HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe

Increased incidence of autoimmune cytopenias after allogeneic haematopoietic stem cell transplantation using a matched unrelated donor in children with β‐thalassaemia

(2021) Christina Oikonomopoulou et al.   BRITISH JOURNAL OF HAEMATOLOGY

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

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

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

(2020) Rajni Agarwal et al.   BLOOD

Matched Family versus Alternative Donor Hematopoietic Stem Cell Transplantation for Patients with Thalassemia Major: Experience from a Tertiary Referral Center in South India

(2020) Venkateswaran Vellaichamy Swaminathan et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Current and future gene therapies for hemoglobinopathies

(2020) Christian Brendel et al.   CURRENT OPINION IN HEMATOLOGY

Gene therapies for transfusion dependent β‐Thalassemia : current status and critical criteria for success

(2020) Sandeep Soni   AMERICAN JOURNAL OF HEMATOLOGY

Innovative Therapies for Hemoglobin Disorders

(2020) Karine Sii-Felice et al.   BIODRUGS

Gene therapy using haematopoietic stem and progenitor cells

(2020) Giuliana Ferrari et al.   NATURE REVIEWS GENETICS

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

(2019) Sarah Marktel et al.   NATURE MEDICINE

Unrelated Donor Peripheral Blood Stem Cell Transplantation for Patients with β-Thalassemia Major Based on a Novel Conditioning Regimen

(2019) Lan Sun et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Effect of increased dose of total body irradiation on graft failure associated with HLA-haploidentical transplantation in patients with severe haemoglobinopathies: a prospective clinical trial

(2019) Javier Bolaños-Meade et al.   Lancet Haematology

HLA-haploidentical hematopoietic stem cell transplantation in pediatric patients with hemoglobinopathies: current practice and new approaches

(2019) Lena Oevermann et al.   BONE MARROW TRANSPLANTATION

Regulators' Advice Can Make a Difference: European Medicines Agency Approval of Zynteglo for Beta Thalassemia

(2019) Martina Schuessler‐Lenz et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Functional profiling of single CRISPR/Cas9-edited human long-term hematopoietic stem cells

(2019) Elvin Wagenblast et al.   Nature Communications

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

(2019) Frank J. T. Staal et al.   Frontiers in Pediatrics

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

(2018) Chiara Antoniani et al.   BLOOD

Haploidentical haematopoietic stem cell transplantation for thalassaemia major based on an FBCA conditioning regimen

(2018) Qixin Sun et al.   BRITISH JOURNAL OF HAEMATOLOGY

Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

(2018) Karine Sii-Felice et al.   EXPERIMENTAL HEMATOLOGY

Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience

(2018) Ilhem Rahal et al.   HAEMATOLOGICA

Complication free survival long-term after hemopoietic cell transplantation in thalassemia

(2018) Emanuele Angelucci   HAEMATOLOGICA

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

Unrelated and related donor transplantation for beta-thalassemia major: A single-center experience from India

(2018) M. Joseph John et al.   PEDIATRIC TRANSPLANTATION

Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience

(2018) Ilhem Rahal et al.   HAEMATOLOGICA

Complication free survival long-term after hemopoietic cell transplantation in thalassemia

(2018) Emanuele Angelucci   HAEMATOLOGICA

Gene Therapy for β-Hemoglobinopathies

(2017) Marina Cavazzana et al.   MOLECULAR THERAPY

Unrelated donor stem cell transplantation for transfusion-dependent thalassemia

(2016) Shalini Shenoy et al.   Annals of the New York Academy of Sciences

Current recommendations for chelation for transfusion-dependent thalassemia

(2016) Janet L. Kwiatkowski   Annals of the New York Academy of Sciences

Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors

(2016) U Anurathapan et al.   BONE MARROW TRANSPLANTATION

Transplantation for thalassemia major

(2016) Franco Locatelli et al.   CURRENT OPINION IN HEMATOLOGY

β-Thalassemia

(2016) Raffaella Origa   GENETICS IN MEDICINE

Optimal Outcomes in Young Class 3 Patients With Thalassemia Undergoing HLA-Identical Sibling Bone Marrow Transplantation

(2016) Javid Gaziev et al.   TRANSPLANTATION

Cord blood transplantation in children with hemoglobinopathies

(2015) Luisa Strocchio et al.   Expert Opinion on Orphan Drugs

Pretransplant Immunosuppression followed by Reduced-Toxicity Conditioning and Stem Cell Transplantation in High-Risk Thalassemia: A Safe Approach to Disease Control

(2013) Usanarat Anurathapan et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach

(2013) J. Gaziev 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

Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major

(2013) Ayad Ahmed Hussein et al.   PEDIATRIC BLOOD & CANCER

Improved Clinical Outcomes of High Risk β Thalassemia Major Patients Undergoing a HLA Matched Related Allogeneic Stem Cell Transplant with a Treosulfan Based Conditioning Regimen and Peripheral Blood Stem Cell Grafts

(2013) Vikram Mathews et al.   PLoS One

Treosulfan-Thiotepa-Fludarabine–Based Conditioning Regimen for Allogeneic Transplantation in Patients with Thalassemia Major: A Single-Center Experience from North India

(2012) Dharma Choudhary et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan

(2012) M. E. Bernardo et al.   BLOOD

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

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

Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients

(2011) T-H Jaing et al.   BONE MARROW TRANSPLANTATION

HLA-matched sibling stem cell transplantation in children with β-thalassemia with anti-thymocyte globulin as part of the preparative regimen: the Greek experience

(2011) E Goussetis et al.   BONE MARROW TRANSPLANTATION

Milestones in umbilical cord blood transplantation

(2011) Eliane Gluckman et al.   BRITISH JOURNAL OF HAEMATOLOGY

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Purified T-depleted, CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia

(2009) P. Sodani et al.   BLOOD

Successful unmanipulated peripheral blood progenitor cell transplantation from an HLA haploidentical 2-locus-mismatched mother in a thalassemic patient with primary graft failure after transplantation of bone marrow and cord blood from unrelated donors

(2007) Tang-Her Jaing et al.   PEDIATRIC TRANSPLANTATION