Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice.

Animals Enzyme Replacement Therapy Gangliosidosis, GM1 / metabolism Mice beta-Galactosidase / metabolism

GM1 gangliosidosis beta-galactosidase biophysics cation-independent mannose-6-phosphate receptor endoplasmic reticulum stress enzyme replacement therapy (ERT) gene therapy lysosomal storage disease lysosome neurodegeneration safety toxicity unfolded protein response (UPR)

Journal

The Journal of biological chemistry

ISSN: 1083-351X

Titre abrégé: J Biol Chem

Pays: United States

ID NLM: 2985121R

Informations de publication

Date de publication:
25 09 2020

Historique:

received: 15 06 2019

revised: 10 08 2019

pubmed: 5 9 2019

medline: 7 2 2021

entrez: 5 9 2019

Statut: ppublish

Résumé

Autosomal recessive mutations in the galactosidase β1 (

Identifiants

DOI: 10.1074/jbc.RA119.009811 PMID: 31481471 PMC: PMC7521651

pubmed: 31481471

pii: S0021-9258(17)50012-5

doi: 10.1074/jbc.RA119.009811

pmc: PMC7521651

pii:

doi:

Substances chimiques

beta-Galactosidase EC 3.2.1.23

Banques de données

PDB

['3THC']

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

Pagination

13532-13555

Informations de copyright

Déclaration de conflit d'intérêts

The authors declare that they have no conflicts of interest with the contents of this article.

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