STAT+: New tumor cases show common gene therapy tool can come with risk

For two decades, researchers debated whether adeno-associated viruses, the small viruses used to deliver new genes into thousands of patients, could in certain cases drive tumors. That debate is over. A series of cases this year has presented powerful evidence that adeno-associated viruses, or AAVs, can cause cancer, forcing researchers and families to weigh the newly confirmed risk against the technology’s lifesaving potential — and hopefully determine how to design better therapies in the future. “It will change the calculus for some patients,” said Sam Mackenzie, a neurologist at University of Rochester, speaking specifically about spinal muscular atrophy patients. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe