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CRISPR-Cas9: a new and promising player in gene therapy
  1. Lu Xiao-Jie1,
  2. Xue Hui-Ying2,
  3. Ke Zun-Ping3,
  4. Chen Jin-Lian4,1,
  5. Ji Li-Juan5
  1. 1Department of Gastroenterology, Shanghai East Hospital, Tongji University School of Medicine, Shanghai, China
  2. 2The Reproductive Center, Jiangsu Huai'an Maternity and Children Hospital, Huai'an, China
  3. 3Department of Cardiology, The Fifth People's Hospital of Shanghai, Fudan University, Shanghai, China
  4. 4Department of Gastroenterology, Shanghai Sixth People's Hospital (South), Shanghai Jiaotong University School of Medicine, Shanghai, China
  5. 5Department of Rehabilitation, The Second People's Hospital of Huai'an, Huai'an, China
  1. Correspondence to Dr Chen Jin-Lian, Department of Gastroenterology, Shanghai Sixth People's Hospital (South), Shanghai Jiaotong University School of Medicine, Shanghai, China; wqq_021002{at}163.com or Ji Li-Juan, Department of Rehabilitation, The Second People's Hospital of Huai'an, Huai'an, China. 189{at}whu.edu.cn

Abstract

First introduced into mammalian organisms in 2013, the RNA-guided genome editing tool CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9) offers several advantages over conventional ones, such as simple-to-design, easy-to-use and multiplexing (capable of editing multiple genes simultaneously). Consequently, it has become a cost-effective and convenient tool for various genome editing purposes including gene therapy studies. In cell lines or animal models, CRISPR-Cas9 can be applied for therapeutic purposes in several ways. It can correct the causal mutations in monogenic disorders and thus rescue the disease phenotypes, which currently represents the most translatable field in CRISPR-Cas9-mediated gene therapy. CRISPR-Cas9 can also engineer pathogen genome such as HIV for therapeutic purposes, or induce protective or therapeutic mutations in host tissues. Moreover, CRISPR-Cas9 has shown potentials in cancer gene therapy such as deactivating oncogenic virus and inducing oncosuppressor expressions. Herein, we review the research on CRISPR-mediated gene therapy, discuss its advantages, limitations and possible solutions, and propose directions for future research, with an emphasis on the opportunities and challenges of CRISPR-Cas9 in cancer gene therapy.

  • Clinical genetics
  • Gene therapy
  • Infection
  • Oncology

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