Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial
出版年份 2022 全文链接
标题
Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial
作者
关键词
-
出版物
NATURE MEDICINE
Volume 28, Issue 1, Pages 63-70
出版商
Springer Science and Business Media LLC
发表日期
2022-01-04
DOI
10.1038/s41591-021-01554-9
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies
- (2021) Claire M. Drysdale et al. Cell Stem Cell
- Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither
- (2021) Richard J. Jones et al. BLOOD
- Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent β-Thalassemia
- (2020) Jennifer Schneiderman et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Retroviral Insertional Mutagenesis in Humans: Evidence for Four Genetic Mechanisms Promoting Expansion of Cell Clones
- (2020) Frederic D. Bushman MOLECULAR THERAPY
- Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease
- (2020) Matthew M. Hsieh et al. Blood Advances
- Gene therapy using haematopoietic stem and progenitor cells
- (2020) Giuliana Ferrari et al. NATURE REVIEWS GENETICS
- HIV proviral DNA integration can drive T cell growth ex vivo
- (2020) John K. Yoon et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- The E3 ligase UBR2 regulates cell death under caspase deficiency via Erk/MAPK pathway
- (2020) Elodie Villa et al. Cell Death & Disease
- Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia
- (2019) Sarah Marktel et al. NATURE MEDICINE
- Myeloid neoplasms in the setting of sickle cell disease: an intrinsic association with the underlying condition rather than a coincidence; report of 4 cases and review of the literature
- (2019) Yang Li et al. MODERN PATHOLOGY
- Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies
- (2019) Elisa Magrin et al. BLOOD
- "Risk Score for Development of Veno-Occlusive Disease After Allogeneic Hematopoietic Cell Transplant"
- (2018) Christopher Strouse et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Thalassaemia
- (2018) Ali T Taher et al. LANCET
- Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
- (2018) Alexis A. Thompson et al. NEW ENGLAND JOURNAL OF MEDICINE
- INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes
- (2017) Eric Sherman et al. Molecular Therapy-Methods & Clinical Development
- INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions
- (2017) Charles C. Berry et al. Molecular Therapy-Methods & Clinical Development
- Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects
- (2016) Jorge Mansilla-Soto et al. HUMAN GENE THERAPY
- Risk of individual malignant neoplasms in patients with sickle cell disease: English national record linkage study
- (2016) Olena O Seminog et al. JOURNAL OF THE ROYAL SOCIETY OF MEDICINE
- Association of busulfan exposure with survival and toxicity after haemopoietic cell transplantation in children and young adults: a multicentre, retrospective cohort analysis
- (2016) Imke H Bartelink et al. Lancet Haematology
- Comparing DNA integration site clusters with scan statistics
- (2014) Charles C. Berry et al. BIOINFORMATICS
- Safe mobilization of CD34+ cells in adults with -thalassemia and validation of effective globin gene transfer for clinical investigation
- (2014) F. Boulad et al. BLOOD
- IthaGenes: An Interactive Database for Haemoglobin Variations and Epidemiology
- (2014) Petros Kountouris et al. PLoS One
- Isolation and functional characterization of human erythroblasts at distinct stages: implications for understanding of normal and disordered erythropoiesis in vivo
- (2013) J. Hu et al. BLOOD
- Long-term health-related quality of life evaluated more than 20 years after hematopoietic stem cell transplantation for thalassemia
- (2013) G. La Nasa et al. BLOOD
- Hematopoietic Stem Cell Transplantation in Thalassemia and Sickle Cell Anemia
- (2013) G. Lucarelli et al. Cold Spring Harbor Perspectives in Medicine
- Estimating abundances of retroviral insertion sites from DNA fragment length data
- (2012) Charles C. Berry et al. BIOINFORMATICS
- HMGA2 regulates the in vitro aging and proliferation of human umbilical cord blood-derived stromal cells through the mTOR/p70S6K signaling pathway
- (2012) Kyung-Rok Yu et al. Stem Cell Research
- A Simple and Effective Method to Generate Lentiviral Vectors for Ex Vivo Gene Delivery to Mature Human Peripheral Blood Lymphocytes
- (2012) Shicheng Yang et al. Human Gene Therapy Methods
- 3'UTR-truncated Hmga2 cDNA causes MPN-like hematopoiesis by conferring a clonal growth advantage at the level of HSC in mice
- (2011) K. Ikeda et al. BLOOD
- Deregulated expression of HMGA2 is implicated in clonal expansion of PIGA deficient cells in paroxysmal nocturnal haemoglobinuria
- (2011) Yoshiko Murakami et al. BRITISH JOURNAL OF HAEMATOLOGY
- The challenge of haemoglobinopathies in resource-poor countries
- (2011) David J. Weatherall BRITISH JOURNAL OF HAEMATOLOGY
- Survival in a large cohort of Greek patients with transfusion-dependent beta thalassaemia and mortality ratios compared to the general population
- (2011) Vassilis Ladis et al. EUROPEAN JOURNAL OF HAEMATOLOGY
- Therapy for β-globinopathies: a brief review and determinants for successful and safe correction
- (2010) Ajay Perumbeti et al. Annals of the New York Academy of Sciences
- Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction
- (2010) S Charrier et al. GENE THERAPY
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
- (2010) Marina Cavazzana-Calvo et al. NATURE
- Platelet transfusion refractoriness in highly immunized beta thalassemia children undergoing stem cell transplantation
- (2010) Sarah Marktel et al. PEDIATRIC TRANSPLANTATION
- Successful correction of murine sickle cell disease with reduced stem cell requirements reinforced by fractionated marrow infusions
- (2009) Hady Felfly et al. BRITISH JOURNAL OF HAEMATOLOGY
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