Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin.

No abstract is available for this paper.

• Publication year

  2000

• Venue

  Nature

• Publication date

  Unknown publication date

• Fields of study

  Medicine

• Identifiers
  PMID 10894546
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Looping, linking, and chromatin activity: new insights into beta-globin locus regulation.

  2000cited by this paper
• The cHS4 Insulator Increases the Probability of Retroviral Expression at Random Chromosomal Integration Sites

  2000cited by this paper
• Looping versus linking: toward a model for long-distance gene activation.

  1999cited by this paper
• Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors.

  1999cited by this paper
• Lentiviral Vectors--the Promise of Gene Therapy Within Reach?

  1999cited by this paper
• Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing.

  1998cited by this paper
• A Third-Generation Lentivirus Vector with a Conditional Packaging System

  1998cited by this paper
• The dynamics of globin gene expression and gene therapy vectors.

  1998cited by this paper
• Bone Marrow Transplantation for Homozygous β‐Thalassemia: The Memorial Sloan‐Kettering Cancer Center Experience

  1998cited by this paper
• GENETIC TREATMENT OF THE HAEMOGLOINOPATHIES: RECOMBINATIONS AND NEW COMBINATIONS

  1997cited by this paper
• Recombinant retroviruses pseudotyped with the vesicular stomatitis virus G glycoprotein mediate both stable gene transfer and pseudotransduction in human peripheral blood lymphocytes.

  1997cited by this paper
• Long-term transfer and expression of the human beta-globin gene in a mouse transplant model.

  1997cited by this paper
• The human beta globin locus introduced by YAC transfer exhibits a specific and reproducible pattern of developmental regulation in transgenic mice.

  1997cited by this paper
• Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

  1997cited by this paper
• A mouse model for beta 0-thalassemia.

  1995cited by this paper
• Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene.

  1995cited by this paper
• A second generation transgenic mouse model expressing both hemoglobin S (HbS) and HbS-Antilles results in increased phenotypic severity.

  1995cited by this paper
• Synergistic regulation of human beta-globin gene switching by locus control region elements HS3 and HS4.

  1995cited by this paper
• Mutagenesis of retroviral vectors transducing human beta‐globin gene and beta‐globin locus control region derivatives results in stable transmission of an active transcriptional structure.

  1994cited by this paper
• High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells.

  1990cited by this paper
• The HIV-1 rev trans-activator acts through a structured target sequence to activate nuclear export of unspliced viral mRNA

  1989cited by this paper
• A dominant control region from the human β-globin locus conferring integration site-independent gene expression

  1989cited by this paper
• Position-independent, high-level expression of the human beta-globin gene in transgenic mice.

  1987cited by this paper
• Evidence for a locus activation region: the formation of developmentally stable hypersensitive sites in globin-expressing hybrids.

  1987cited by this paper
• The "beta-like-globin" gene domain in human erythroid cells.

  1985cited by this paper
• Synthesis of haemoglobins specified by allelic genes in human heterozygotes.

  1971cited by this paper

• G2B: an optimized lentiviral vector with enhanced titer and β-globin expression for improved β-thalassemia gene therapy

  2025cites this paper
• The β-Hemoglobinopathies as a Model for the Development of Nonviral, Episomal Vectors for Gene Therapy

  2025cites this paper
• Ex Vivo Gene and Cell Therapy in Hematopoietic Stem Cells

  2025cites this paper
• Ex vivo Modification of Hematopoietic Stem and Progenitor Cells for Gene Therapy.

  2025cites this paper
• Epigenetic Regulation of Erythropoiesis: From Developmental Programs to Therapeutic Targets

  2025cites this paper
• Screening and treatment of thalassemia.

  2025cites this paper
• A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease

  2024cites this paper
• Current and emerging drug treatment strategies to tackle sickle cell anemia

  2024cites this paper
• A new frontier: FDA approvals for gene therapy in sickle cell disease.

  2024cites this paper
• Toward life without sickle cell disease.

  2024cites this paper
• Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials

  2023cites this paper
• Cellular and animal models for the investigation of β-thalassemia.

  2023cites this paper
• Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies

  2023cites this paper
• Thalassemia (Beta-Thalassemia)

  2023cites this paper
• Gene Therapy for Hemoglobinopathies

  2023cites this paper
• Nanotechnology-enabled gene delivery for cancer and other genetic diseases

  2023cites this paper
• Reducing the transcriptional read‐through rate of a lentiviral vector for β‐thalassemia gene therapy

  2023cites this paper
• Gene therapy for sickle cell disease.

  2023cites this paper
• A Journey from Blood Cells to Genes and Back.

  2023cites this paper
• Lentiviral Vectors for Ocular Gene Therapy

  2022cites this paper
• Gene therapy for sickle cell disease.

  2022cites this paper
• Development and clinical translation of ex vivo gene therapy

  2022influential citation
• Diverse Approaches to Gene Therapy of Sickle Cell Disease.

  2022cites this paper
• Globin vector regulatory elements are active in early hematopoietic progenitor cells

  2022cites this paper
• Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial

  2022cites this paper
• Evolution of Gene Therapy, Historical Perspective.

  2022cites this paper
• Carrier strategies boost the application of CRISPR/Cas system in gene therapy

  2022cites this paper
• Emergent treatments for β-thalassemia and orphan drug legislations.

