Hengge U R, Bardenheuer W
Department of Dermatology, University of Düsseldorf, Germany.
Am J Med Genet C Semin Med Genet. 2004 Nov 15;131C(1):93-100. doi: 10.1002/ajmg.c.30038.
Significant progress has been made during the past decade in corrective gene therapy of the skin. This includes advances in vector technology, targeted gene expression, gene replacement, gene correction, and the availability of appropriate animal models for a variety of candidate diseases. While non-viral integration of large genes such as essential basement membrane proteins has been mastered, new challenges such as the control of immune responses lie ahead of the research community. Among the first skin diseases, patients with junctional epidermolysis bullosa (JEB) and xeroderma pigmentosum (XP) will enter clinical trials.
在过去十年中，皮肤矫正基因治疗取得了重大进展。这包括载体技术、靶向基因表达、基因替代、基因校正方面的进展，以及针对各种候选疾病的合适动物模型的可用性。虽然已经掌握了如必需基底膜蛋白等大基因的非病毒整合，但免疫反应控制等新挑战仍摆在研究界面前。在首批皮肤病中，交界性大疱性表皮松解症（JEB）和着色性干皮病（XP）患者将进入临床试验。
