Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral 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:
12 Mar 2021
12 Mar 2021
Historique:
received:
10
08
2020
accepted:
28
11
2020
entrez:
29
1
2021
pubmed:
30
1
2021
medline:
30
1
2021
Statut:
epublish
Résumé
Gaucher disease type 1 (GD1) is an inherited lysosomal disorder with multisystemic effects in patients. Hallmark symptoms include hepatosplenomegaly, cytopenias, and bone disease with varying degrees of severity. Mutations in a single gene, glucosidase beta acid 1 (
Identifiants
pubmed: 33511245
doi: 10.1016/j.omtm.2020.11.018
pii: S2329-0501(20)30245-X
pmc: PMC7806948
doi:
Types de publication
Journal Article
Langues
eng
Pagination
312-323Informations de copyright
© 2020 The Authors.
Déclaration de conflit d'intérêts
This work was fully funded by AVROBIO (Cambridge, MA, USA). C.M., A.G., C.H., R.P., and D.P. are employees at AVROBIO. A.S., S.K., and M.D. are employed consultants for AVROBIO.
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