Khavari P A
Dermatology Service, V.A. Palo Alto Health Care System, CA 94304, USA.
Mol Med Today. 1997 Dec;3(12):533-8. doi: 10.1016/S1357-4310(97)01143-X.
The skin represents a site for treatment of cutaneous and systemic disease and is the most accessible somatic tissue for therapeutic gene transfer in humans. Monogenic hereditary skin diseases, such as ichthyosis and epidermolysis bullosa subtypes, and disorders characterized by low levels of polypeptides in the systemic circulation, are current central foci of efforts in cutaneous-gene transfer. Additional efforts center on the treatment of wounds and malignancies. Recent developments in models of gene delivery to the skin underscore key challenges that must be met before successful treatment of human disease by cutaneous gene delivery can be achieved.
皮肤是治疗皮肤疾病和全身性疾病的部位,也是人类治疗性基因转移最易触及的体细胞组织。单基因遗传性皮肤病,如鱼鳞病和大疱性表皮松解症亚型,以及以全身循环中多肽水平较低为特征的疾病,是目前皮肤基因转移研究的核心焦点。其他研究集中在伤口和恶性肿瘤的治疗上。皮肤基因递送模型的最新进展凸显了在通过皮肤基因递送成功治疗人类疾病之前必须应对的关键挑战。