AAV Gene Therapy Linked to Brain Tumor in Hurler Syndrome Case
TL;DR
A NEJM case report documents a young Hurler syndrome patient who received adeno-associated virus–delivered gene therapy and later developed a walnut-sized brain tumor believed to be caused by an unintended gene mutation; the tumor was removed, cognitive development progressed, highlighting rare but serious risks associated with gene therapies.
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- ASGCT 2026: Rare Instance of AAV Integration into Human Genome Linked to Brain Tumor Genetic Engineering and Biotechnology News
- Scientists link boy’s tumor to gene therapy viruses, in rare finding statnews.com
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