Generation of two induced pluripotent stem cell lines from Duchenne muscular dystrophy patients

Wenqiang Liu; Wenshu Zeng; Xiaohui Kong; Min Htet; Rebecca Yu; Matthew Wheeler; John W. Day; Joseph C. Wu

Stem Cell Research (Oct 2023)

Generation of two induced pluripotent stem cell lines from Duchenne muscular dystrophy patients

Wenqiang Liu,
Wenshu Zeng,
Xiaohui Kong,
Min Htet,
Rebecca Yu,
Matthew Wheeler,
John W. Day,
Joseph C. Wu

Affiliations

Wenqiang Liu: Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA; Department of Medicine, Division of Cardiology, Stanford University School of Medicine, Stanford, CA 94305, USA
Wenshu Zeng: Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA; Department of Medicine, Division of Cardiology, Stanford University School of Medicine, Stanford, CA 94305, USA
Xiaohui Kong: Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA; Department of Medicine, Division of Cardiology, Stanford University School of Medicine, Stanford, CA 94305, USA
Min Htet: Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA
Rebecca Yu: Greenstone Biosciences, Palo Alto, CA 94304, USA
Matthew Wheeler: Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA; Department of Medicine, Division of Cardiology, Stanford University School of Medicine, Stanford, CA 94305, USA
John W. Day: Department of Neurology, Stanford University, Stanford, CA 94305, USA
Joseph C. Wu: Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA; Department of Medicine, Division of Cardiology, Stanford University School of Medicine, Stanford, CA 94305, USA; Corresponding author at: 265 Campus Drive, G1120B, Stanford, CA 94305, USA.

Journal volume & issue: Vol. 72
p. 103207

Abstract

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Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder that leads to death in early adulthood. Patients with DMD have null mutations leading to loss of functional dystrophin protein. Here we generated two DMD induced pluripotent stem cell (iPSC) lines, one with deletion of exon 51 and the other with a single nucleotide nonsense mutation (c.10171C > T). Both lines expressed high levels of pluripotency markers, had the capability of differentiating into derivatives of the three germ layers, and possessed normal karyotypes. These iPSC lines can serve as powerful tools to model DMD in vitro and as a platform for therapeutic development.

Published in Stem Cell Research

ISSN: 1873-5061 (Print); 1876-7753 (Online)
Publisher: Elsevier
Country of publisher: Netherlands
LCC subjects: Science: Biology (General)
Website: https://www.journals.elsevier.com/stem-cell-research

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Abstract

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