Author

A. Gonzalez-Cordero

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Open access Sep 2026

Pre-clinical validation of AAV-mediated gene therapy for KCNV2 retinopathy improves visual function and expression in mouse and patient models.

It is demonstrated that AAV-mediated gene replacement therapy delivering a codon-optimised human KCNV2 gene subretinally into Kv8.2 knock-out mice significantly restores retinal function, providing the preclinical proof-of-concept for the safety and therapeutic potential of gene therapy for KCNV2 retinopathy.

R. Rashwan, Paula I. Fuller-Carter, Xin-Ru Lim et al. · Molecular Therapy · 0 citations

Open access Sep 2026

Culture media alter retinal organoid physiology promoting AAV transduction and retinal ganglion cell survival.

Human iPSC-derived retinal organoids offer a human-relevant platform for inherited retinal disease (IRD) gene therapy, yet robust AAV transduction in vitro remains challenging. Here we show that culture in BrainPhys™ (BP) medium markedly enhances AAV-mediated gene delivery. Brief BP exposure during the transduction win...

Michelle O'Hara-Wright, Benjamin Y. Lim, Melissa M. Mangala et al. · Gene Therapy · 0 citations