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.
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Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapyLong-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cellsalpha-Galactosidase A deficient mice: a model of Fabry diseaseLentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry DiseaseLong-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 traffickingPreselective gene therapy for Fabry disease.Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transferCombination 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 diseaseBioluminescent 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
P2860
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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.
description
article científic
@ca
article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
@tr
scientific article published on July 1996
@en
vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Correction in trans for Fabry ...... recombinant retroviral vector.
@en
Correction in trans for Fabry ...... recombinant retroviral vector.
@nl
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
P2093
P2860
P356
P1476
Correction in trans for Fabry ...... recombinant retroviral vector.
@en
P2093
Jacobson S
Simovitch R
P2860
P304
P356
10.1073/PNAS.93.15.7917
P407
P577
1996-07-01T00:00:00Z