Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors mediate marked visual restoration
K. M. NISHIGUCHI, K. Fujita, F. Miya, S. Katayama, T. Nakazawa
The paper develops an AAV-based genome editing platform to replace mutations in retinal neurons, achieving significant visual restoration in blind mice.
bioRxiv · Dec 1, 2019 · PreprintBioengineering