Development of Genetic Therapies for the Hemidesmosomal Subtypes of Junctional Epidermolysis Bullosa
Abstract
The overall goal of the studies in our proposal is to establish the feasibility of delivering corrective genes to chronically wounded skin, using JEB keratinocytes as an in vitro model system, and address the issues of sustained targeted temporal and cell-type specific expression. The next step in the transfer of this technology to chronically wounded skin will be the application of ex vivo gene delivery by grafting of genetically engineered keratinocytes. The data produced in this study will provide results that demonstrate the safety, efficacy and feasibility of a genetic therapy for wound healing using JEB as a cell culture model system. It is anticipated that these results may be directly relevant to the development of novel wound-care treatment modalities which synthetically recapitulate basement membrane components, particularly in light of the recent success demonstrated by enhanced wound healing using genetically-engineered "bandages" of keratinocytes expressing genes of interest.
Document Details
- Document Type
- Technical Report
- Publication Date
- Nov 01, 2001
- Accession Number
- ADA410899
Entities
People
- Angela M. Christiano
Organizations
- Columbia University