AAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31(+/-) iPSC-Derived RPE Cells.

Brydon EM, Bronstein R, Buskin A, Lako M, Pierce EA, Fernandez-Godino R
Mol Ther Methods Clin Dev. 2019 Nov 11;15:392-402. doi: 10.1016/j.omtm.2019.10.014. eCollection 2019 Dec 13. PubMed Article

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