Researchers at Stanford University Medical Center have devised a new gene therapy technique that appears to eliminate one of the major health risks linked to gene therapy. The technique, published in the Oct. 15 advanced online edition of the journal Nature Biotechnology, overcomes the need for viral vectors that have plagued gene therapy trials, while retaining the ability to insert therapeutic DNA into specific sites in the chromosomes.
"Our approach provides an alternative that didnt exist before," said Michele Calos, PhD, associate professor of genetics at the School of Medicine and lead author on the study.
The goal of gene therapy is to insert a healthy copy of a gene into a cell where it can take over for a faulty version. If the therapeutic DNA does not integrate into the human chromosome, it produces its protein for a short time before being turned off or broken down within the cell. For a long-term cure, the gene has to wedge itself into a chromosome where it remains indefinitely integrated, getting passed on when the cell divides.
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