Gene addition for beta thalassemia

In vivo hematopoietic stem cell modification by mRNA delivery

(2023) Laura Breda et al.   SCIENCE

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

(2022) Elisa Magrin et al.   NATURE MEDICINE

Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial

(2022) Farid Boulad et al.   NATURE MEDICINE

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

(2022) Isabelle Thuret et al.   Stem Cells Translational Medicine

Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB ‐206 study

(2022) Julie Kanter et al.   AMERICAN JOURNAL OF HEMATOLOGY

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

(2022) Mark C. Walters et al.   BLOOD

Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

(2021) Laura Breda et al.   MOLECULAR THERAPY

Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-Thalassemia

(2021) Franco Locatelli et al.   NEW ENGLAND JOURNAL OF MEDICINE

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

(2020) Ashutosh Lal et al.   BLOOD

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

(2019) Sarah Marktel et al.   NATURE MEDICINE

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

(2019) Agnieszka Czechowicz et al.   Nature Communications

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

(2019) Janet L. Kwiatkowski et al.   BLOOD

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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

Comparable Outcomes of Allogeneic Peripheral Blood versus Bone Marrow Hematopoietic Stem Cell Transplantation in Major Thalassemia—A Multivariate Long-term Cohort Analysis

(2018) Ardeshir Ghavamzadeh et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Dynamics of fertility impairment and recovery after allogeneic haematopoietic stem cell transplantation in childhood and adolescence: results from a longitudinal study

(2014) C. Pfitzer et al.   JOURNAL OF CANCER RESEARCH AND CLINICAL ONCOLOGY

Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

(2011) Chuanfeng Wu et al.   Frontiers of Medicine

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

(2010) Marina Cavazzana-Calvo et al.   NATURE