《生命科学》 2015, 27(1): 71-82

基因编辑技术在基因治疗中的应用进展

季海艳，朱焕章*

(复旦大学生命科学学院遗传学研究所，上海 200438)

摘　要：摘　要：基因治疗领域面临关键问题之一是缺乏理想的靶向基因修饰技术，而基于锌指核酸酶(zinc finger nucleases， ZFNs)、转录激活子样效应因子核酸酶(transcription activator like effector nucleases， TALENs)、规律性重复短回文序列簇[clustered regularly interspaced short palindromic repeats/CRISPR-associated (cas9)， CRISPR/Cas9] 等基因编辑技术可以对基因组进行高效靶向修饰，因而，基因编辑技术将成为基因治疗领域研究的有用工具。现就基于基因编辑技术在基因治疗中的应用进展做一综述。

Progress of genome editing approaches towards gene therapy

JI Hai-Yan， ZHU Huan-Zhang*

(Institute of Genetics， School of Life Sciences， Fudan University， Shanghai 200438， China)

Abstract: Abstract: One of the key issues towards gene therapy is short of ideal targeted genetic modification technologies. However， gene editing techniques including zinc finger nucleases， transcription activator like effector nucleases and CRISPR/cas9 [clustered regularly interspaced short palindromic repeats/CRISPR-associated (Cas9)] achieve targeted gene modification efficiently， thus gene editing technologies will become a useful tool in the field of gene therapy. A brief overview of recent advances relating gene targeting methods in gene therapy is provided below.