Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

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

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

(2019) Marina Cavazzana et al.   NATURE REVIEWS DRUG DISCOVERY

Toxicity-Free Hematopoietic Stem Cell Engraftment Achieved with Anti-CD117 Monoclonal Antibody Conditioning

(2019) Rajni Agarwal et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

(2019) Francesca Ferrua et al.   Lancet Haematology

Antibody Conditioning Enables MHC-Mismatched Hematopoietic Stem Cell Transplants and Organ Graft Tolerance

(2019) Benson M. George et al.   Cell Stem Cell

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

(2019) Mark C. Walters et al.   BLOOD

Prostaglandin E 2 Increases Lentiviral Vector Transduction Efficiency of Adult Human Hematopoietic Stem and Progenitor Cells

(2018) Garrett C. Heffner et al.   MOLECULAR THERAPY

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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

Multiple integrated non-clinical studies predict safety of lentiviral mediated gene therapy for beta thalassemia

(2018) Maria Rosa Lidonnici et al.   Molecular Therapy-Methods & Clinical Development

Accelerated leukemic transformation after haplo-identical transplantation for hydroxyurea-treated sickle cell disease

(2017) Murali Janakiram et al.   LEUKEMIA & LYMPHOMA

Gene Therapy in a Patient with Sickle Cell Disease

(2017) Jean-Antoine Ribeil et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene

(2016) Olivier Negre 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

Devouring the Hematopoietic Stem Cell: Setting the Table for Marrow Cell Transplantation

(2016) Morton J Cowan et al.   MOLECULAR THERAPY

Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin

(2016) Rahul Palchaudhuri et al.   NATURE BIOTECHNOLOGY

Hematopoietic stem cell transplantation in immunocompetent hosts without radiation or chemotherapy

(2016) Akanksha Chhabra et al.   Science Translational Medicine

Treating Immunodeficiency through HSC Gene Therapy

(2016) Claire Booth et al.   TRENDS IN MOLECULAR MEDICINE

Myelodysplastic syndrome with clonal cytogenetic abnormalities followed by fatal erythroid leukemia after 14 years of exposure to hydroxyurea for sickle cell anemia

(2015) Cédric Aumont et al.   AMERICAN JOURNAL OF HEMATOLOGY

Systematic Review: Hydroxyurea for the Treatment of Adults with Sickle Cell Disease

(2013) Sophie Lanzkron et al.   ANNALS OF INTERNAL MEDICINE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Correction of murine  -thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

(2011) O. Negre et al.   BLOOD

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

(2011) A. Biffi et al.   BLOOD