ArticleThe Journal of neuroscience : the official journal of the Society for Neuroscience2003
Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.
Article in The Journal of neuroscience : the official journal of the Society for Neuroscience, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.
What it found
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The trial behind it
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Who cites it
32 citing papers in PubMed, 79 citations in OpenAlex.
- Light-evoked activity and BDNF regulate mitochondrial dynamics and mitochondrial localized translation in CNS axons.iScience · 2025Article
- Evaluation of neuroretina following i.v. or intra-CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia.CNS neuroscience & therapeutics · 2024Article
- Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome).Therapeutics and clinical risk management · 2022Review
- Routes of administration for adeno-associated viruses carrying gene therapies for brain diseases.Frontiers in molecular neuroscience · 2022Review
- Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.Molecular therapy. Methods & clinical development · 2021Article
- Article
- Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.The Journal of clinical investigation · 2020Article
- AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains.Molecular therapy. Methods & clinical development · 2020Article
- Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.Human gene therapy · 2017Article
- Gene therapy for the neurological manifestations in lysosomal storage disorders.Journal of lipid research · 2014Review
- Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is trafficked in a highly motile Rab7-positive compartment.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain.Gene therapy · 2014Article
- Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain.Gene therapy · 2013Article
- A rapid gene delivery-based mouse model for early-stage Alzheimer disease-type tauopathy.Journal of neuropathology and experimental neurology · 2013Article
- Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.Human gene therapy · 2012Article
- Article
- Adipocytes as a vehicle for ex vivo gene therapy: Novel replacement therapy for diabetes and other metabolic diseases.Journal of diabetes investigation · 2011Review
- Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses.Current pharmaceutical biotechnology · 2011Review
- Genetic therapy for the nervous system.Human molecular genetics · 2011Review
- Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Human molecular genetics · 2011Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 3 institutions in 1 country.
Funding
Abstract
The mucopolysaccharidoses (MPSs) are lysosomal storage diseases resulting from impaired catabolism of sulfated glycosaminoglycans. MPS VII mice lack lysosomal beta-glucuronidase (GUSB) activity, leading to the accumulation of partially degraded chondroitin, dermatan, and heparan sulfates in most tissues. Consequently, these mice develop most of the symptoms exhibited by human MPS VII patients, including progressive visual and cognitive deficits. To investigate the effects of reducing lysosomal storage in nervous tissues, we injected recombinant adeno-associated virus encoding GUSB directly into the vitreous humor of young adult mice. Interestingly, GUSB activity was subsequently detected in the brains of the recipients. At 8-12 weeks after treatment, increased GUSB activity and reduced lysosomal distension were found in regions of the thalamus and tectum that received inputs from the injected eye. Lysosomal storage was also reduced in adjacent nonvisual regions, including the hippocampus, as well as in the visual cortex. The findings suggest that both diffusion and trans-synaptic transfer contribute to the dissemination of enzyme activity within the CNS. Intravitreal injection may thus provide a means of delivering certain therapeutic gene products to specific areas within the CNS.
Indexed as
Identifiers
12716937PMC6742327W1918313756What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.