AAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31 iPSC-Derived RPE Cells
Brydon, Elizabeth, Revital Bronstein, Adriana Buskin, Majlinda Lako, Eric Pierce, and Rosario Fernandez-Godino. 2019. “AAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31 IPSC-Derived RPE Cells”. Mol Ther Methods Clin Dev 15: 392-402.