Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy.
Animals
Dependovirus
/ genetics
Disease Models, Animal
Gene Expression
Gene Order
Genetic Therapy
/ methods
Genetic Vectors
/ administration & dosage
Homeostasis
Liver
/ metabolism
Mice
Mice, Knockout
Mitochondria, Liver
/ genetics
Mitochondrial Encephalomyopathies
/ genetics
Promoter Regions, Genetic
Thymidine Phosphorylase
/ genetics
Transduction, Genetic
alpha 1-Antitrypsin
/ genetics
None
AAV
MNGIE
mitochondria
promoter
thymidine
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
08 2019
08 2019
Historique:
pubmed:
23
3
2019
medline:
13
3
2020
entrez:
23
3
2019
Statut:
ppublish
Résumé
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a devastating disease caused by mutations in
Identifiants
pubmed: 30900470
doi: 10.1089/hum.2018.217
pmc: PMC7647930
doi:
Substances chimiques
SERPINA1 protein, human
0
alpha 1-Antitrypsin
0
Thymidine Phosphorylase
EC 2.4.2.4
Tymp protein, mouse
EC 2.4.2.4
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
985-998Références
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