Generation of three Duchenne muscular dystrophy patient-derived induced pluripotent stem cell (iPSC) lines ICGi002-A, ICGi002-B and ICGi002-C

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 indu...

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Bibliographic Details
Published in:Stem cell research Vol. 48; p. 101941
Main Authors: Valetdinova, K.R., Maretina, M.A., Vyatkin, Y.V., Perepelkina, M.P., Egorova, A.A., Baranov, V.S., Kiselev, A.V., Gershovich, P.M., Zakian, S.M.
Format: Journal Article
Language:English
Published: Elsevier B.V 01-10-2020
Elsevier
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Summary: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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ISSN:1873-5061
1876-7753
DOI:10.1016/j.scr.2020.101941