Greenhalgh D A, Rothnagel J A, Roop D R
Department of Cell Biology and Dermatology, Baylor College of Medicine, Houston, Texas 77030.
J Invest Dermatol. 1994 Nov;103(5 Suppl):63S-69S. doi: 10.1111/1523-1747.ep12399070.
Important advances have been made within the past several years in understanding diseases at the molecular and cellular levels, which may enable the application of somatic gene therapy to a wide variety of genetic and acquired diseases. The initial clinical trials involving somatic gene therapy have demonstrated that gene transfer into human subjects can be performed safely and with public acceptance. This review focuses on use of the epidermis as a target tissue for gene therapy and assesses various delivery systems for both ex vivo and in vivo approaches. In addition, we discuss candidate diseases that may be amenable to epidermal gene therapy and the advantages of employing transgenic mouse model systems to test the efficacy of a given gene therapy prior to clinical trials.
在过去几年里，我们在分子和细胞水平上对疾病的认识取得了重要进展，这可能使体细胞基因治疗能够应用于多种遗传性和后天性疾病。最初涉及体细胞基因治疗的临床试验表明，将基因导入人体受试者可以安全地进行，并且得到公众认可。本综述重点关注将表皮作为基因治疗的靶组织，并评估用于离体和体内方法的各种递送系统。此外，我们还讨论了可能适合表皮基因治疗的候选疾病，以及在临床试验之前利用转基因小鼠模型系统来测试特定基因治疗效果的优势。
