In vitro characterization of genetically modified embryonic stem cells as a therapy for murine mucopolysaccharidosis type IIIA. (Q40596298)

21 September 2017

In vitro characterization of genetically modified embryonic stem cells as a therapy for murine mucopolysaccharidosis type IIIA. (English)

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21 September 2017

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21 September 2017

Kim M Hemsley

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John Hopwood

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John J Hopwood

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21 September 2017

Adrian Meedeniya

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21 September 2017

1 February 2004

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Molecular Genetics and Metabolism

21 September 2017

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21 September 2017

81

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21 September 2017

86-95

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Prevalence of lysosomal storage disorders

21 September 2017

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The frequency of lysosomal storage diseases in The Netherlands.

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Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications

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Cloning of the sulphamidase gene and identification of mutations in Sanfilippo A syndrome

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Structure and sequence of the human sulphamidase gene

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Recombinant human sulphamidase: expression, amplification, purification and characterization

7 January 2021

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Expression and characterization of wild type and mutant recombinant human sulfamidase. Implications for Sanfilippo (Mucopolysaccharidosis IIIA) syndrome

7 January 2021

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Correction of Sanfilippo A skin fibroblasts by retroviral vector-mediated gene transfer

7 January 2021

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Cellular pathology of lysosomal storage disorders.

7 January 2021

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Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation

7 January 2021

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Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome

7 January 2021

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Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.

7 January 2021

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Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain

7 January 2021

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Primary adult human astrocytes as an ex vivo vehicle for beta-glucuronidase delivery in the brain

7 January 2021

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Long-term expression of beta-glucuronidase by genetically modified human neural progenitor cells grafted into the mouse central nervous system.

7 January 2021

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Multilineage differentiation from human embryonic stem cell lines

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.11.007

Embryonic stem cells express neuronal properties in vitro

7 January 2021

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Differentiation of embryonic stem cell to astrocytes visualized by green fluorescent protein

7 January 2021

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Differentiation of radial glia-like cells from embryonic stem cells.

7 January 2021

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Differentiation of nonbeating embryonic stem cells into beating cardiomyocytes is dependent on downregulation of PKC beta and zeta in concert with upregulation of PKC epsilon

7 January 2021

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Reconstituted skin from murine embryonic stem cells.

7 January 2021

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Evidence for hepatocyte differentiation from embryonic stem cells in vitro.

7 January 2021

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Nestin-specific green fluorescent protein expression in embryonic stem cell-derived neural precursor cells used for transplantation

7 January 2021

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Transplanted embryonic stem cells survive, differentiate and promote recovery in injured rat spinal cord

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.11.007

Lysosomal storage diseases

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.11.007

Diagnosis of Sanfilippo type A syndrome by estimation of sulfamidase activity using a radiolabelled tetrasaccharide substrate

7 January 2021

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Characterization and properties of α-d-mannosidase of human polymorphonuclear leucocytes

7 January 2021

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Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

7 January 2021

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Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

7 January 2021

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Enzyme replacement therapy in Fabry disease: a randomized controlled trial

7 January 2021

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Enzyme-replacement therapy in mucopolysaccharidosis I.

7 January 2021

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A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome).

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Mucopolysaccharidosis IIIA (Sanfilippo syndrome) in a New Zealand Huntaway dog with ataxia

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Sulfamidase deficiency in a family of Dachshunds: a canine model of mucopolysaccharidosis IIIA (Sanfilippo A).

7 January 2021

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Directed neuronal differentiation of human embryonic stem cells

7 January 2021

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Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

7 January 2021

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Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

7 January 2021

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Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/cathepsin A under control of the colony-stimulating factor-1 receptor promoter

7 January 2021

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Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors

7 January 2021

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Neural subtype specification of fertilization and nuclear transfer embryonic stem cells and application in parkinsonian mice.

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Stem cells and neuropoiesis in the adult human brain

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Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain.

7 January 2021

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

7 January 2021

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Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

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Gene therapy: shining hopes dented - but not dashed

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Looking into the safety of AAV vectors

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10.1016/J.YMGME.2003.11.007

7 January 2021

Identifiers

DOI

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21 September 2017

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PubMed ID

14741188