Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study.
AAV
GM2 gangliosidosis
Sandhoff
Tay-Sachs
dosage
gene therapy
hexosaminidase A
viral vector
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
14 Mar 2024
14 Mar 2024
Historique:
received:
10
04
2023
accepted:
30
11
2023
medline:
11
1
2024
pubmed:
11
1
2024
entrez:
11
1
2024
Statut:
epublish
Résumé
The pathological accumulation of GM2 ganglioside associated with Tay-Sachs disease (TSD) and Sandhoff disease (SD) occurs in individuals who possess mutant forms of the heterodimer β-hexosaminidase A (Hex A) because of mutation of the
Identifiants
pubmed: 38205442
doi: 10.1016/j.omtm.2023.101168
pii: S2329-0501(23)00207-3
pmc: PMC10777117
doi:
Banques de données
ClinicalTrials.gov
['NCT04798235']
Types de publication
Journal Article
Langues
eng
Pagination
101168Informations de copyright
© 2023 The Authors.
Déclaration de conflit d'intérêts
The authors declare no competing interests.