Ever since their successful cloning of the type VII collagen gene, Woodley, along with Keck School of Medicine associate professors of research Mei Chen, Ph.D., and Wei Li, Ph.D., as well as gene therapy expert Nori Kasahara, M.D., Ph.D., from USC's Institute for Genetic Medicine, has been working to insert that gene into cells that are missing it. They have been able to get the collagen gene into both fibroblasts (the cells that normally produce collagen and other fibrous tissues) and keratinocytes (the cells which normally differentiate to form the outmost layer of skin). And, in the Nature Genetics article, they have shown that these cells are capable of expressing type VII collagen and constructing anchoring fibrils in a mouse model.
Producing anchoring fibril structures in an animal, notes Chen, who is the first author on the paper, is a major step forward towards the use of gene therapy to actually treat patients with epidermolysis bullosa.
In subsequent work, Woodley adds, the engineered cells have shown that they are capable of continuing to pump out type VII collagen for at least six months--but so far, they have only done so in lab dishes. The question, of course, is whether they will be able to do the same in mice--and, eventually, in humans.
"I see patients all the time who would definitely benefit from our better understanding of the basic mechanisms of skin biology," Woodley says. "That's the goal: to help the patients who need it. Hopefully, that's what we're doing."
The research published in the Nature Genetics article was supported by grants from the National Institutes of Health.
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Contact: Jon Weiner
jonweine@usc.edu
323-442-2830
University of Southern California
23-Dec-2002