Establishment and characterization of three human pluripotent stem cell lines from Charcot-Marie-Tooth disease Type 4B3 patients bearing mutations in MTMR5/Sbf1 gene.


Journal

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

Informations de publication

Date de publication:
22 Oct 2024
Historique:
received: 22 08 2024
revised: 08 10 2024
accepted: 20 10 2024
medline: 27 10 2024
pubmed: 27 10 2024
entrez: 26 10 2024
Statut: aheadofprint

Résumé

Myotubularin-Related Protein 5 (MTMR5) is an inactive, poorly characterized D3-phosphatidylinositol phosphatase. Mutations in MTMR5 have been linked to Charcot-Marie-Tooth Disease Type 4B3 (CMT4B3), a rare, early-onset, recessive peripheral neuropathy. Here, we describe the establishment and validation of three human induced pluripotent stem cell (iPSC) lines derived from unrelated CMT4B3 patients, each harboring homozygous MTMR5/Sbf1 mutations. Current MTMR5

Identifiants

pubmed: 39461113
pii: S1873-5061(24)00297-6
doi: 10.1016/j.scr.2024.103599
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

103599

Informations de copyright

Copyright © 2024. Published by Elsevier B.V.

Déclaration de conflit d'intérêts

Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Mario Saporta reports financial support was provided by Hunter’s CMT4B3 Research Foundation. If there are other authors, they declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Auteurs

Elizabeth H Jacobs (EH)

Medical Scientist Training Program, University of Miami Miller School of Medicine, Miami, FL, USA.

Jacquelyn Schatzman Raposo (J)

Hussmann Institute for Human Genetics and Genomics, University of Miami Miller School of Medicine, Miami, FL, USA.

Annarita Scardamaglia (A)

Department of Neuromuscular Diseases, University College London Queen Square Institute of Neurology, London, United Kingdom.

Fowzan S Alkuraya (FS)

Department of Translational Genomics, Center for Genomic Medicine, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.

Shahriar Nafissi (S)

Iranian Neuromuscular Research Center, Department of Neurology, Tehran University of Medical Sciences, Tehran, Iran.

Henry Houlden (H)

Department of Neuromuscular Diseases, University College London Queen Square Institute of Neurology, London, United Kingdom.

Stephan Zuchner (S)

Hussmann Institute for Human Genetics and Genomics, University of Miami Miller School of Medicine, Miami, FL, USA.

Mario A Saporta (MA)

Department of Neurology, University of Miami Miller School of Medicine, Miami, FL, USA. Electronic address: mas638@med.miami.edu.

Classifications MeSH