Molecular Therapy - Methods & Clinical Development
Volume 15, 13 December 2019, Pages 392-402
Original ArticleAAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31+/− iPSC-Derived RPE Cells
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open access
Graphical Abstract
Keywords
PRPF31
RPE
phagocytosis
haploinsuficiency
gene therapy
AAV
cilia
microvilli
iPSC-RPE
Cited by (0)
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These authors contributed equally to this work.
© 2019 The Author(s).