remain challenging for most tissue types. Here, we show that nonreplicable Cas9/sgRNA...
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