Braun-Falco M, Hallek M
Institut für Biochemie, Genzentrum, Ludwig-Maximilians-Universität, München.
Hautarzt. 1998 Jul;49(7):536-44. doi: 10.1007/s001050050785.
The skin is an attractive target tissue for gene therapy. Because of its accessibility, it is suitable for in vivo as well as for ex vivo gene delivery. The ability to monitor the treatment site is an advantage for safety considerations. The epidermis is a self-renewing tissue, which provides the possibility of achieving long-term treatment through gene transfer into keratinocyte stem cells. This opens new perspectives for the treatment of dermatological and even systemic diseases. The possibility for corrective intervention in disorders with inherited gene defects appears particularly attractive.
皮肤是基因治疗中一个颇具吸引力的靶组织。因其易于接近，它既适用于体内基因递送，也适用于体外基因递送。能够监测治疗部位是出于安全考虑的一个优势。表皮是一种自我更新的组织，这使得通过将基因转移到角质形成干细胞中实现长期治疗成为可能。这为皮肤病甚至全身性疾病的治疗开辟了新的前景。对具有遗传性基因缺陷的疾病进行纠正干预的可能性显得尤为诱人。
