基因编辑在艾滋病治疗中的研究进展

Gene Therapy Strategies for the Treatment of HIV/AIDS

随着HIV病毒学研究的不断深入,传统抗反转录病毒治疗在HIV病毒清除和疾病治愈中的局限性日益凸显,因此通过基因编辑达到HIV病毒控制甚至完全治愈的策略倍受重视。目前基因编辑的研究主要包括以下两个方面:RNA水平的编辑技术和DNA水平的编辑技术。近年来包括锌指核酶技术(zinc finger nucleases,ZFN),转录激活样效应因子技术(transcription activatorlike effector nucleases,TALEN),成簇规律间隔短回文重复序列技术(clustered regulatory interspaced short palindromic repeat,CRISPR)和Ng Ago-g DNA技术在内的DNA编辑技术进展迅猛,并在HIV治疗中取得了长足的发展。本文比较了不同DNA水平编辑方法的特点和差异,综述了新一代基因编辑技术在HIV治疗中的进展以及潜在的医学应用前景。

With the further understanding of the molecular virological mechanisms of HIV pathogenesis, the limitation of traditional antiretroviral therapy for a cure of HIV is appearing. Therefore, gene therapy has attracted more and more attention. Currently, two kinds ofgene editing strategies for gene therapy were based on RNA and DNA respectively. The latter includes ZFN （zinc finger nucleases）, TALEN（transcription activator-like effector nucleases）, CRISPR（clustered regulatory interspaced short palindromic repeat） and NgAgo-gDNA, which lead the development of gene editing and achieve great success in HIV therapy. In this review, we first compare the specialty and distinction between different DNA editing methods, and then summarize the advances and prospects of these new methods in HIV gene therapy.