A blood-brain-barrier penetrant AAV gene therapy rescues neurological deficits in mucolipidosis IV mice.
Madison Sangster, Martha Bishop, Yizheng Yao, Jéssica F. Feitor, Sanjid Shahriar, Maxwell Miller, Anil K. Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk
bioRxiv · Nov 3, 2023 · PreprintGenetics