Cancer gene therapy: innovations in therapeutic delivery of CRISPR-Cas9

Q3 Pharmacology, Toxicology and Pharmaceutics

Drug Discovery Today: Disease Models Pub Date : 2016-09-01 DOI:10.1016/j.ddmod.2017.02.009

Nicole Lindsay-Mosher , Cathy Su

引用次数: 9

Abstract

Gene therapy has great potential for use in safe and effective cancer treatments, as it can target oncogenic pathways at a molecular level. The recent development of a genome editing system using clustered regularly interspersed palindromic repeats (CRISPR) has made gene therapy for human diseases much more feasible. To realize the full potential of CRISPR and CRISPR-associated systems (Cas) for cancer treatment, however, this gene editing system must be efficiently and safely delivered to cancer cells in vivo. Here we review innovations made in the design of both viral and non-viral vectors to accommodate CRISPR-Cas systems, addressing the challenges of size constraints, immunogenicity, and specificity.

查看原文本刊更多论文

癌症基因治疗:CRISPR-Cas9治疗传递的创新

基因治疗在安全有效的癌症治疗中具有巨大的潜力，因为它可以在分子水平上靶向致癌途径。最近，利用聚集规律穿插回文重复序列(CRISPR)的基因组编辑系统的发展，使人类疾病的基因治疗变得更加可行。然而，为了充分发挥CRISPR和CRISPR相关系统(Cas)在癌症治疗中的潜力，这种基因编辑系统必须在体内高效、安全地传递到癌细胞中。在这里，我们回顾了在设计病毒和非病毒载体以适应CRISPR-Cas系统方面所做的创新，解决了大小限制、免疫原性和特异性的挑战。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Drug Discovery Today: Disease Models Pharmacology, Toxicology and Pharmaceutics-Drug Discovery

自引率

0.00%

发文量

期刊介绍： Drug Discovery Today: Disease Models discusses the non-human experimental models through which inference is drawn regarding the molecular aetiology and pathogenesis of human disease. It provides critical analysis and evaluation of which models can genuinely inform the research community about the direct process of human disease, those which may have value in basic toxicology, and those which are simply designed for effective expression and raw characterisation.