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Updated: May 16, 2026

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
07:44

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

Published on: September 14, 2019

[Challenge toward gene-therapy using iPS cells for Duchenne muscular dystrophy]

Mitsuo Oshimura1, Yasuhiro Kazuki, Narumi Uno

  • 1Department of Biomedical Science, Institute of Regenerative Medicine and Biofunction, Graduate School of Medical Science, Tottori University.

Rinsho Shinkeigaku = Clinical Neurology
|December 1, 2012
PubMed
Summary

Human artificial chromosomes (HACs) carrying the dystrophin gene successfully corrected genetic deficiencies in Duchenne muscular dystrophy (DMD) models. Engineered cells, including iPS cells and mesoangioblasts, showed dystrophin expression and potential for regenerative therapy.

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