Boudes Pol F
152 East Delaware Avenue, Pennington, NJ 08534, USA.
Eur J Intern Med. 2014 Jan;25(1):31-6. doi: 10.1016/j.ejim.2013.09.009. Epub 2013 Oct 12.
Gene therapy, replacing a defective gene by a functional copy, has been in development for more than 40years. Initial efforts involved engineering viral vectors to deliver genes to the appropriate cells. Early successes in severe combined immunodeficiency (SCID) were later derailed by safety issues including host reaction to the vector and gene insertion near promoters that favored secondary leukemia.
Systematic review of the literature using PubMed.gov with key word gene therapy from 1972 to March 2013. Google search with key word gene therapy.
Despite early setbacks, progresses for monogenic diseases continued unabated. Patients with SCIDs have been cured and the first gene therapy has been approved for lipoprotein lipase deficiency. Many clinical research studies are ongoing as part of systematic clinical development program with a view to have more gene therapies approved.
Our review highlights progresses and questions that remain to be answered to make gene therapy an integral part of our therapeutic arsenal.
基因治疗，即用功能拷贝替换缺陷基因，已经发展了40多年。最初的努力包括构建病毒载体将基因传递到合适的细胞。严重联合免疫缺陷（SCID）的早期成功后来因安全问题而受阻，这些问题包括宿主对载体的反应以及基因在启动子附近插入导致继发性白血病。
使用PubMed.gov对1972年至2013年3月期间以“基因治疗”为关键词的文献进行系统综述。使用谷歌以“基因治疗”为关键词进行搜索。
尽管早期遭遇挫折，但单基因疾病的研究进展仍在持续。患有SCID的患者已被治愈，首个基因治疗已被批准用于治疗脂蛋白脂肪酶缺乏症。作为系统性临床开发项目的一部分，许多临床研究正在进行，以期有更多的基因治疗获得批准。
我们的综述强调了基因治疗取得的进展以及为使基因治疗成为我们治疗手段不可或缺的一部分仍有待解答的问题。
