CRISPR-mediated correction of skeletal muscle Ca

Humans Dystrophin / genetics Clustered Regularly Interspaced Short Palindromic Repeats CRISPR-Cas Systems Muscular Dystrophy, Duchenne / genetics Muscle, Skeletal / pathology Muscle Fibers, Skeletal / pathology Pluripotent Stem Cells / metabolism
CRISPR Ca(2+) handling Duchenne muscular dystrophy Dystrophin Human pluripotent stem cells

Journal

Neuromuscular disorders : NMD
ISSN: 1873-2364
Titre abrégé: Neuromuscul Disord
Pays: England
ID NLM: 9111470

Informations de publication

Date de publication:
12 2022
Historique:
received: 09 06 2022
revised: 28 10 2022
accepted: 30 10 2022
pubmed: 24 11 2022
medline: 17 12 2022
entrez: 23 11 2022
Statut: ppublish

Résumé

Mutations in the dystrophin gene cause the most common and currently incurable Duchenne muscular dystrophy (DMD) characterized by progressive muscle wasting. Although abnormal Ca

Identifiants

DOI: 10.1016/j.nmd.2022.10.007 PMID: 36418198
pubmed: 36418198
pii: S0960-8966(22)00702-7
doi: 10.1016/j.nmd.2022.10.007
pii:
doi:

Substances chimiques

Dystrophin 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

908-922

Informations de copyright

Copyright © 2022 The Author(s). Published by Elsevier B.V. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest Yung-Yao Lin is the principal investigator and Andrew Tinker is the co-investigator on a research grant from Pfizer. Francesco Muntoni is a scientific advisor of the Pfizer Rare Disease program, which includes Duchenne muscular dystrophy. All other authors declare no competing interests.

Auteurs

Cristina Morera (C)

Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, United Kingdom; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, United Kingdom.

Jihee Kim (J)

Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, United Kingdom; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, United Kingdom.

Amaia Paredes-Redondo (A)

Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, United Kingdom; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, United Kingdom; Centre for Predictive in vitro Model, Queen Mary University of London, Mile End Road, London E1 4NS, United Kingdom.

Muriel Nobles (M)

William Harvey Research Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, United Kingdom.

Denis Rybin (D)

Rare Disease Research Unit, Pfizer, 610 Main Street, Cambridge, MA 02139, USA.

Robert Moccia (R)

Rare Disease Research Unit, Pfizer, 610 Main Street, Cambridge, MA 02139, USA.

Anna Kowala (A)

Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, United Kingdom; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, United Kingdom; Centre for Predictive in vitro Model, Queen Mary University of London, Mile End Road, London E1 4NS, United Kingdom.

Jinhong Meng (J)

UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, United Kingdom.

Seth Garren (S)

Rare Disease Research Unit, Pfizer, 610 Main Street, Cambridge, MA 02139, USA.

Pentao Liu (P)

School of Biomedical Sciences, Stem Cell and Regenerative Medicine Consortium, Li Ka Shing Faculty of Medicine, the University of Hong Kong, Hong Kong, China.

Jennifer E Morgan (JE)

UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, United Kingdom; NIHR Biomedical Research Centre at Great Ormond Street Hospital, Great Ormond Street, London, United Kingdom.

Francesco Muntoni (F)

UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, United Kingdom; NIHR Biomedical Research Centre at Great Ormond Street Hospital, Great Ormond Street, London, United Kingdom.

Nicolas Christoforou (N)

Rare Disease Research Unit, Pfizer, 610 Main Street, Cambridge, MA 02139, USA.

Jane Owens (J)

Rare Disease Research Unit, Pfizer, 610 Main Street, Cambridge, MA 02139, USA.

Andrew Tinker (A)

William Harvey Research Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, United Kingdom.

Yung-Yao Lin (YY)

Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, United Kingdom; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, United Kingdom; Centre for Predictive in vitro Model, Queen Mary University of London, Mile End Road, London E1 4NS, United Kingdom. Electronic address: yy.lin@qmul.ac.uk.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › CRISPR-mediated correction of skeletal muscle Ca
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines membranaires › Dystrophine › CRISPR-mediated correction of skeletal muscle Ca
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Génome › Composants de génome › Séquences répétées en tandem › Clustered regularly interspaced short palindromic repeats › CRISPR-mediated correction of skeletal muscle Ca
questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Systèmes CRISPR-Cas › CRISPR-mediated correction of skeletal muscle Ca
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Dystrophies musculaires › Myopathie de Duchenne › CRISPR-mediated correction of skeletal muscle Ca
questionsmedicales.fr › Tissus › Muscles › Muscle strié › Muscles squelettiques › CRISPR-mediated correction of skeletal muscle Ca
questionsmedicales.fr › Cellules › Cellules musculaires › Fibres musculaires squelettiques › CRISPR-mediated correction of skeletal muscle Ca
questionsmedicales.fr › Cellules › Cellules souches › Cellules souches pluripotentes › CRISPR-mediated correction of skeletal muscle Ca