Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia

A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy

(2021) Sherif M. Badawy et al.   Blood Advances

β-Thalassemias

(2021) Ali T. Taher et al.   NEW ENGLAND JOURNAL OF MEDICINE

A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease

(2021) Alexis Leonard et al.   MOLECULAR THERAPY

Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen

(2020) Usanarat Anurathapan et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

The changing epidemiology of the ageing thalassaemia populations: A position statement of the Thalassaemia International Federation

(2020) Dimitrios Farmakis et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia

(2020) M. Domenica Cappellini et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

(2019) Sarah Marktel et al.   NATURE MEDICINE

Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation

(2019) Séverine Coquerelle et al.   HUMAN GENE THERAPY

Gene therapy of hemoglobinopathies: progress and future challenges

(2019) Yasuhiro Ikawa et al.   HUMAN MOLECULAR GENETICS

Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study

(2019) Mary Eapen et al.   Lancet Haematology

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

(2019) Angela R. Smith et al.   BLOOD

Benefit Corporation: a path to affordable gene therapies?

(2019) Alain Fischer et al.   NATURE MEDICINE

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene therapy: evidence, value and affordability in the US health care system

(2018) Grace Hampson et al.   Journal of Comparative Effectiveness Research

Health-related quality of life (HRQoL) in beta-thalassemia major (β-TM) patients assessed by 36-item short form health survey (SF-36): a meta-analysis

(2018) Mahdieh Arian et al.   QUALITY OF LIFE RESEARCH

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

(2017) Robert Hettle et al.   HEALTH TECHNOLOGY ASSESSMENT

Gene Therapy Approaches to Hemoglobinopathies

(2017) Giuliana Ferrari et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000–2010

(2016) D Baronciani et al.   BONE MARROW TRANSPLANTATION

Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects

(2016) Jorge Mansilla-Soto et al.   HUMAN GENE THERAPY

Healthcare costs and outcomes of managing β-thalassemia major over 50 years in the United Kingdom

(2016) Diana Weidlich et al.   TRANSFUSION

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

(2014) E. Angelucci et al.   HAEMATOLOGICA

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

Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling

(2013) F. Locatelli et al.   BLOOD

Hematopoietic Stem Cell Mobilization for Gene Therapy: Superior Mobilization by the Combination of Granulocyte–Colony Stimulating Factor Plus Plerixafor in Patients with β-Thalassemia Major

(2013) Evangelia Yannaki et al.   HUMAN GENE THERAPY

Umbilical Cord Blood Transplantation for Children with Thalassemia and Sickle Cell Disease

(2011) Annalisa Ruggeri et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

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

Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia

(2011) Keshet Ronen et al.   MOLECULAR THERAPY

Quality of life in thalassemia: A comparison of SF-36 results from the thalassemia longitudinal cohort to reported literature and the US norms

(2010) A. Sobota et al.   AMERICAN JOURNAL OF HEMATOLOGY

Prospective Assessment of Health-Related Quality of Life in Pediatric Patients with Beta-Thalassemia following Hematopoietic Stem Cell Transplantation

(2010) Giovanni Caocci et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

What is the minimum level of donor chimerism necessary to sustain transfusion independence in thalassaemia?

(2010) A Alfred et al.   BONE MARROW TRANSPLANTATION

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region

(2009) Paritha I Arumugam et al.   MOLECULAR THERAPY

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

(2008) Daniela Zychlinski et al.   MOLECULAR THERAPY

Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific RepressorBCL11A

(2008) Vijay G. Sankaran et al.   SCIENCE