Gene therapy for mucopolysaccharidosis

No abstract is available for this paper.

• Publication year

  2007

• Venue

  Expert Opinion on Biological Therapy

• Publication date

  2007-08-29

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1517/14712598.7.9.1333PMID 17727324PMCID PMC3340574
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Genetic Vaccines and Therapy

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• AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma

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• Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

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• Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon‐mediated gene delivery: implications for non‐viral gene therapy of mucopolysaccharidoses

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• Clinical response to persistent, low-level β-glucuronidase expression in the murine model of mucopolysaccharidosis type VII

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• Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice.

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• and insertional genotoxicity Cell culture assays reveal the importance of retroviral vector design for

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• Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII.

  2006cited by this paper
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  2003cited by this paper
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  2003cited by this paper
• Intravitreal Gene Therapy Reduces Lysosomal Storage in Specific Areas of the CNS in Mucopolysaccharidosis VII Mice

  2003cited by this paper
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  2003cited by this paper
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  2002cited by this paper
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  2002cited by this paper
• Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

  2002cited by this paper
• Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

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• Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

  2001cited by this paper
• Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII.

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• Treatment of a Lysosomal Storage Disease, Mucopolysaccharidosis VII, with Microencapsulated Recombinant Cells

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• Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII.

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• Gene Transfer of Low Levels of β-Glucuronidase Corrects Hepatic Lysosomal Storage in a Large Animal Model of Mucopolysaccharidosis VII

  2000cited by this paper
• Reversal of Pathology in the Entire Brain of Mucopolysaccharidosis Type VII Mice after Lentivirus-Mediated Gene Transfer

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• Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice.

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• Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

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• Neonatal Intramuscular Injection with Recombinant Adeno-Associated Virus Results in Prolonged beta-Glucuronidase Expression in Situ and Correction of Liver Pathology in Mucopolysaccharidosis Type VII Mice

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• Functional gene delivery to and across brain vasculature of systemic AAVs with endothelial-specific tropism in rodents and broad tropism in primates

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