Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets

Conway, A; Mendel, M; Kim, K; McGovern, K; Boyko, A; Zhang, L; Miller, JC; DeKelver, RC; Paschon, DE; Mui, BL; Lin, PJC; Tam, YK; Barbosa, C; Redelmeier, T; Holmes, MC; Lee, G

Conway, A (reprint author), Sangamo Therapeut, 501 Canal Blvd,Suite A, Richmond, CA 94804 USA.

MOLECULAR THERAPY, 2019; 27 (4): 866

Abstract

It has previously been shown that engineered zinc finger nucleases (ZFNs) can be packaged into adeno-associated viruses (AAVs) and delivered intraveno......

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