A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

(2021) Richard J. Jones et al.   BLOOD

Changing the Natural History of Fanconi Anemia Complementation Group-À with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

(2020) Agnieszka Czechowicz et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

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

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

(2020) Suk See De Ravin et al.   BLOOD

Retroviral Insertional Mutagenesis in Humans: Evidence for Four Genetic Mechanisms Promoting Expansion of Cell Clones

(2020) Frederic D. Bushman   MOLECULAR THERAPY

Lentiviral gene therapy for X-linked chronic granulomatous disease

(2020) Donald B. Kohn et al.   NATURE MEDICINE

Opportunities and challenges associated with the evaluation of chimeric antigen receptor T cells in real-life

(2020) Eoin McGrath et al.   CURRENT OPINION IN ONCOLOGY

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

Survival and toxicity outcomes of hematopoietic stem cell transplantation for pediatric patients with Fanconi anemia: a unified multicentric national study from the Spanish Working Group for Bone Marrow Transplantation in Children

(2020) L. Murillo-Sanjuán et al.   BONE MARROW TRANSPLANTATION

Gene therapy using haematopoietic stem and progenitor cells

(2020) Giuliana Ferrari et al.   NATURE REVIEWS GENETICS

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

(2020) Francesca Tucci et al.   MOLECULAR THERAPY

Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice

(2019) Hye-Sook Kwon 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

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 Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

(2019) Ewelina Mamcarz et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Editorial: The Relationship Between Cancer Predisposition and Primary Immunodeficiency

(2019) Fabian Hauck et al.   Frontiers in Immunology

Occurrence of acute myeloid leukemia in hydroxyurea-treated sickle cell disease patient

(2019) Samuel Regan et al.   CANCER BIOLOGY & THERAPY

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

(2019) Paula Río et al.   NATURE MEDICINE

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

(2019) Roxane Labrosse et al.   BLOOD

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

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

(2019) Ewelina Mamcarz et al.   BLOOD

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

(2019) Erica B. Esrick et al.   BLOOD

Treosulfan and Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Children with Primary Immunodeficiency: UK Experience

(2018) Mary A. Slatter et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Hematopoietic Stem Cell Transplantation in Thalassemia

(2018) Luisa Strocchio et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Cancer in primary immunodeficiency diseases: Cancer incidence in the United States Immune Deficiency Network Registry

(2018) Paul C. Mayor et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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

Allogeneic stem cell transplantation in adolescents and young adults with primary immunodeficiencies

(2018) Michael H. Albert et al.   Journal of Allergy and Clinical Immunology-In Practice

Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation

(2018) Gerald V. Raymond et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency

(2017) Kit L. Shaw et al.   JOURNAL OF CLINICAL INVESTIGATION

Gene Therapy in a Patient with Sickle Cell Disease

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

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

(2017) Florian Eichler et al.   NEW ENGLAND JOURNAL OF MEDICINE

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

(2016) Eliane Gluckman et al.   BLOOD

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

(2016) Maria Pia Cicalese et al.   BLOOD

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

(2016) Maria Sessa et al.   LANCET

Safer conditioning for blood stem cell transplants

(2016) Alessandro Aiuti et al.   NATURE BIOTECHNOLOGY

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

(2016) Rahul Palchaudhuri et al.   NATURE BIOTECHNOLOGY

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

(2016) Suk See De Ravin et al.   Science Translational Medicine

Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines

(2015) Mieke Aldenhoven et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing

(2015) Ulrich Siler et al.   CURRENT GENE THERAPY

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

(2015) Salima Hacein-Bey Abina et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Outcomes in Two Japanese Adenosine Deaminase-Deficiency Patients Treated by Stem Cell Gene Therapy with No Cytoreductive Conditioning

(2015) Makoto Otsu et al.   JOURNAL OF CLINICAL IMMUNOLOGY

Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report

(2015) Alexander A. Boucher et al.   Orphanet Journal of Rare Diseases

Transplantation Outcomes for Severe Combined Immunodeficiency, 2000–2009

(2014) Sung-Yun Pai et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

(2014) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

(2014) C. J. Braun et al.   Science Translational Medicine

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency

(2012) A. Hassan et al.   BLOOD

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

(2012) F. Candotti et al.   BLOOD

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency

(2011) R. Sokolic et al.   BLOOD

Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II Trial

(2011) Hyoung Jin Kang et al.   MOLECULAR THERAPY

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

Random effects meta-analysis of event outcome in the framework of the generalized linear mixed model with applications in sparse data

(2010) Theo Stijnen et al.   STATISTICS IN MEDICINE

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

(2009) E. M. Kang et al.   BLOOD

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

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION