Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in
Biological sciences
Neuroscience
Pharmacology
Journal
iScience
ISSN: 2589-0042
Titre abrégé: iScience
Pays: United States
ID NLM: 101724038
Informations de publication
Date de publication:
21 Jul 2023
21 Jul 2023
Historique:
received:
17
04
2023
revised:
15
05
2023
accepted:
13
06
2023
medline:
3
8
2023
pubmed:
3
8
2023
entrez:
3
8
2023
Statut:
epublish
Résumé
There is no approved therapy for Becker muscular dystrophy (BMD), a genetic muscle disease caused by in-frame dystrophin deletions. We previously developed the dissociative corticosteroid vamorolone for treatment of the allelic, dystrophin-null disease Duchenne muscular dystrophy. We hypothesize vamorolone can treat BMD by safely reducing inflammatory signaling in muscle and through a novel mechanism of increasing dystrophin protein via suppression of dystrophin-targeting miRNAs. Here, we test this in the
Identifiants
pubmed: 37534133
doi: 10.1016/j.isci.2023.107161
pii: S2589-0042(23)01238-5
pmc: PMC10391915
doi:
Types de publication
Journal Article
Langues
eng
Pagination
107161Subventions
Organisme : NIAMS NIH HHS
ID : L40 AR070539
Pays : United States
Organisme : NICHD NIH HHS
ID : U54 HD090257
Pays : United States
Organisme : NHLBI NIH HHS
ID : R00 HL130035
Pays : United States
Organisme : NIAMS NIH HHS
ID : L40 AR068727
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL153054
Pays : United States
Organisme : NICHD NIH HHS
ID : P50 HD090254
Pays : United States
Organisme : NHLBI NIH HHS
ID : K99 HL130035
Pays : United States
Informations de copyright
© 2023 The Author(s).
Déclaration de conflit d'intérêts
A.A.F. and an immediate family member of C.R.H. have a patent related to this work. C.R.H. and A.A.F. have filed a provisional intellectual property application related to research in the manuscript.
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