Generation of CHOPe003-A ESC line to study an ACTG2 variant affecting smooth muscle development and function.


Journal

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

Informations de publication

Date de publication:
09 2023
Historique:
received: 15 07 2023
accepted: 21 08 2023
medline: 8 9 2023
pubmed: 29 8 2023
entrez: 29 8 2023
Statut: ppublish

Résumé

Dysfunction of visceral smooth muscle ("visceral myopathy") impairs bowel, bladder, and uterine function. Symptoms of this life-threatening condition include massive intestinal distension with slow transit, vomiting, feeding intolerance, growth failure, poor bladder emptying, and difficult vaginal delivery. The most common genetic cause of visceral myopathy is a heterozygous point mutation (R257C) in gamma smooth muscle actin (ACTG2). We genetically modified the WAe0009-A human embryonic stem cell line to carry the c.769C>T p.R257C/+ mutation. This cell line will facilitate studies of how the ACTG2 R257C heterozygous variant affects smooth muscle development and function.

Identifiants

pubmed: 37643495
pii: S1873-5061(23)00172-1
doi: 10.1016/j.scr.2023.103186
pmc: PMC10509821
mid: NIHMS1929378
pii:
doi:

Substances chimiques

ACTG2 protein, human 0
Actins 0

Types de publication

Journal Article Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

103186

Subventions

Organisme : NIDDK NIH HHS
ID : R01 DK128282
Pays : United States

Informations de copyright

Copyright © 2023 The Authors. Published by Elsevier B.V. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Références

Stem Cell Reports. 2021 Nov 9;16(11):2617-2627
pubmed: 34653400
Methods Mol Biol. 2014;1185:181-94
pubmed: 25062629
Stem Cell Res. 2019 Jan;34:101361
pubmed: 30611021
Curr Protoc. 2022 Nov;2(11):e590
pubmed: 36426905
Nat Commun. 2023 May 6;14(1):2628
pubmed: 37149717

Auteurs

Sohaib K Hashmi (SK)

Department of Pediatrics, The Children's Hospital of Philadelphia Research Institute, Abramson Research Center, 3615 Civic Center Blvd, Philadelphia, PA 19104, United States; Perelman School of Medicine at the University of Pennsylvania, 3400 Civic Center Blvd, Philadelphia, PA 19104, United States; Department of Bioengineering, The University of Pennsylvania School of Engineering and Applied Science, 220 S 33rd St, Philadelphia, PA 19104, United States; Department of Internal Medicine, Hospital of the University of Pennsylvania, 3400 Civic Center Blvd, Philadelphia, PA 19104, United States.

Sabine Schneider (S)

Department of Pediatrics, The Children's Hospital of Philadelphia Research Institute, Abramson Research Center, 3615 Civic Center Blvd, Philadelphia, PA 19104, United States; Perelman School of Medicine at the University of Pennsylvania, 3400 Civic Center Blvd, Philadelphia, PA 19104, United States; Department of Neurology, Hospital of the University of Pennsylvania, 3400 Spruce Street, Philadelphia, PA 19104, United States.

Alyssa L Gagne (AL)

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.

Jean Ann Maguire (JA)

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.

Sierra Anderson (S)

Department of Pediatrics, The Children's Hospital of Philadelphia Research Institute, Abramson Research Center, 3615 Civic Center Blvd, Philadelphia, PA 19104, United States.

Paul Gadue (P)

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States; Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, United States.

Robert O Heuckeroth (RO)

Department of Pediatrics, The Children's Hospital of Philadelphia Research Institute, Abramson Research Center, 3615 Civic Center Blvd, Philadelphia, PA 19104, United States; Perelman School of Medicine at the University of Pennsylvania, 3400 Civic Center Blvd, Philadelphia, PA 19104, United States; Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, United States.

Deborah L French (DL)

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States; Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, United States. Electronic address: frenchd@email.chop.edu.

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Classifications MeSH