ArticleProceedings of the National Academy of Sciences of the United States of America1996
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.
Article in Proceedings of the National Academy of Sciences of the United States of America, 1996. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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Who cites it
31 citing papers in PubMed, 75 citations in OpenAlex.
- Lentivirus-mediated gene therapy for Fabry disease: 5-year End-of-Study results from the Canadian FACTs trial.Clinical and translational medicine · 2025Article
- Fabry disease: a rare disorder calling for personalized medicine.International urology and nephrology · 2024Review
- Treatment of Fabry Disease: Established and Emerging Therapies.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Cardiovascular Involvement in Fabry's Disease: New Advances in Diagnostic Strategies, Outcome Prediction and Management.Cardiac failure review · 2023Review
- Autologous, lentivirus-modified, T-rapa cell "micropharmacies" for lysosomal storage disorders.EMBO molecular medicine · 2022Article
- Lentivirus-mediated gene therapy for Fabry disease.Nature communications · 2021Article
- Developments in the treatment of Fabry disease.Journal of inherited metabolic disease · 2020Review
- Inhibition of Mitochondrial Complex I Impairs Release of α-Galactosidase by Jurkat Cells.International journal of molecular sciences · 2019Article
- Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34Molecular therapy. Methods & clinical development · 2017Article
- Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy.World journal of stem cells · 2015Review
- Altered dynamics of a lipid raft associated protein in a kidney model of Fabry disease.Molecular genetics and metabolism · 2014Article
- Engineered human Tmpk fused with truncated cell-surface markers: versatile cell-fate control safety cassettes.Gene therapy · 2013Article
- Lysosomal delivery of therapeutic enzymes in cell models of Fabry disease.Journal of inherited metabolic disease · 2012Article
- Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Sequencing and characterization of the porcine α-galactosidase A gene: towards the generation of a porcine model for Fabry disease.Molecular biology reports · 2011Article
- Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Human molecular genetics · 2011Review
- α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine trafficking.Human gene therapy · 2011Article
- Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.Gene therapy · 2007Article
- Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors.Proceedings of the National Academy of Sciences of the United States of America · 2004Article
- Clinical Case Presentation - Fabry's Disease.EJIFCC · 2004Article
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Fabry disease is an X-linked metabolic disorder due to a deficiency of alpha-galactosidase A (alpha-gal A; EC 3.2.1.22). Patients accumulate glycosphingolipids with terminal alpha-galactosyl residues that come from intracellular synthesis, circulating metabolites, or from the biodegradation Of senescent cells. Patients eventually succumb to renal, cardio-, or cerebrovascular disease. No specific therapy exists. One possible approach to ameliorating this disorder is to target corrective gene transfer therapy to circulating hematopoietic cells. Toward this end, an amphotropic virus-producer cell line has been developed that produces a high titer (>10(6) i.p. per ml) recombinant retrovirus constructed to transduce and correct target cells. Virus-producer cells also demonstrate expression of large amounts of both intracellular and secreted alpha-gal A. To examine the utility of this therapeutic vector, skin fibroblasts from Fabry patients were corrected for the metabolic defect by infection with this recombinant virus and secreted enzyme was observed. Furthermore, the secreted enzyme was found to be taken up by uncorrected cells in a mannose-6-phosphate receptor-dependent manner. In related experiments, immortalized B cell lines from Fabry patients, created as a hematologic delivery test system, were transduced. As with the fibroblasts, transduced patient B cell lines demonstrated both endogenous enzyme correction and a small amount of secretion together with uptake by uncorrected cells. These studies demonstrate that endogenous metabolic correction in transduced cells, combined with secretion, may provide a continuous source of corrective material in trans to unmodified patient bystander cells (metabolic cooperativity).
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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.