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- Title
Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer.
- Authors
Ortiz-Urda, S; Lin, Q; Yant, S R; Keene, D; Kay, M A; Khavari, P A
- Abstract
Sustainable correction of severe human genetic disorders of self-renewing tissues, such as the blistering skin disease junctional epidermolysis bullosa (JEB), is facilitated by stable genomic integration of therapeutic genes into somatic tissue stem cells. While integrating viral vectors can achieve this, they suffer from logistical and biosafety concerns. To circumvent these limitations, we used the Sleeping Beauty transposable element to integrate the LAMB3 cDNA into genomes of epidermal holoclones from six unrelated JEB patients. These cells regenerate human JEB skin that is normalized at the level of laminin 5 protein expression, hemidesmosome formation and blistering. Transposon-mediated gene delivery therefore affords an opportunity for stable gene delivery in JEB and other human diseases.
- Subjects
EPIDERMOLYSIS bullosa; SKIN diseases; STEM cells; GENOMES; GENES
- Publication
Gene Therapy, 2003, Vol 10, Issue 13, p1099
- ISSN
0969-7128
- Publication type
Article
- DOI
10.1038/sj.gt.3301978