使用病毒载体的罕见病基因疗法临床进展

 很大一部分的罕见病由遗传因素决定，难以用普通的小分子或大分子药物治愈，而基因治疗有望从根本上修正人体功能的缺失或异常，给罕见病患者带来改善生活质量的希望。目前许多基因疗法的临床试验正在开展，病毒载体是常用的基因递送方法，本文讨论了用于临床基因递送的多种病毒载体，包括腺相关病毒、逆转录病毒和慢病毒，重点列举了这些病毒在罕见病临床试验中的研究、应用和进展，评价了这些病毒的优缺点，并简述了基因疗法的研究方向及应用前景。

Advances in gene therapy for rare diseases using viral vectors

A large part of rare diseases are caused by genetic factors, making them difficult to be cured using the conventional small or large molecular drugs. Nevertheless, the gene therapy could potentially correct such absence or abnormality of body functions caused by certain genetic disorders, thus bringing better quality of life to those rare-disease patients. Currently, many clinical trials of gene therapies are carried out, and some gene therapies have already been available on the market. The viral vectors are commonly used for the gene delivery. In this paper, a variety of viral vectors for the clinical gene delivery are discussed, including the adeno-associated virus (AAV), the retrovirus, and the lentivirus, focusing on their researches, applications, and developments in clinical trials of rare diseases. Furthermore, the advantages and disadvantages of the viral vectors are evaluated, and the potential applications, as well as the research directions of gene therapies are suggested.