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, David John Ryan, Francesco Catapano, Pierpaolo Ala, Christin Luft, Elizabeth Stevens, Evangelos Konstantinidis, Sandra Louzada, Beiyuan Fu, Amaia Paredes‐Redondo, A. W. Edith Chan, Fengtang Yang, Derek L. Stemple, , 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.
EMBO Reports · Sep 30, 2019Peer-reviewedMolecular Biology