Researchers at Stanford University Medical Center have devised a way to sneak DNA into skin cells taken from people with a potentially deadly genetic skin disorder. These modified cells later formed normal, healthy skin when transplanted onto the skin of mice. The technique, published in the advance online publication of the October issue of the journal Nature Medicine, marks the first time researchers have stably replaced the mutated gene in this disease and introduces a new gene therapy technique that could be useful in a wide range of diseases.
"Were very hopeful," said study leader Paul Khavari, PhD, associate professor of dermatology at Stanford. "This study was performed in mice but it targets grafted human disease tissue."
The technique has advantages that could one day help it treat a variety of diseases. Not only can it accommodate large genes such as those responsible for Duchenes muscular dystrophy and cystic fibrosis, but it causes the gene to integrate into human chromosomes in locations where it will continue to make protein as long as the cell is alive. In other techniques, the inserted DNA does not become part of the chromosome and eventually breaks down or else integrates in positions where the gene gets turned off.
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