Ontology highlight
ABSTRACT:
SUBMITTER: Siprashvili Z
PROVIDER: S-EPMC2957245 | biostudies-literature | 2010 Oct
REPOSITORIES: biostudies-literature
Siprashvili Zurab Z Nguyen Ngon T NT Bezchinsky Maria Y MY Marinkovich M Peter MP Lane Alfred T AT Khavari Paul A PA
Human gene therapy 20101001 10
In spite of advances in the molecular diagnosis of recessive dystrophic epidermolysis bullosa (RDEB), an inherited blistering disease due to a deficiency of type VII collagen at the basement membrane zone (BMZ) of stratified epithelium, current therapy is limited to supportive palliation. Gene delivery has shown promise in short-term experiments; however, its long-term sustainability through multiple turnover cycles in human tissue has awaited confirmation. To characterize approaches for long-te ...[more]