Article
Dual-AAV delivering split prime editor system for in vivo genome editing.
Molecular therapy : the journal of the American Society of Gene Therapy - 5 Jan 2022
Zhi Shengyao, Chen Yuxi, Wu Guanglan, Wen Jinkun, Wu Jinni, Liu Qianyi, Li Yang, Kang Rui, Hu Sihui, Wang Jiahui, Liang Puping, Huang Junjiu
Abstract excerpt
Prime editor (PE), a new genome editing tool, can generate all 12 possible base-to-base conversions, insertion, and deletion of short fragment DNA. PE has the potential to correct the majority of known human genetic disease-related mutations. Adeno-associated viruses (AAVs), the safe vector widely used in clinics, are not capable of delivering PE (∼6.3 kb) in a single vector because of the limited loading...
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