Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases.

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作者： Shao-Shuai,Wu ^[1] ; Qing-Cui,Li ^[1] ; Chang-Qing,Yin ^[1] ; Wen,Xue ^[2] ; Chun-Qing,Song ^[3]

作者单位： Key Laboratory of Growth Regulation and Transformation Research of Zhejiang Province, School of Life Sciences, Westlake University, 18 Shilongshan Road, Hangzhou 310024, Zhejiang Province, China. ^[1] RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts. ^[2] Program in Molecular Medicine and Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, Massachusetts. ^[3]

关键词 CRISPR/Cas Gene editing Gene therapy Genetic disease Human disease

主题词动物(Animals);细胞系(Cell Line);遗传性疾病, 先天性(Genetic Diseases, Inborn);人类(Humans)

DOI 10.7150/thno.43360

PMID 32292501

发布时间 2021-04-01

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