Targeting the 5' untranslated region of
5′ UTR
SMN2
antisense oligonucleotides
spinal muscular atrophy
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
05 Mar 2021
05 Mar 2021
Historique:
received:
22
07
2020
accepted:
30
12
2020
entrez:
12
2
2021
pubmed:
13
2
2021
medline:
13
2
2021
Statut:
epublish
Résumé
Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations in the survival motor neuron 1 (
Identifiants
pubmed: 33575118
doi: 10.1016/j.omtn.2020.12.027
pii: S2162-2531(20)30408-X
pmc: PMC7851419
doi:
Types de publication
Journal Article
Langues
eng
Pagination
731-742Subventions
Organisme : Wellcome Trust
Pays : United Kingdom
Organisme : Wellcome Trust
ID : 205162/Z/16/Z
Pays : United Kingdom
Déclaration de conflit d'intérêts
A patent application was filed for the 5′ UTR ASOs described in this manuscript. M.J.W. is a founder of and shareholder in PepGen.
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