Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells

Tasca, F; Brescia, M; Wang, Q; Liu, J; Janssen, JM; Szuhai, K; Goncalves, MAFV

Goncalves, MAFV (通讯作者),Leiden Univ, Dept Cell & Chem Biol, Med Ctr, Einthovenweg 20, NL-2333 ZC Leiden, Netherlands.

NUCLEIC ACIDS RESEARCH, 2022; 50 (13): 7761

Abstract

Targeted chromosomal insertion of large genetic payloads in human cells leverages and broadens synthetic biology and genetic therapy efforts. Yet, obt......

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