ArticleGene therapy2007
Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.
Article in Gene therapy, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
26 citing papers in PubMed, 55 citations in OpenAlex.
- Gene and Cellular Therapies for Leukodystrophies.Pharmaceutics · 2023Review
- Treatment of Fabry Disease: Established and Emerging Therapies.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.Molecular therapy. Methods & clinical development · 2022Review
- Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders.Frontiers in genome editing · 2022Review
- Review
- The Cardiovascular Phenotype in Fabry Disease: New Findings in the Research Field.International journal of molecular sciences · 2021Review
- Essential thrombocytosis attributed to JAK2-T875N germline mutation.International journal of hematology · 2019Article
- Inhibition of Mitochondrial Complex I Impairs Release of α-Galactosidase by Jurkat Cells.International journal of molecular sciences · 2019Article
- Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.Genetics and molecular biology · 2019Article
- Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34Molecular therapy. Methods & clinical development · 2017Article
- Glycosphingolipid storage in Fabry mice extends beyond globotriaosylceramide and is affected by ABCB1 depletion.Future science OA · 2016Article
- Prevention of Lethal Murine Hypophosphatasia by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow Cells.Human gene therapy · 2015Article
- Article
- Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.Human gene therapy · 2014Review
- The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases.Journal of lipid research · 2014Review
- Engineered human Tmpk fused with truncated cell-surface markers: versatile cell-fate control safety cassettes.Gene therapy · 2013Article
- MicroRNA-24 regulates cardiac fibrosis after myocardial infarction.Journal of cellular and molecular medicine · 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
- Treatment of adult MPSI mouse brains with IDUA-expressing mesenchymal stem cells decreases GAG deposition and improves exploratory behavior.Genetic vaccines and therapy · 2012Article
- Application of an endothelialized modular construct for islet transplantation in syngeneic and allogeneic immunosuppressed rat models.Tissue engineering. Part A · 2011Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 4 institutions in 2 countries.
Funding
Abstract
A deficiency in alpha-galactosidase A (alpha-gal A) activity causes Fabry disease. Virus-based delivery of genes can correct cells and establish a sustained supply of therapeutic proteins. Recombinant lentiviral vectors (LVs) show promise in this context. We first demonstrate LV-mediated marking of peripheral blood (PB) cells by transduction/transplantation of hematopoietic stem/progenitor cells. Stable enGFP expression was observed in PB for 37 weeks. Next, we transplanted Fabry mice with bone marrow mononuclear cells (BMMNCs) transduced a single time with a LV encoding the human alpha-gal A cDNA. Sustained expression of functional alpha-gal A in Fabry mice was observed over 24 weeks. Plasma alpha-gal A activity from treated Fabry mice was two-fold higher than wild-type controls. Increased alpha-gal A activity, often to supra-normal levels, and reduction of globotriaosylceramide, a glycolipid that accumulates in Fabry disease, was observed in all organs assessed. In secondary bone marrow transplantations, Fabry mice showed multilineage marking of PB, splenocytes and BMMNCs, along with therapeutic levels of alpha-gal A activity in plasma and organs over 20 weeks. Lastly, we transduced mobilized PB CD34(+) cells from a Fabry patient and observed corresponding enzymatic increases. Thus a single LV-mediated transduction of primitive hematopoietic cells can result in sustained correction for Fabry disease.
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Registered trials
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.