血友病非病毒载体基因治疗研究进展

血友病是由于凝血因子的缺陷而导致的血液凝血功能的降低,是一类遗传背景明确且临床特点突出的疾病,基因治疗可能是血友病的唯一希望,非病毒载体因其安全性较高而有逐步发展成为基因治疗研究重点的趋势。非病毒载体主要包括定点整合载体系统、裸质粒、转座子和人工染色体,载体传递系统主要包括靶向纳米微粒、口腔传递型纳米微粒、高压尾静脉注射和电穿孔。在此总结了非病毒载体及载体传递系统两方面阐释治疗血友病的进展。

Research Progress of for Non-viral Gene Therapy for Hemophilia

Hemophilia is an inherited bleeding disorder caused by a deficiency of functional clotting factors in the blood plasma.Hemophilia is well suited for gene therapy since it is due to a single gene defect and the therapeutic window is relatively broad and gene therapy perhaps is the only way to cure this disease.Non-viral vector is expected to dominate the trend of gene therapy due to its higher safety.Non-viral vector mainly includes site-specific integration vector system,naked plasmid,transposon and artificial chromosome;vector delivery system mainly includes:targeted nanoparticles,oral-delivered nanoparticles,and high-pressure tail vein injection and electroporation.Here is to make a summary of the progress in hemophilia treatment from the perspective of non-viral vectors and vector delivery system.