Gene therapies can be used to benefit patients: NIH research

By TNT Bureau

Jan 8, 2016: Researchers at the National Institutes of Health have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans.

Researchers at the National Human Genome Research Institute (NHGRI), part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation.

?Effective and safe gene therapies have the potential to dramatically reverse diseases that are life-threatening for affected children,? said NHGRI Scientific Director Dan Kastner, M.D., Ph.D. ?This study is an important step in developing gene therapies that can be safely used to benefit patients.?

Toxic side effects actually are rarely observed by researchers who have designed gene therapies using an adeno-associated virus (AAV) as a vector to deliver the corrected gene to a specific point in the cell?s DNA. AAVs are small viruses that infect humans but do not cause disease. A vector is a DNA molecule of AAV used as a vehicle to carry corrected genetic material into a cell. AAV viruses are uniquely suited for gene therapy applications.

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