Kay M A, Woo S L
Markey Molecular Medicine Center, Department of Medicine, University of Washington, Seattle 98195.
Trends Genet. 1994 Jul;10(7):253-7. doi: 10.1016/0168-9525(94)90173-2.
Most diseases caused by genetic deficiencies could, in theory, be treated by the introduction and expression of a normal gene into an appropriate target tissue. It seems likely that gene therapy strategies for most metabolic disorders will not require strict gene regulation, as a fraction of the normal levels of gene activity could result in amelioration or significant improvement in the clinical outcome. Gene therapy is making rapid progress towards the goal of treating various disorders: here, we summarize the state of gene therapy for metabolic disorders.
理论上，大多数由基因缺陷引起的疾病都可以通过将正常基因导入合适的靶组织并使其表达来进行治疗。对于大多数代谢紊乱疾病而言，基因治疗策略似乎并不需要严格的基因调控，因为基因活性达到正常水平的一部分就可能改善临床结果或使其显著好转。基因治疗在治疗各种疾病的目标上正取得迅速进展：在此，我们总结代谢紊乱疾病的基因治疗现状。
