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Generation of two spinal muscular atrophy (SMA) type I patient-derived induced pluripotent stem cell (iPSC) lines and two SMA type II patient-derived iPSC lines

Valetdinova, K.R. ; Maretina, M.A. ; Kuranova, M.L. ; Grigor'eva, E.V. ; Minina, Y.M. ; Kizilova, E.A. ; Kiselev, A.V. ; Medvedev, S.P. ; Baranov, V.S. ; Zakian, S.M.

Stem cell research, 2019-01, Vol.34, p.101376-101376, Article 101376 [Periódico revisado por pares]

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Título:
Generation of two spinal muscular atrophy (SMA) type I patient-derived induced pluripotent stem cell (iPSC) lines and two SMA type II patient-derived iPSC lines
Autor: Valetdinova, K.R. ; Maretina, M.A. ; Kuranova, M.L. ; Grigor'eva, E.V. ; Minina, Y.M. ; Kizilova, E.A. ; Kiselev, A.V. ; Medvedev, S.P. ; Baranov, V.S. ; Zakian, S.M.
Assuntos: Adult ; Cell Culture Techniques - methods ; Cell Line ; Child ; Humans ; Induced Pluripotent Stem Cells - pathology ; Muscular Atrophy, Spinal - pathology
É parte de: Stem cell research, 2019-01, Vol.34, p.101376-101376, Article 101376
Notas: ObjectType-Article-1
SourceType-Scholarly Journals-1
ObjectType-Feature-2
content type line 23
Descrição: 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.
Editor: England: Elsevier B.V
Idioma: Inglês

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