ArticleProceedings of the National Academy of Sciences of the United States of America2002
Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers.
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46 citing papers in PubMed, 162 citations in OpenAlex.
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- Cre-recombinase systems for induction of neuron-specific knockout models: a guide for biomedical researchers.Neural regeneration research · 2023Review
- Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction.Human gene therapy · 2023Article
- Genetics behind Cerebral Disease with Ocular Comorbidity: Finding Parallels between the Brain and Eye Molecular Pathology.International journal of molecular sciences · 2022Review
- Oligonucleotide Microarrays Identified Potential Regulatory Genes Related to Early Outward Arterial Remodeling Induced by Tissue Plasminogen Activator.Frontiers in physiology · 2019Article
- Viral vectors for therapy of neurologic diseases.Neuropharmacology · 2017Review
- Low-Affinity Neurotrophin Receptor p75 Promotes the Transduction of Targeted Lentiviral Vectors to Cholinergic Neurons of Rat Basal Forebrain.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2016Article
- Integrated analysis of proteome and transcriptome changes in the mucopolysaccharidosis type VII mouse hippocampus.Molecular genetics and metabolism · 2016Article
- Bilateral single-site intracerebral injection of a nonpathogenic herpes simplex virus-1 vector decreases anxiogenic behavior in MPS VII mice.Molecular therapy. Methods & clinical development · 2015Article
- Reversibility of neuropathology in Tay-Sachs-related diseases.Human molecular genetics · 2014Article
- Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease.Human gene therapy · 2011Article
- Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses.Current pharmaceutical biotechnology · 2011Review
- Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.Nature reviews. Genetics · 2011Review
- Feline immunodeficiency virus as a gene transfer vector in the rat nucleus tractus solitarii.Cellular and molecular neurobiology · 2010Article
- Gateway to the diseased brain.Nature medicine · 2009Article
- Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases.Experimental neurology · 2009Article
- Large animal models of neurological disorders for gene therapy.ILAR journal · 2009Review
- Mitochondrial imaging in dorsal root ganglion neurons following the application of inducible adenoviral vector expressing two fluorescent proteins.Journal of neuroscience methods · 2008Article
- Human gene therapy vectors derived from feline lentiviruses.Veterinary immunology and immunopathology · 2008Review
- CNS-directed gene therapy for lysosomal storage diseases.Acta paediatrica (Oslo, Norway : 1992) · 2008Review
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10 authors at 2 institutions in 2 countries.
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Abstract
Gene transfer vectors based on lentiviruses can transduce terminally differentiated cells in the brain; however, their ability to reverse established behavioral deficits in animal models of neurodegeneration has not previously been tested. When recombinant feline immunodeficiency virus (FIV)-based vectors expressing beta-glucuronidase were unilaterally injected into the striatum of adult beta-glucuronidase deficient [mucopolysaccharidosis type VII (MPS VII)] mice, an animal model of lysosomal storage disease, there was bihemispheric correction of the characteristic cellular pathology. Moreover, after the injection of FIV-based vectors expressing beta-glucuronidase into brains of beta-glucuronidase-deficient mice with established impairments in spatial learning and memory, there was dramatic recovery of behavioral function. Cognitive improvement resulting from expression of beta-glucuronidase was associated with alteration in expression of genes associated with neuronal plasticity. These data suggest that enzyme replacement to the MPS VII central nervous system goes beyond restoration of beta-glucuronidase activity in the lysosome, and imparts improvements in plasticity and spatial learning.
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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.