Molecular BiologyEMBO ReportsPeer-reviewed
Targeted gene correction of FKRP by CRISPR/Cas9 restores functional glycosylation of α-dystroglycan in cortical neurons derived from human induced pluripotent stem cells
Jihee Kim, Beatrice Lana, Silvia Torelli…·Yung‐Yao Lin
This paper corrects FKRP mutations in iPSCs using CRISPR/Cas9, restoring functional glycosylation of α-dystroglycan in cortical neurons and validating the role of FKRP in dystroglycanopathies.
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