罕见病的基因治疗研究进展

Advances in Gene Therapy for Rare Diseases

罕见病是发病率很低的一大类少见疾病,其中很多疾病为慢性、严重的遗传病,常常危及生命,多数为基因缺陷导致,缺乏有效的治疗方法。基因治疗是一种新型的治疗方法,可利用载体将遗传物质引入靶细胞,通过纠正或补充缺陷基因来治疗或预防罕见病。本文将概述基因治疗的策略,并详细阐述应用最广泛的腺相关病毒载体在眼病、血液病、神经系统疾病、肌肉疾病治疗中的应用,总结展望我国基因治疗行业的发展前景。

A class of illnesses with a very low incidence is known as rare diseases.Many rare diseases are serious,chronic conditions that frequently pose a threat to life.Most of the rare diseases are caused by genetic disorders and a lack of effective treatments.Gene therapy is a novel therapeutic approach that can be used to correct or replace genetic defects in target cells to treat or prevent rare diseases.In this article,we outlined gene therapy strategies and provided a detailed description of the most popular adeno-associated virus used to treat diseases affecting the eyes,blood,nervous system,and muscles.Finally,we reviewed and predicted the future development possibilities for China's gene therapy market.