Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector. - Wikidata - wikimedia - TriplyDB

Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector.

Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector.

http://www.wikidata.org/entity/Q37449924

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Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells alpha-Galactosidase A deficient mice: a model of Fabry disease Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer.α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine trafficking Preselective gene therapy for Fabry disease.Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice.Novel therapeutic targets for the treatment of Fabry disease.An approach to achieve long-term expression in skin gene therapy.Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice.Altered dynamics of a lipid raft associated protein in a kidney model of Fabry disease Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors.Gene therapy for fabry disease: a review of the literature.Lysosomal delivery of therapeutic enzymes in cell models of Fabry disease.Engineered human Tmpk fused with truncated cell-surface markers: versatile cell-fate control safety cassettes.Alpha-galactosidase A-Tat fusion enhances storage reduction in hearts and kidneys of Fabry mice.Specific hammerhead ribozymes reduce synthesis of cation-independent mannose 6-phosphate receptor mRNA and protein.Sequencing and characterization of the porcine α-galactosidase A gene: towards the generation of a porcine model for Fabry disease.Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.How realistic is cutaneous gene therapy?Early cerebral manifestations in a young female with Fabry disease with skewed X-inactivation.Clinical Case Presentation - Fabry's Disease

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P2860

Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector.

http://www.wikidata.org/entity/Q37449924

description

article científic

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article scientifique

@fr

articolo scientifico

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artigo científico

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bilimsel makale

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scientific article published on July 1996

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vedecký článok

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vetenskaplig artikel

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videnskabelig artikel

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vědecký článek

@cs

name

Correction in trans for Fabry ...... recombinant retroviral vector.

@en

Correction in trans for Fabry ...... recombinant retroviral vector.

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type

label

Correction in trans for Fabry ...... recombinant retroviral vector.

@en

Correction in trans for Fabry ...... recombinant retroviral vector.

@nl

prefLabel

Correction in trans for Fabry ...... recombinant retroviral vector.

@en

Correction in trans for Fabry ...... recombinant retroviral vector.

@nl

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PNAS.93.15.7917

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Proceedings of the National Academy of Sciences of the United States of America

P1476

Correction in trans for Fabry ...... recombinant retroviral vector.

@en

P2093

Brady RO

http://www.w3.org/2001/XMLSchema#string

Jacobson S

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Medin JA

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Murray GJ

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Quirk JM

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Simovitch R

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Tudor M

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P2860

Overexpression of human alpha-galactosidase A results in its intracellular aggregation, crystallization in lysosomes, and selective secretion

Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

Production of high-titer helper-free retroviruses by transient transfection

Enzyme therapy in Fabry disease: differential in vivo plasma clearance and metabolic effectiveness of plasma and splenic alpha-galactosidase A isozymes.

Treatment of genetic defects in hematopoietic cell function by gene transfer.

Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.

Purification and properties of the two major isozymes of alpha-galactosidase from human placenta.

Recent progress in gene therapy for inherited diseases.

A bicistronic therapeutic retroviral vector enables sorting of transduced CD34+ cells and corrects the enzyme deficiency in cells from Gaucher patients.

Construction and use of a safe and efficient amphotropic packaging cell line.

P304

7917-7922

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scholarly article

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10.1073/PNAS.93.15.7917

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P433

15

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93

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1996-07-01T00:00:00Z

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14446246

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8755577

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vector-borne disease

P932

38849

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