Designed U7 snRNAs inhibit
DUX4
FSHD
RNA silencing
U7
gene therapy
snRNA
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:
11
08
2020
accepted:
06
12
2020
entrez:
29
1
2021
pubmed:
30
1
2021
medline:
30
1
2021
Statut:
epublish
Résumé
Facioscapulohumeral muscular dystrophy (FSHD) arises from epigenetic changes that de-repress the
Identifiants
pubmed: 33510937
doi: 10.1016/j.omtn.2020.12.004
pii: S2162-2531(20)30385-1
pmc: PMC7807095
doi:
Types de publication
Journal Article
Langues
eng
Pagination
476-486Subventions
Organisme : NIAMS NIH HHS
ID : P50 AR070604
Pays : United States
Organisme : NICHD NIH HHS
ID : P50 HD060848
Pays : United States
Informations de copyright
© 2020 The Author(s).
Déclaration de conflit d'intérêts
The sequences and methods described here were included in a provisional patent application filed on December 1, 2020 (USPTO serial no. 63/120,190). S.Q.H., A.R., and N.W. are listed as inventors.
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