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Large Study Finds Gene Therapy Using AAV Has a Very Low Risk of Causing Liver Cancer

    Introduction Recombinant adeno-associated virus (rAAV) vectors have become the leading platform for in vivo gene therapy, transforming the treatment of numerous inherited diseases, including spinal muscular atrophy (SMA) and hemophilia. Hundreds of AAV-based therapies are currently in clinical development or have reached clinical practice because of their ability to achieve long-term gene expression […]

Genome-Wide Analysis of Recombinant AAV Integration Reveals a Low Risk of Insertional Oncogenesis in the Human Liver

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