This study optimizes AAV serotypes and promoters to specifically target Muller glial cells in rats, identifying ShH10Y and GFAP promoter as the most effective combination for in vivo delivery.

Molecular Therapy Advances · Jul 3, 2026Peer-reviewedMolecular Biology

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

Engineering single-AAV CRISPR-Cas13 RNA base editors for treatment of inherited retinal diseases

Satheesh Kumar, Heng-Ai Chang, Deborah Aubin, Yi-Wen Hsiao, Alicia A. Brunet, Jun Yang, Liuhui Huang, Chi D. Luu, Alex William Hewitt, Fan Li, Lewis E Fry, Lívia S. Carvalho, Anai Gonzalez-Cordero, Guei‐Sheung Liu

This paper develops an RNA base editor using CRISPR-Cas13 and ADAR2DD to correct mutations in inherited retinal diseases, showing up to 60% editing efficiency in vitro and partial rescue in mouse models.

bioRxiv · Aug 6, 2025 · PreprintBioengineering

Knockout of AMD-associated gene POLDIP2 reduces mitochondrial superoxide in human retinal pigment epithelial cells

Tu Nguyen, Daniel Urrutia-Cabrera, Luozixian Wang, Jarmon G. Lees, Jiang-Hui Wang, Sandy S. C. Hung, Alex William Hewitt, Thomas L. Edwards, Samuel McLenachan, Fred Kuanfu Chen, Shiang Yong Lim, Chi D. Luu, Robyn H. Guymer, Raymond C.B. Wong

This study shows that knocking out POLDIP2 in human RPE cells reduces mitochondrial superoxide levels, linking POLDIP2 to oxidative stress in AMD.

Aging · Feb 16, 2023Peer-reviewedNeuroscience