Abstract |
Duchenne muscular dystrophy (DMD) is a severe and rapidly progressive hereditary muscular disease with X-linked recessive inheritance, occurring mainly in males. A complete loss of dystrophin resulted from out-of-frame deletion mutations in the DMD gene leads to Duchenne muscular dystrophy. DMD induced pluripotent stem cells (iPSCs) are a suitable cell model to study muscle development and disease mechanisms underlying muscular dystrophy and to screen novel compounds with potential therapeutic effects. We generated iPSCs from a DMD patient using non-integrating episomal plasmid vectors. The obtained iPSC lines showed ESC-like morphology, expression pluripotency markers, displayed a normal karyotype and possessed trilineage differentiation potential.
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Authors | K R Valetdinova, M A Maretina, Y V Vyatkin, M P Perepelkina, A A Egorova, V S Baranov, A V Kiselev, P M Gershovich, S M Zakian |
Journal | Stem cell research
(Stem Cell Res)
Vol. 48
Pg. 101941
(10 2020)
ISSN: 1876-7753 [Electronic] England |
PMID | 32777771
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
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Copyright | Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved. |
Chemical References |
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Topics |
- Cell Differentiation
- Dystrophin
(genetics)
- Humans
- Induced Pluripotent Stem Cells
- Male
- Muscular Dystrophy, Duchenne
(genetics)
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