10-23脱氧核酶体内转染方法研究进展

Research development of in vivo transfection of 10-23 DNAzyme

10-23脱氧核酶（10-23 DNAzyme，10-23DZ）是近年来利用体外分子进化技术筛选得到的一种具有催化功能的单链DNA分子，能高效、特异催化RNA特定部位的切割反应，实现mRNA水平的基因沉默。目前，10-23DZ已被广泛应用于肿瘤、动脉粥样硬化、感染性疾病等的治疗研究，逐渐成为基因治疗研究的重要手段。如何将其有效地导人机体是10—23DZ进入临床应用的关键问题之一。目前，已有多种递送载体和方法用于10-23DZ的体内转染，如直接注射、电穿孔、脂质体、纳米颗粒、体内表达等。

10-23 DNAzymes （10-23DZ） are a set of single-stranded DNA segments derived from a combinatorial library of sequences by molecular evolution technique in vitro. 10-23DZ can cleave specific sites of any target RNA in an efficient and sequence-specific manner and implement gene silence at mRNA. At present, 10-23DZ has been widely used in the research on gene therapy for cancer, atherosclerosis, infectious diseases, and so on. For this reason , it has become an important tool for the research of gene therapy gradually. How to effectively deliver 10-23DZ to body is one of the key problems for its clinical application. Several delivery means have been developed for in vivo transfection of 10-23DZ, such as direct injection, electroporation, liposomes, nanoparticles, in vivo expression, and so on.