ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 25 citations in OpenAlex.
- Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome).Therapeutics and clinical risk management · 2022Review
- Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.Biomolecules · 2021Review
- Editorial: Tropism, Mapping, Modeling, or Therapy Using Canine Adenovirus Type 2 (CAV-2) Vectors in the CNS.Frontiers in molecular neuroscience · 2021Article
- CAV-2-Mediated GFP and LRRK2Frontiers in molecular neuroscience · 2020Article
- Location of the Cell Adhesion Molecule "Coxsackievirus and Adenovirus Receptor" in the Adult Mouse Brain.Frontiers in neuroanatomy · 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
- Canine and Feline Models of Human Genetic Diseases and Their Contributions to Advancing Clinical Therapies .The Yale journal of biology and medicine · 2017Review
- Adenovirus tales: from the cell surface to the nuclear pore complex.PLoS pathogens · 2015Review
- Canine helper-dependent vectors production: implications of Cre activity and co-infection on adenovirus propagation.Scientific reports · 2015Article
- Human coagulation factor X-adenovirus type 5 complexes poorly stimulate an innate immune response in human mononuclear phagocytes.Journal of virology · 2015Article
- Impact of adenovirus life cycle progression on the generation of canine helper-dependent vectors.Gene therapy · 2015Article
- 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
Corrections and comments
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Authors and funding
17 authors at 7 institutions in 2 countries.
Funding
Abstract
Severe deficiency in lysosomal β-glucuronidase (β-glu) enzymatic activity results in mucopolysaccharidosis (MPS) VII, an orphan disease with symptoms often appearing in early childhood. Symptoms are variable, but many patients have multiple organ disorders including neurological defects. At the cellular level, deficiency in β-glu activity leads to abnormal accumulation of glycosaminoglycans (GAGs), and secondary accumulation of GM2 and GM3 gangliosides, which have been linked to neuroinflammation. There have been encouraging gene transfer studies in the MPS VII mouse brain, but this is the first study attempting the correction of the >200-fold larger and challenging canine MPS VII brain. Here, the efficacy of a helper-dependent (HD) canine adenovirus (CAV-2) vector harboring a human GUSB expression cassette (HD-RIGIE) in the MPS VII dog brain was tested. Vector genomes, β-glu activity, GAG content, lysosome morphology and neuropathology were analyzed and quantified. Our data demonstrated that CAV-2 vectors preferentially transduced neurons and axonal retrograde transport from the injection site to efferent regions was efficient. HD-RIGIE injections, associated with mild and transient immunosuppression, corrected neuropathology in injected and noninjected structures throughout the cerebrum. These data support the clinical evaluation of HD CAV-2 vectors to treat the neurological defects associated with MPS VII and possibly other neuropathic lysosomal storage diseases.
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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.