基因治疗在儿童遗传性肾脏病中的研究进展  

Advances on gene therapy for inherited kidney diseases in children

作　　者：孙蕾(综述)[1] 黄文彦(审校)[1] Sun Lei;Huang Wenyan(Department of Nephrology and Rheumatology,Shanghai Children′s Hospital,Children′s Hospital of Shanghai Jiaotong University,Shanghai 200062,China)

机构地区：[1]上海市儿童医院上海交通大学附属儿童医院肾脏风湿科,200062

出　　处：《国际儿科学杂志》2020年第2期73-76,共4页International Journal of Pediatrics

基　　金：上海市卫生和计划生育委员会科研课题青年项目(20164Y0192)。

摘　　要：基因治疗目前已在多个疾病领域发挥重要作用.随着致病基因的明确,基因治疗成为包括Alport综合征、多囊肾、Fabry病等在内的多种儿童遗传性肾脏病精准治疗的新选择.然而,基因治疗的临床应用仍面临安全性、有效性及伦理问题等巨大挑战.该文就基因治疗的发展、在儿童常见遗传性肾脏病中的应用进展及面临的问题进行综述.Gene therapy has enjoyed a renaissance with recent successes in the treatment of many diseases.The further studies had identified the defected gene of inherited kidney diseases,just like Alport syndrome,polycystic kidney and Fabry′s disease,and make it possible for gene therapy.However,the clinical application still faces enormous challenges such as safety,effectiveness and ethical issues.In this review,we summarized the development and the treatment of gene therapy for genetic kidney diseases.Finally,we discussed the future development prospects of gene editing therapy.

关 键 词：基因治疗 儿童 遗传性肾脏病 

分 类 号：R73[医药卫生—肿瘤]