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

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

Результат исследования: Научные публикации в периодических изданияхстатья

Аннотация

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.

Язык оригиналаанглийский
Номер статьи101941
ЖурналStem Cell Research
Том48
Ранняя дата в режиме онлайн3 авг 2020
DOI
СостояниеОпубликовано - 1 окт 2020

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