Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin.
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Animal models of β-hemoglobinopathies: utility and limitationsAdvances in sickle cell therapies in the hydroxyurea eraStatins protect against fulminant pneumococcal infection and cytolysin toxicity in a mouse model of sickle cell diseaseTransduction of human primitive repopulating hematopoietic cells with lentiviral vectors pseudotyped with various envelope proteins.Hydroxyurea therapy of a murine model of sickle cell anemia inhibits the progression of pneumococcal disease by down-modulating E-selectinβ-globin gene transfer to human bone marrow for sickle cell disease.Integration-specific In Vitro Evaluation of Lentivirally Transduced Rhesus CD34(+) Cells Correlates With In Vivo Vector Copy Number.Hydroxyurea therapy requires HbF induction for clinical benefit in a sickle cell mouse model.Iron overload cardiomyopathy: better understanding of an increasing disorder.Gene therapy for hemoglobinopathies: the state of the field and the future.Erythroid promoter confines FGF2 expression to the marrow after hematopoietic stem cell gene therapy and leads to enhanced endosteal bone formation.Chicken HS4 insulators have minimal barrier function among progeny of human hematopoietic cells transduced with an HIV1-based lentiviral vector.Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer.The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells.The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells.Correction of murine hemoglobinopathies by prenatal tolerance induction and postnatal nonmyeloablative allogeneic BM transplants.Cotransduction with MGMT and Ubiquitous or Erythroid-Specific GFP Lentiviruses Allows Enrichment of Dual-Positive Hematopoietic Progenitor Cells In Vivo.High-efficiency transduction of rhesus hematopoietic repopulating cells by a modified HIV1-based lentiviral vector.Amelioration of murine sickle cell disease by nonablative conditioning and γ-globin gene-corrected bone marrow cells.Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Analysis of alpha hemoglobin stabilizing protein overexpression in murine β-thalassemiaGene therapy for hemoglobinopathies: progress and challenges.Amelioration of murine beta-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both gamma-globin and the MGMT drug-resistance gene.Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseasesA novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correctionLineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.Vascular Permeability Drives Susceptibility to Influenza Infection in a Murine Model of Sickle Cell Disease.Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells.Genetic strategies for the treatment of sickle cell anaemia.Development of gene therapy for thalassemia.Recent trends in the gene therapy of β-thalassemia.Gene therapy for monogenic disorders of the bone marrow.Clinically-oriented proteomic investigation of sickle cell disease: Opportunities and challenges.Use of genome-editing tools to treat sickle cell disease.Bidirectional immune tolerance in nonmyeloablative MHC-mismatched BMT for murine β-thalassemia.Emerging cellular and gene therapies for congenital anemias.Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.Safe and Efficient Gene Therapy for Pyruvate Kinase DeficiencyGene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats.Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.
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P2860
Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
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scientific article published on 02 December 2008
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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 of murine sickle ce ...... xpression of fetal hemoglobin.
@en
Correction of murine sickle ce ...... xpression of fetal hemoglobin.
@nl
type
label
Correction of murine sickle ce ...... xpression of fetal hemoglobin.
@en
Correction of murine sickle ce ...... xpression of fetal hemoglobin.
@nl
prefLabel
Correction of murine sickle ce ...... xpression of fetal hemoglobin.
@en
Correction of murine sickle ce ...... xpression of fetal hemoglobin.
@nl
P2093
P2860
P356
P1433
P1476
Correction of murine sickle ce ...... xpression of fetal hemoglobin.
@en
P2093
Dennis Jay
Derek A Persons
John T Gray
Kelli M Boyd
Phillip W Hargrove
Tamara I Pestina
P2860
P304
P356
10.1038/MT.2008.259
P577
2008-12-02T00:00:00Z