Abstract 2090 The Effect of Cellular Differentiation on Adeno-Associated Viral Vector Genome Status

Gene therapy is a cutting-edge technique used to treat genetic disorders by introducing a functional copy of a mutated or absent gene. This type of treatment requires a vector for delivery of the functional gene, and among the most successful gene therapy vectors is Adeno-associated virus (AAV). Critical to the success of the therapy, AAV vectors are known for their long-term persistence following a single administration of the vector. Specifically, the vector genome persists in the host cell nucleus in an episomal circular state independent of the host cell chromosomes.