Herzog Roland W, Cao Ou, Srivastava Arun
Department of Pediatrics, University of Florida College of Medicine, 2033 Mowry Road, Gainesville, Florida 32610, USA.
Discov Med. 2010 Feb;9(45):105-11.
Human gene therapy has made substantial progress since the initiation of the first clinical trials 20 years ago. Here, we summarized important applications of gene transfer protocols in the treatment of various human diseases using different viral vectors. Recent successful trials on the treatment of ocular diseases and inherited immune deficiencies are particularly encouraging and have raised hopes that human gene therapy as a standard treatment option will finally become a reality. While immune responses and insertional mutagenesis pose obstacles for this novel form of molecular medicine, continuous progress suggests that a wider range of diseases can be treated with gene therapy in the future.
自20年前首次开展临床试验以来，人类基因治疗已取得了重大进展。在此，我们总结了使用不同病毒载体的基因转移方案在治疗各种人类疾病中的重要应用。近期在眼科疾病和遗传性免疫缺陷治疗方面的成功试验尤其令人鼓舞，并燃起了人们的希望，即人类基因治疗作为一种标准治疗选择最终将成为现实。虽然免疫反应和插入诱变给这种新型分子医学带来了障碍，但持续的进展表明，未来基因治疗可以治疗更广泛的疾病。
