ArticleHuman gene therapy2014
Central nervous system delivery of helper-dependent canine adenovirus corrects neuropathology and behavior in mucopolysaccharidosis type VII mice.
Article in Human gene therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
15 citing papers in PubMed, 30 citations in OpenAlex.
- Role of homologous recombination/recombineering on human adenovirus genome engineering: Not the only but the most competent solution.Engineering microbiology · 2024Review
- Article
- Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.Biomolecules · 2021Review
- High-Capacity Adenoviral Vectors: Expanding the Scope of Gene Therapy.International journal of molecular sciences · 2020Review
- TrkB-expressing paraventricular hypothalamic neurons suppress appetite through multiple neurocircuits.Nature communications · 2020Article
- Impact of Social Isolation on the Behavioral, Functional Profiles, and Hippocampal Atrophy Asymmetry in Dementia in Times of Coronavirus Pandemic (COVID-19): A Translational Neuroscience Approach.Frontiers in psychiatry · 2020Article
- CAV-2 Vector Development and Gene Transfer in the Central and Peripheral Nervous Systems.Frontiers in molecular neuroscience · 2019Review
- Lysosomal and network alterations in human mucopolysaccharidosis type VII iPSC-derived neurons.Scientific reports · 2018Article
- Crystal structure of the fibre head domain of bovine adenovirus 4, a ruminant atadenovirus.Virology journal · 2015Article
- Canine helper-dependent vectors production: implications of Cre activity and co-infection on adenovirus propagation.Scientific reports · 2015Article
- Impact of adenovirus life cycle progression on the generation of canine helper-dependent vectors.Gene therapy · 2015Article
- Recent advances in gene therapy for lysosomal storage disorders.The application of clinical genetics · 2015Review
- Corrective GUSB transfer to the canine mucopolysaccharidosis VII cornea using a helper-dependent canine adenovirus vector.Journal of controlled release : official journal of the Controlled Release Society · 2014Article
- Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Methods for gene transfer to the central nervous system.Advances in genetics · 2014Review
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Canine adenovirus type 2 vectors (CAV-2) are promising tools to treat global central nervous system (CNS) disorders because of their preferential transduction of neurons and efficient retrograde axonal transport. Here we tested the potential of a helper-dependent CAV-2 vector expressing β-glucuronidase (HD-RIGIE) in a mouse model of mucopolysaccharidosis type VII (MPS VII), a lysosomal storage disease caused by deficiency in β-glucuronidase activity. MPS VII leads to glycosaminoglycan accumulation into enlarged vesicles in peripheral tissues and the CNS, resulting in peripheral and neuronal dysfunction. After intracranial administration of HD-RIGIE, we show long-term expression of β-glucuronidase that led to correction of neuropathology around the injection site and in distal areas. This phenotypic correction correlated with a decrease in secondary-elevated lysosomal enzyme activity and glycosaminoglycan levels, consistent with global biochemical correction. Moreover, HD-RIGIE-treated mice show significant cognitive improvement. Thus, injections of HD-CAV-2 vectors in the brain allow a global and sustained expression and may have implications for brain therapy in patients with lysosomal storage disease.
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Registered trials
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