Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9

Liu, ZQ; Chen, SY; Xie, WH; Song, YN; Li, JZ; Lai, LX; Li, ZJ

Li, ZJ (通讯作者)，Jilin Univ, Coll Anim Sci, Key Lab Zoonosis Res, Minist Educ, Changchun 130062, Peoples R China.;Lai, LX (通讯作者)，Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangdong Prov Key Lab Stem Cell & Regenerat Med, CAS Key Lab Regenerat Biol,Guangzhou Inst Biomed, Guangzhou 510530, Peoples R China.

MOLECULAR THERAPY, 2022; 30 (1): 256

Abstract

Compact CRISPR-Cas9 systems that can be packaged into an adeno-associated virus (AAV) show promise for gene therapy. However, the requirement of proto......

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