  2022cites this paper
• Of mice and men: From hematopoiesis in mouse models to curative gene therapy for sickle cell disease

  2022cites this paper
• The Future of Gene Therapy for Transfusion-Dependent Beta-Thalassemia: The Power of the Lentiviral Vector for Genetically Modified Hematopoietic Stem Cells

  2021cites this paper
• Thalassemias: From gene to therapy.

  2021cites this paper
• Novel therapies in β‐thalassaemia

  2021cites this paper
• Genome-based therapeutic interventions for β-type hemoglobinopathies

  2021cites this paper
• Success Stories and Challenges Ahead in Hematopoietic Stem Cell Gene Therapy: Hemoglobinopathies as Disease Models

  2021influential citation
• Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies.

  2021cites this paper
• Genome Editing for β-Hemoglobinopathies: Advances and Challenges

  2021cites this paper
• Advancing the care of β-thalassaemia patients with novel therapies.

  2021cites this paper
• A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

  2021cites this paper
• Improving outcomes and quality of life for patients with transfusion-dependent β-thalassemia: recommendations for best clinical practice and the use of novel treatment strategies

  2021cites this paper
• Treating sickle cell anemia

  2020cites this paper
• Emerging therapies in β-thalassemia: toward a new era in management

  2020cites this paper
• Restoration of β-Globin Expression with Optimally Designed Lentiviral Vector for β-Thalassemia Treatment in Chinese Patients

  2020cites this paper
• Gene therapy for sickle cell disease.

  2020cites this paper
• Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences

  2020cites this paper
• Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements.

  2020cites this paper
• Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing

  2020cites this paper
• Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small -Globin Locus Control Region Elements

  2020cites this paper
• Innovative Therapies for Hemoglobin Disorders

  2020influential citation
• Beta thalassemia gene therapy using lentiviral vectors

  2020cites this paper
• Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies.

  2019cites this paper
• Modelling human haemoglobin switching.

  2019cites this paper
• Gene Therapy For Beta-Thalassemia: Updated Perspectives

  2019cites this paper
• Gene Therapy For Beta-Thalassemia: Updated Perspectives

  2019cites this paper
• Mouse Models in Hematopoietic Stem Cell Gene Therapy and Genome Editing.

  2019cites this paper
• In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

  2019cites this paper
• Emerging Genetic Therapy for Sickle Cell Disease.

  2019cites this paper
• Gene therapy of hemoglobinopathies: progress and future challenges.

  2019cites this paper
• Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

  2019cites this paper
• Development and characterization of cellular biosensors for HTS of erythroid differentiation inducers targeting the transcriptional activity of γ-globin and β-globin gene promoters

  2019cites this paper
• Advances in the gene therapy of monogenic blood cell diseases

  2019cites this paper
• Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

  2019cites this paper
• CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantation

  2019cites this paper
• Oral ferroportin inhibitor VIT‐2763: First‐in‐human, phase 1 study in healthy volunteers

  2019cites this paper
• TACG_A_178546 167..180

  2019cites this paper
• High-level b-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells

  2018cites this paper
• Recent Progress on Genetic Diagnosis and Therapy for β-Thalassemia in China and Around the World

  2018cites this paper
• An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

  2018cites this paper
• Engineering Globin Gene Expression

  2018cites this paper
• Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced

  2018cites this paper
• Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease

  2018cites this paper
• Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.

  2018cites this paper
• Gene therapy and gene editing strategies for hemoglobinopathies.

  2018cites this paper
• Promise of gene therapy to treat sickle cell disease

  2018cites this paper
• Gene Therapy in Patients with Transfusion‐Dependent β‐Thalassemia

  2018cites this paper
• The Optimization of Self-deleting Lentiviral Vector Carrying Human β-globin Gene and Promoter

  2018cites this paper
• High-level embryonic globin production with efficient erythroid differentiation from a K562 erythroleukemia cell line.

  2018cites this paper
• Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies

  2018cites this paper
• Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice.

  2018cites this paper
• Gene therapy for sickle cell disease.

  2018cites this paper
• Erythropoiesis: insights into pathophysiology and treatments in 2017

  2018cites this paper
• Gene Therapy for Hemoglobinopathies

  2018cites this paper
• Advanced personalized gene therapy of B-thalassaemia

  2018cites this paper
• Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy.

  2018cites this paper
• Gene therapy comes of age

  2018cites this paper
• Pharmacological and molecular approaches for the treatment of β‐hemoglobin disorders

  2018cites this paper
• Gene replacement therapy for hemoglobinopathies: clinical benefit & challenges for widespread utilization

  2018cites this paper
• Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

  2018cites this paper
• Gene Therapy and Genome Editing.

  2018cites this paper
• Gene therapy for sickle cell disease: An update.

  2018cites this paper
• A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?

  2018cites this paper
• Sickle cells produce functional immune modulators and cytotoxics

  2017cites this paper
• Scalable Lentiviral Vector Production Using Stable HEK293SF Producer Cell Lines

  2017cites this paper
• Therapeutic strategies in Sickle Cell Anemia: The past present and future

  2017cites this paper
• Recent advances in the management of Thalassaemia: A Review Update

  2017cites this paper
• Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients

  2017cites this paper
• Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy.

  2017cites this paper
• Re-educating immunity in respiratory allergies: the potential for hematopoietic stem cell-mediated gene therapy

  2017cites this paper
• Gene and Cell Therapy for β-Thalassemia and Sickle Cell Disease with Induced Pluripotent Stem Cells (iPSCs): The Next Frontier.

  2017cites this paper
• In situ programming of leukaemia-specific T cells using synthetic DNA nanocarriers.

  2017cites this paper
• Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.

  2017cites this paper