Sustainable cutaneous gene delivery.
Durable gene delivery to human skin is necessary for lasting correction of human genetic skin disease. Current cutaneous gene-delivery strategies, however, have achieved only transient gene expression, often only within a small percentage of tissue cells. The recent inability to sustain phenotypic correction of human genetic skin disease due to loss of therapeutic gene expression in regenerated epidermal tissue has highlighted this current limitation. In an effort to surmount this problem, we have generated gene delivery vectors that produce more durable gene delivery in human skin tissue in vivo.
Deng
H
H
V.A. Palo Alto Health Care System, CA 94304, USA.
Lin
Q
Q
Khavari
P A
PA
D000818:Animals; D002478:Cells, Cultured; D015967:Gene Expression Regulation, Viral / Q000235:genetics*; D005818:Genetic Engineering; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D015603:Keratinocytes / Q000166:cytology; D008099:Liver / Q000166:cytology / Q000187:drug effects*; D051379:Mice; D008822:Mice, Transgenic; D010641:Phenotype; D011401:Promoter Regions, Genetic; D012190:Retroviridae / Q000235:genetics*; D012867:Skin / Q000166:cytology / Q000378:metabolism; D012871:Skin Diseases / Q000235:genetics* / Q000628:therapy*