Optimization of AAV tools to target Müller glial cells for retinal gene therapy
Daniel Urrutia-Cabrera, Genevieve Huppert, Stephanie Chu, Luozixian Wang, Cat Binh Nguy, Chia Fei Liu, Leszek Lisowski, Chi D. Luu, Jiang-Hui Wang, Sandy S. C. Hung, Alex William Hewitt, Chien‐Ling Huang, Tom Edwards, Keith R G Martin, Raymond C.B. Wong
This study optimizes AAV serotypes and promoters to specifically target Müller glial cells in rats, identifying ShH10Y and GFAP promoter as the most effective combination for in vivo delivery.
bioRxiv · Apr 11, 2026 · PreprintBioengineering