期刊
STEM CELL RESEARCH
卷 34, 期 -, 页码 -出版社
ELSEVIER
DOI: 10.1016/j.scr.2018.101376
关键词
-
资金
- Russian Science Foundation [17-75-10041]
- Russian Science Foundation [17-75-10041] Funding Source: Russian Science Foundation
Spinal muscular atrophy (SMA) is a neuromuscular disease caused by deletion or mutation in SMN1 gene. SMA human induced pluripotent stem cells (iPSCs) represent a useful and valid model for the study of the disorder, as they provide in vitro the target cells. We generated iPSCs from a SMA type I patient and SMA type II patient by using non-integrating episomal plasmid vectors. The resulting iPSCs are episomal-free, express pluripotency markers, display a normal karyotype, retain the mutation (homozygous deletion of SMN1) and are able to differentiate into the three germ layers.
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