Dog Study Revives Concerns About Virus Used for Gene Therapy

Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.

Written byJef Akst

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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 ...

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Meet the Author

Jef Akst
Jef (an unusual nickname for Jennifer) got her master’s degree from Indiana University in April 2009 studying the mating behavior of seahorses. After four years of diving off the Gulf Coast of Tampa and performing behavioral experiments at the Tennessee Aquarium in Chattanooga, she left research to pursue a career in science writing. As The Scientist's managing editor, Jef edited features and oversaw the production of the TS Digest and quarterly print magazine. In 2022, her feature on uterus transplantation earned first place in the trade category of the Awards for Excellence in Health Care Journalism. She is a member of the National Association of Science Writers.
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