AAVs Can Cause Tumors, Ending Long Debate — SkimNews

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- Adeno-associated viruses (AAVs) have been shown to cause tumors in new patient cases this year, ending a two-decade debate over their potential carcinogenicity.
- Researchers now face the challenge of balancing AAVs’ lifesaving therapeutic benefits against their newly confirmed cancer risk, particularly in treating genetic diseases.
- Sam Mackenzie, a neurologist at University of Rochester, stated the findings will change treatment decisions, especially for spinal muscular atrophy patients.
- Gene therapy developers must now work to design safer vectors, as the confirmed tumor risk complicates regulatory and clinical pathways for AAV-based treatments.
Why it matters: For patients with conditions like spinal muscular atrophy, the confirmed cancer risk from AAVs makes treatment decisions more complex, altering the benefit-risk calculation that families and doctors must weigh. This shifts the development trajectory for gene therapies that rely on AAVs, a platform used in thousands of patients.
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