Gorell Emily, Nguyen Ngon, Lane Alfred, Siprashvili Zurab
Department of Dermatology, Stanford School of Medicine, Palo Alto, California 94305.
Cold Spring Harb Perspect Med. 2014 Apr 1;4(4):a015149. doi: 10.1101/cshperspect.a015149.
The skin possesses qualities that make it desirable for gene therapy, and studies have focused on gene therapy for multiple cutaneous diseases. Gene therapy uses a vector to introduce genetic material into cells to alter gene expression, negating a pathological process. This can be accomplished with a variety of viral vectors or nonviral administrations. Although results are promising, there are several potential pitfalls that must be addressed to improve the safety profile to make gene therapy widely available clinically.
皮肤具有一些特质，使其适用于基因治疗，并且已有研究聚焦于多种皮肤疾病的基因治疗。基因治疗利用载体将遗传物质导入细胞以改变基因表达，从而消除病理过程。这可以通过多种病毒载体或非病毒给药方式来实现。尽管结果很有前景，但仍有几个潜在的问题需要解决，以改善安全性，使基因治疗能够在临床上广泛应用。
