Generation of the Becker muscular dystrophy patient derived induced pluripotent stem cell line carrying the DMD splicing mutation c.1705-8 T>C.


Journal

Stem cell research
ISSN: 1876-7753
Titre abrégé: Stem Cell Res
Pays: England
ID NLM: 101316957

Informations de publication

Date de publication:
05 2020
Historique:
received: 30 01 2020
revised: 20 03 2020
accepted: 12 04 2020
pubmed: 30 4 2020
medline: 22 6 2021
entrez: 30 4 2020
Statut: ppublish

Résumé

Becker Muscular dystrophy (BMD) is an X-linked syndrome characterized by progressive muscle weakness. BMD is generally less severe than Duchenne Muscular Dystrophy. BMD is caused by mutations in the dystrophin gene that normally give rise to the production of a truncated but partially functional dystrophin protein. We generated an induced pluripotent cell line from dermal fibroblasts of a BMD patient carrying a splice mutation in the dystrophin gene (c.1705-8 T>C). The iPSC cell-line displayed the characteristic pluripotent-like morphology, expressed pluripotency markers, differentiated into cells of the three germ layers and had a normal karyotype.

Identifiants

pubmed: 32348941
pii: S1873-5061(20)30121-5
doi: 10.1016/j.scr.2020.101819
pmc: PMC7264707
pii:
doi:

Substances chimiques

Dystrophin 0

Types de publication

Case Reports Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

101819

Informations de copyright

Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest The authors declare that they are unaware of any conflict of interests associated with this work.

Références

Nature. 2007 Jul 19;448(7151):313-7
pubmed: 17554338
Neurol Clin. 2014 Aug;32(3):671-88, viii
pubmed: 25037084
Nat Protoc. 2013 Jan;8(1):162-75
pubmed: 23257984
Biochim Biophys Acta. 2014 Feb;1838(2):635-42
pubmed: 24021238
Lancet Neurol. 2003 Dec;2(12):731-40
pubmed: 14636778

Auteurs

Davide Rovina (D)

Centro Cardiologico Monzino-IRCCS, Unit of Vascular Biology and Regenerative Medicine, Milan, Italy. Electronic address: davide.rovina@ccfm.it.

Elisa Castiglioni (E)

Centro Cardiologico Monzino-IRCCS, Unit of Vascular Biology and Regenerative Medicine, Milan, Italy.

Francesco Niro (F)

Centro Cardiologico Monzino-IRCCS, Unit of Vascular Biology and Regenerative Medicine, Milan, Italy.

Andrea Farini (A)

Stem Cell Laboratory, Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Unit of Neurology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Centro Dino Ferrari, Milan, Italy.

Marzia Belicchi (M)

Stem Cell Laboratory, Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Unit of Neurology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Centro Dino Ferrari, Milan, Italy.

Elisabetta Di Fede (E)

Medical Genetics, Department of Health Sciences, Università degli Studi di Milano, Milan, Italy.

Cristina Gervasini (C)

Medical Genetics, Department of Health Sciences, Università degli Studi di Milano, Milan, Italy.

Stefania Paganini (S)

Laboratory of Medical Genetics, Fondazione IRCCS Ca' Grande, Ospedale Maggiore Policlinico, Milan, Italy.

Marina Di Segni (M)

Laboratory of Medical Genetics, Fondazione IRCCS Ca' Grande, Ospedale Maggiore Policlinico, Milan, Italy.

Yvan Torrente (Y)

Stem Cell Laboratory, Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Unit of Neurology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Centro Dino Ferrari, Milan, Italy.

Rosaria Santoro (R)

Centro Cardiologico Monzino-IRCCS, Unit of Vascular Biology and Regenerative Medicine, Milan, Italy.

Giulio Pompilio (G)

Centro Cardiologico Monzino-IRCCS, Unit of Vascular Biology and Regenerative Medicine, Milan, Italy; Centro Cardiologico Monzino-IRCCS, Department of Cardiac Surgery, Centro Cardiologico Monzino IRCCS, Milan, Italy; Department of Clinical Sciences and Community Health, Università degli Studi di Milano, Italy.

Aoife Gowran (A)

Centro Cardiologico Monzino-IRCCS, Unit of Vascular Biology and Regenerative Medicine, Milan, Italy.

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Classifications MeSH