AAV gene therapy for Cockayne syndrome
Ana Rita Batista, Aine C. Scholand, William S. Callahan, McKenna K. Watson, Cassandra M. Sion, Tyler Mola, Kennedy O’Hara, Oliver D. King, Robert M King, Miguel Sena‐Esteves
This study demonstrates that AAV9-CSA gene therapy extends lifespan and distributes human CSA in the brain and heart of Csa-/-;Xpa-/- mice, but also causes adverse effects.
bioRxiv · Jun 6, 2025 · PreprintNeuroscience