Researchers at the National Human Genome Research Institute (NHGRI) may have taken a major step towards safer gene therapy for patients. In a report in this week’s issue of Science, a research team from NHGRI’s Division of Intramural Research demonstrates for the first time that the genetically engineered mouse virus used in gene therapy trials tends to insert itself at the beginning of genes in the target cell, potentially disrupting the genes’ normal function.
“Gene therapy is a promising field that offers fundamentally new ways of curing human illness,” said NHGRI Director Francis S. Collins, M.D., Ph.D. “A decade of clinical studies has demonstrated the complexity of the biology behind gene therapy and the technical problems researchers have experienced. This study provides insight into one of the most serious, current technical hurdles.”
The discovery may lead to safer gene therapy techniques.
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