title: CRISPR-Cas9: a new and promising player in gene therapy.
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.
authors.affiliation: Department of Gastroenterology, Shanghai East Hospital, Tongji University School of Medicine, Shanghai, China.
authors.forename: Lu
authors.initials: L
authors.lastname: Xiao-Jie
authors.affiliation: The Reproductive Center, Jiangsu Huai'an Maternity and Children Hospital, Huai'an, China.
authors.forename: Xue
authors.initials: X
authors.lastname: Hui-Ying
authors.affiliation: Department of Cardiology, The Fifth People's Hospital of Shanghai, Fudan University, Shanghai, China.
authors.forename: Ke
authors.initials: K
authors.lastname: Zun-Ping
authors.affiliation: Department of Gastroenterology, Shanghai Sixth People's Hospital (South), Shanghai Jiaotong University School of Medicine, Shanghai, China Department of Gastroenterology, Shanghai East Hospital, Tongji University School of Medicine, Shanghai, China.
authors.forename: Chen
authors.initials: C
authors.lastname: Jin-Lian
authors.affiliation: Department of Rehabilitation, The Second People's Hospital of Huai'an, Huai'an, China.
authors.forename: Ji
authors.initials: J
authors.lastname: Li-Juan
mesh_terms: D000818:Animals; D002460:Cell Line; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D016678:Genome; D006801:Humans; D023421:Models, Animal; D017393:RNA Editing
keywords: Clinical genetics; Gene therapy; Infection; Oncology