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Recent Ex Vivo Gene Therapy through Genome Editing


Zhu Biyun, Li Linxi, Wang Liren, Li Dali*

(Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Institute of Life Medicine, Shanghai 200241, China)
Abstract:

Gene editing technology is a technique for genetically operating specific sites in eukaryotic genomes to achieve specific targeted genetic modification. Because gene editing technology has particular advantages in accurately correcting sequences in the genome, gene editing mediated therapy is being actively developed as treatments for a variety of diseases. With the development of genome editing technologies, such as zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), clustered regularly interspaced short palindromic repeats/CRISPR-associated systems (CRISPR/Cas), gene therapy comes to a new era. This review mainly focuses on recent progress of genome editing technology and its applications in cell mediated ex vivo gene therapy.



CSTR: 32200.14.cjcb.2019.04.0004