Tag: Transgene

Companion Penn Medicine Studies on AAV-based Gene Therapies in Non-Human Primates Suggest Integration into Human DNA is Unlikely to Drive Cancer Mutations While Offering the Potential for Durable Expression of the Transgene

sarah JonasResearch Results

Gene therapy adeno-associated viruses (AAVs)—viruses that can be engineered to deliver DNA to target cells—are unlikely to cause cancer-triggering insertions in humans or monkeys and may contribute to long-term efficacy, according to new research from the University of Pennsylvania’s Gene Therapy Program (GTP).