NeurosciencebioRxivPreprint
AAV gene therapy for Cockayne syndrome
Ana Rita Batista, Aine C. Scholand, William S. Callahan…·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.
DOI 10.1101/2025.06.06.658349