Gene therapy for monogenic disorders of the bone marrow

Lysosomal Storage Diseases: From Pathophysiology to Therapy

(2015) Giancarlo Parenti et al.   Annual Review of Medicine

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1

(2015) F. Touzot et al.   BLOOD

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

B-cell reconstitution after lentiviral vector–mediated gene therapy in patients with Wiskott-Aldrich syndrome

(2015) Maria Carmina Castiello et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Perforin Gene Transfer Into Hematopoietic Stem Cells Improves Immune Dysregulation in Murine Models of Perforin Deficiency

(2015) Marlene Carmo et al.   MOLECULAR THERAPY

Safe mobilization of CD34+ cells in adults with  -thalassemia and validation of effective globin gene transfer for clinical investigation

(2014) F. Boulad et al.   BLOOD

Targeted gene therapy and cell reprogramming in Fanconi anemia

(2014) P. Rio et al.   EMBO Molecular Medicine

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac

(2014) Fei Xie et al.   GENOME RESEARCH

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

Gene Therapy for Hemoglobinopathies

(2014) Shanmuganathan Chandrakasan et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Recent advances in chronic granulomatous disease

(2014) David Goldblatt   JOURNAL OF INFECTION

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

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

Modern management of primary T-cell immunodeficiencies

(2014) Jana Pachlopnik Schmid et al.   PEDIATRIC ALLERGY AND IMMUNOLOGY

An Overview of Hemoglobinopathies and the Interpretation of Newborn Screening Results

(2014) Shanmuganathan Chandrakasan et al.   PEDIATRIC ANNALS

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

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

Management of sickle cell disease in the community

(2014) V. Brousse et al.   BMJ-British Medical Journal

Wiskott-Aldrich syndrome: a comprehensive review

(2013) Michel J. Massaad et al.   Annals of the New York Academy of Sciences

Seamless correction of the sickle cell disease mutation of theHBBgene in human induced pluripotent stem cells using TALENs

(2013) Ning Sun et al.   BIOTECHNOLOGY AND BIOENGINEERING

Management of sickle cell disease from childhood through adulthood

(2013) Julie Kanter et al.   BLOOD REVIEWS

The Wiskott-Aldrich Syndrome: The Actin Cytoskeleton and Immune Cell Function

(2013) Michael P. Blundell et al.   DISEASE MARKERS

Clinical, functional, and genetic characterization of chronic granulomatous disease in 89 Turkish patients

(2013) Mustafa Yavuz Köker et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Clinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation

(2013) Theresa Cole et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Recombination-activating gene 1 (Rag1)–deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

(2013) Niek P. van Til et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

β-globin gene transfer to human bone marrow for sickle cell disease

(2013) Zulema Romero et al.   JOURNAL OF CLINICAL INVESTIGATION

Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study

(2013) Tayfun Güngör et al.   LANCET

Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency

(2013) Denise A Carbonaro 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

Gene therapy for hemoglobinopathies: progress and challenges

(2013) Alisa Dong et al.   Translational Research

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

Consequences and management of iron overload in sickle cell disease

(2013) J. Porter et al.   Hematology-American Society of Hematology Education Program

Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

(2012) D. A. Carbonaro et al.   BLOOD

SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

(2012) C. Rivat 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

Gene therapy for primary immunodeficiencies: Part 2

(2012) Alessandro Aiuti et al.   CURRENT OPINION IN IMMUNOLOGY

Chronic Granulomatous Disease

(2012) Steven M. Holland   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Gene Therapy for Fanconi Anemia: One Step Closer to the Clinic

(2012) Jakub Tolar et al.   HUMAN GENE THERAPY

Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene

(2012) Niek P van Til et al.   MOLECULAR THERAPY

Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice

(2012) Alexander Langford-Smith et al.   MOLECULAR THERAPY

Current clinical management of Fanconi anemia

(2012) Angela R Smith et al.   Expert Review of Hematology

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

(2011) D. Moratto et al.   BLOOD

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome

(2011) Marita Bosticardo et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Chronic granulomatous disease: Overview and hematopoietic stem cell transplantation

(2011) Elizabeth M. Kang et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Restoration of anti-Aspergillus defense by neutrophil extracellular traps in human chronic granulomatous disease after gene therapy is calprotectin-dependent

(2011) Matteo Bianchi et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

(2011) K Pike-Overzet et al.   LEUKEMIA

Development of Novel Efficient SIN Vectors with Improved Safety Features for Wiskott–Aldrich Syndrome Stem Cell Based Gene Therapy

(2011) Inés Avedillo Díez et al.   MOLECULAR PHARMACEUTICS

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

(2011) Hyoung Jin Kang et al.   MOLECULAR THERAPY

Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting

(2011) Jakub Tolar et al.   MOLECULAR THERAPY

Modern management of primary B-cell immunodeficiencies

(2011) Miriam Hoernes et al.   PEDIATRIC ALLERGY AND IMMUNOLOGY

Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency

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

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

Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in β-thalassemia

(2010) Michel Sadelain et al.   Annals of the New York Academy of Sciences

Therapeutic levels of fetal hemoglobin in erythroid progeny of  -thalassemic CD34+ cells after lentiviral vector-mediated gene transfer

(2010) A. Wilber et al.   BLOOD

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

(2010) N. P. van Til et al.   BLOOD

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

Advances in the diagnosis and treatment of chronic granulomatous disease

(2010) Reinhard A Seger   CURRENT OPINION IN HEMATOLOGY

Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?

(2010) Andrew R. Gennery et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma

(2010) Manuel Grez et al.   MOLECULAR THERAPY

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

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

Restoration of NET formation by gene therapy in CGD controls aspergillosis

(2009) M. Bianchi et al.   BLOOD

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

A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

(2009) A. Perumbeti et al.   BLOOD

How I treat ADA deficiency

(2009) H. B. Gaspar et al.   BLOOD

Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models

(2009) Francesco Marangoni et al.   MOLECULAR THERAPY

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

New insights into the biology of Wiskott-Aldrich syndrome (WAS)

(2009) A. J. Thrasher   Hematology-American Society of Hematology Education Program

Clinical and immunologic consequences of a somatic reversion in a patient with X-linked severe combined immunodeficiency

(2008) C. Speckmann et al.   BLOOD

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION

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

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

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

Stable and Functional Lymphoid Reconstitution in Artemis-deficient Mice Following Lentiviral Artemis Gene Transfer Into Hematopoietic Stem Cells

(2008) Fatine Benjelloun et al.   MOLECULAR THERAPY

Restoration of Human B-cell Differentiation Into NOD-SCID Mice Engrafted With Gene-corrected CD34+ Cells Isolated From Artemis or RAG1-deficient Patients

(2008) Chantal Lagresle-Peyrou et al.   MOLECULAR THERAPY

Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency

(2008) Susannah I Thornhill et al.   MOLECULAR THERAPY

Correction of Murine Sickle Cell Disease Using γ-Globin Lentiviral Vectors to Mediate High-level Expression of Fetal Hemoglobin

(2008) Tamara I Pestina et al.   MOLECULAR THERAPY