Splicing defects and CRISPR-Cas9 correction in isogenic homozygous photoreceptor precursors harboring clustered deep-intronic ABCA4 variants.
ABCA4
CRISPR-Cas9
MT: RNA/DNA Editing
Stargardt
gene editing
iPSCs
inherited retinal disease
isogenic
photoreceptor precursors
splicing
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:
12 Mar 2024
12 Mar 2024
Historique:
received:
19
09
2023
accepted:
21
12
2023
medline:
26
1
2024
pubmed:
26
1
2024
entrez:
26
1
2024
Statut:
epublish
Résumé
Splicing defects from deep-intronic variants significantly contribute to the mutational spectrum in
Identifiants
pubmed: 38274366
doi: 10.1016/j.omtn.2023.102113
pii: S2162-2531(23)00331-1
pmc: PMC10809099
doi:
Types de publication
Journal Article
Langues
eng
Pagination
102113Informations de copyright
© 2023 The Author(s).
Déclaration de conflit d'intérêts
The authors declare no competing interests.