Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease.
Batten disease
CLN3
bipolar cells
gene therapy
retina
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
07 2020
07 2020
Historique:
pubmed:
25
6
2020
medline:
24
8
2021
entrez:
25
6
2020
Statut:
ppublish
Résumé
The neuronal ceroid lipofuscinoses (NCLs), often referred to as Batten disease, are inherited lysosomal storage disorders that represent the most common neurodegeneration during childhood. Symptoms include seizures, vision loss, motor and cognitive decline, and premature death. The development of brain-directed treatments for NCLs has made noteworthy progress in recent years. Clinical trials are currently ongoing or planned for different forms of the disease. Despite these promising advances, it is unlikely that therapeutic interventions targeting the brain will prevent loss of vision in patients as retinal cells remain untreated and will continue to degenerate. Here, we demonstrate that
Identifiants
pubmed: 32578444
doi: 10.1089/hum.2020.038
pmc: PMC7404834
doi:
Substances chimiques
CLN3 protein, mouse
0
Membrane Glycoproteins
0
Molecular Chaperones
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
709-718Subventions
Organisme : Medical Research Council
ID : MR/N026101/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/S036784/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/R025134/1
Pays : United Kingdom
Organisme : Department of Health
Pays : United Kingdom
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