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

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

(2020) Julie Kanter et al.   BLOOD

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

Pathologic angiogenesis in the bone marrow of humanized sickle cell mice is reversible by blood transfusion

(2020) Shin-Young Park et al.   BLOOD

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

(2020) Emmanuelle Six et al.   BLOOD

Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients

(2020) Francesca Fumagalli et al.   MOLECULAR GENETICS AND METABOLISM

Lentiviral gene therapy for X-linked chronic granulomatous disease

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

Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche

(2020) Annamaria Aprile et al.   BLOOD

Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions

(2020) Francesca Ferrua et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

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

(2019) Sarah Marktel et al.   NATURE MEDICINE

Bone marrow stromal cells from β-thalassemia patients have impaired hematopoietic supportive capacity

(2019) Stefania Crippa et al.   JOURNAL OF CLINICAL INVESTIGATION

Lentiviral gene therapy corrects platelet phenotype and function in Wiskott-Aldrich patients

(2019) Lucia Sereni et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Microglia damage precedes major myelin breakdown in X-linked adrenoleukodystrophy and metachromatic leukodystrophy

(2019) Caroline G. Bergner et al.   GLIA

Two decades of excellent transplant survival in children with chronic granulomatous disease

(2019) Su Han Lum et al.   BLOOD

The role of WASp in T cells and B cells

(2019) Xizi Sun et al.   CELLULAR IMMUNOLOGY

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

Gene correction for SCID-X1 in long-term hematopoietic stem cells

(2019) Mara Pavel-Dinu et al.   Nature Communications

Title: Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements

(2019) Richard A. Morgan et al.   MOLECULAR THERAPY

Hematopoietic Stem Cell Transplantation in Primary Immunodeficiency Diseases: Current Status and Future Perspectives

(2019) Riccardo Castagnoli et al.   Frontiers in Pediatrics

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 Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

(2019) Donald B. Kohn et al.   BLOOD

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

(2019) Alexis A. Thompson et al.   BLOOD

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

(2019) Elisa Magrin et al.   BLOOD

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

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

Hematopoietic Stem Cell Transplantation for Primary Immunodeficiencies

(2019) Andrew R. Gennery et al.   Frontiers in Pediatrics

Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

(2019) Emily Y. Tan et al.   Frontiers in Pediatrics

The influence of environment and origin on brain resident macrophages and implications for therapy

(2019) Mariko L. Bennett et al.   NATURE NEUROSCIENCE

Hematopoietic cell transplantation in primary immunodeficiency – conventional and emerging indications

(2018) Mary A. Slatter et al.   Expert Review of Clinical Immunology

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

Adenosine deaminase deficiency: a review

(2018) Aisling M. Flinn et al.   Orphanet Journal of Rare Diseases

Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

(2018) Heide Stirnadel-Farrant et al.   Orphanet Journal of Rare Diseases

Hematopoietic stem cell transplantation in its 60s: A platform for cellular therapies

(2018) Christian Chabannon et al.   Science Translational Medicine

First Occurrence of Plasmablastic Lymphoma in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Disease Patient and Review of the Literature

(2018) Maddalena Migliavacca et al.   Frontiers in Immunology

Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease

(2018) Christian Brendel et al.   Human Gene Therapy Clinical Development

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency

(2018) Donald B. Kohn et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans

(2018) Serena Scala et al.   NATURE MEDICINE

Pathophysiology of Sickle Cell Disease

(2018) Prithu Sundd et al.   Annual Review of Pathology-Mechanisms of Disease

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

Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug

(2017) Francesca Ferrua et al.   HUMAN GENE THERAPY

Clinical Manifestations and Pathophysiological Mechanisms of the Wiskott-Aldrich Syndrome

(2017) Fabio Candotti   JOURNAL OF CLINICAL IMMUNOLOGY

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

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

(2017) Giulia Schiroli et al.   Science Translational Medicine

Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells

(2017) Alessia Capotondo et al.   Science Advances

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

(2016) Maria Pia Cicalese et al.   BLOOD

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

Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy

(2016) Vasco Meneghini et al.   EMBO Molecular Medicine

Lentiviral Vector Gene Therapy Protects XCGD Mice From Acute Staphylococcus aureus Pneumonia and Inflammatory Response

(2016) Giada Farinelli et al.   MOLECULAR THERAPY

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

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

Adenosine Deaminase Deficiency – More Than Just an Immunodeficiency

(2016) Kathryn V. Whitmore et al.   Frontiers in Immunology

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

Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment

(2015) Elsa G. Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

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

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

Dual-regulated Lentiviral Vector for Gene Therapy of X-linked Chronic Granulomatosis

(2014) Maria Chiriaco et al.   MOLECULAR THERAPY

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

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

Global migration and the changing distribution of sickle haemoglobin: a quantitative study of temporal trends between 1960 and 2000

(2014) Frédéric B Piel et al.   Lancet Global Health

Fetal hemoglobin in sickle cell anemia: a glass half full?

(2013) M. H. Steinberg et al.   BLOOD

Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease

(2013) Ana Sergijenko et al.   MOLECULAR THERAPY

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

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

(2011) Hyoung Jin Kang et al.   MOLECULAR THERAPY

Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction

(2011) H. B. Gaspar et al.   Science Translational Medicine

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model

(2010) I. Visigalli et al.   BLOOD

Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients

(2010) Emanuela Anna Roselli et al.   EMBO Molecular Medicine

Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease

(2010) M. Andreani et al.   HAEMATOLOGICA

Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells

(2010) Giorgia Santilli et al.   MOLECULAR THERAPY

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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

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

Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options

(2008) Volkmar Gieselmann   ACTA PAEDIATRICA

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION

Lysosomal Storage Diseases and the Blood-Brain Barrier

(2008) David Begley et al.   CURRENT PHARMACEUTICAL DESIGN

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA