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Article

A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy.

Cell reports - 13 Jul 2021

Dhoke Neha R, Kim Hyunkee, Selvaraj Sridhar, Azzag Karim, Zhou Haowen, Oliveira Nelio A J, Tungtur Sudheer, Ortiz-Cordero Carolina, Kiley James, Lu Qi Long, Bang Anne G, Perlingeiro Rita C R

Abstract excerpt

Mutations in the fukutin-related protein (FKRP) gene result in a broad spectrum of muscular dystrophy (MD) phenotypes, including the severe Walker-Warburg syndrome (WWS). Here, we develop a gene-editing approach that replaces the entire mutant open reading frame with the wild-type sequence to universally correct all FKRP mutations. We apply this approach to correct FKRP mutations in induced pluripotent stem (iPS)...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Induced Pluripotent Stem Cells

Share to Muscle Fibers, Skeletal

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Source

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Identifiers

PMID: 34260922
DOI: 10.1016/j.celrep.2021.109360
PMCID: PMC8327854

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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