Work

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

Year: 2023

Type: article

Abstract: 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.

Source: Stem Cell Research

DOI: 10.1016/j.scr.2023.103207

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

Institutions: Stanford Medicine, Stanford Cardiovascular Institute, Stanford University

Language: English

Topic: Pluripotent Stem Cells Research

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $2,210

Funders: National Institutes of Health, U.S. Food and Drug Administration, National Heart, Lung, and Blood Institute

Awards: G2168952822, G3897007235, G5170875372, G6926568331, Human iPSCs for Elucidating Intercellular Crosstalk Signaling in Dilated Cardiomyopathy, Single-cell Multi-omic Profiling of Drug Responses Using Pooled iPSC-CM Differentiation, Elucidating ECM Signaling in Cardiac Organoids with Machine Learning and Single-cell Multiomics, Elucidating Anthracycline-Induced Cell Type-Specific Cardiovascular Toxicity with CRISPRi/a Screens

OpenAlex ID: W4386827201

FWCI: 0.475

Cites: 5

Cited by: 6

Related to: 10