Outcomes of Progranulin Gene Therapy in the Retina are Dependent on Time of Delivery
Emilia A. Zin, Daisy Han, Jennifer Tran, Nikolas Morrison‐Welch, Meike Visel, Mervi Kuronen, John G. Flannery
This study shows that progranulin gene therapy in a CLN11 mouse model is time-sensitive, with early administration reducing retinal thinning but no benefit at later ages.
Molecular Therapy — Methods & Clinical Development · May 29, 2021Peer-reviewedNeuroscience