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Adeno-associated viruses are popular vectors for delivering gene therapies to patients’ cells because, researchers believed, the DNA these viruses carry rarely inserts into the host genome. But according to results presented last month (December 9) at the American Society of Hematology (ASH) meeting in Orlando, Florida, this assumption may be wrong. In six dogs that were treated with an AAV-based gene therapy for the blood-clotting disorder hemophilia A, all of them carried the therapeutic DNA within their genomes, and in some cases, the genetic material had inserted near genes known to play a role in cell growth, Science reports.
While integration of therapeutic DNA could help ensure long-lasting expression, physician-scientist David Lillicrap of Queen’s University who was not involved in the work tells Science, but it raises concerns about the cancer risk posed by this supposedly safe viral vector. “We don’t know enough yet.”
Currently, hemophilia ...
