STAT+: Zolgensma tumor cases show risks of a common gene therapy tool

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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 life-saving 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. 

Continue to STAT+ to read the full story…

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