Limited time window for retinal gene therapy in a preclinical model of ciliopathy
Poppy Datta, Avri Ruffcorn, Seongjin Seo
This study shows that retinal gene therapy in a ciliopathy mouse model is most effective when administered early, with efficacy decreasing as treatment is delayed.
Human Molecular Genetics · Jun 16, 2020Peer-reviewedNeuroscience