Cases tie Zolgensma-class AAV gene therapy to cancer risk after long scientific debate
For about twenty years, scientists argued over whether adeno-associated viruses — the small delivery vehicles used to put new genes into thousands of patients — could, in some cases, drive tumors. STAT News reports that the argument is now closed. A series of cases this year has supplied strong evidence that AAVs can cause cancer, including in gene therapies of the Zolgensma class. Researchers and families now have to weigh that newly confirmed tumor risk against the treatments’ life-saving potential, and the field is under pressure to design safer therapies.
The finding matters especially for spinal muscular atrophy. University of Rochester neurologist Sam Mackenzie said SMA patients will recalculate how much confirmed tumor risk they will accept for gene therapy that can save a life: “It will change the calculus for some patients.” The same evidence raises wider questions for genetic medicine, because AAV platforms are used far beyond a single drug. The concrete news is not a hypothetical warning but case reports that settled roughly two decades of uncertainty about whether these viruses can cause cancer.
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