Gene therapy for primary immunodeficiencies

276. Immunological and Metabolic Correction After Lentiviral Vector Mediated Haematopoietic Stem Cell Gene Therapy for ADA Deficiency

(2016)   MOLECULAR THERAPY

Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs

(2014) A. Girard-Gagnepain et al.   BLOOD

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

(2014) C. X. Wang et al.   BLOOD

HLA-haploidentical stem cell transplantation after removal of   + T and B cells in children with nonmalignant disorders

(2014) A. Bertaina et al.   BLOOD

Reprogramming Committed Murine Blood Cells to Induced Hematopoietic Stem Cells with Defined Factors

(2014) Jonah Riddell et al.   CELL

Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

(2014) Burcu Bestas et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2014) Maria Chiriaco et al.   MOLECULAR THERAPY

Repair and replace

(2014) Alain Fischer   NATURE

Reprogramming finds its niche

(2014) Daniel Lucas et al.   NATURE

Cell competition is a tumour suppressor mechanism in the thymus

(2014) Vera C. Martins et al.   NATURE

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

Reprogramming human endothelial cells to haematopoietic cells requires vascular induction

(2014) Vladislav M. Sandler et al.   NATURE

Wnt signaling controls the specification of definitive and primitive hematopoiesis from human pluripotent stem cells

(2014) Christopher M Sturgeon et al.   NATURE BIOTECHNOLOGY

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

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal

(2014) I. Fares et al.   SCIENCE

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

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

How I treat severe combined immunodeficiency

(2013) H. B. Gaspar et al.   BLOOD

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

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

Long-Term Follow-up of Foamy Viral Vector-Mediated Gene Therapy for Canine Leukocyte Adhesion Deficiency

(2013) Thomas R Bauer 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

CD4+ T Cells from IPEX Patients Convert into Functional and Stable Regulatory T Cells by FOXP3 Gene Transfer

(2013) L. Passerini et al.   Science Translational Medicine

Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

(2012) A. Astrakhan 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

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

Leukocyte Adhesion Deficiencies

(2012) Edith van de Vijver et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

(2012) Samantha Scaramuzza et al.   MOLECULAR THERAPY

Transduction of Human CD34+ Repopulating Cells with a Self-Inactivating Lentiviral Vector for SCID-X1 Produced at Clinical Scale by a Stable Cell Line

(2012) Michael R. Greene et al.   Human Gene Therapy Methods

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

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

Prostaglandin E2 Enhances Human Cord Blood Stem Cell Xenotransplants and Shows Long-Term Safety in Preclinical Nonhuman Primate Transplant Models

(2011) Wolfram Goessling et al.   Cell Stem Cell

Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter

(2011) C Brendel et al.   GENE 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

B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

(2010) H. M. Kerns et al.   BLOOD

X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease

(2010) C. Booth et al.   BLOOD

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

Assessing the Risk of T-cell Malignancies in Mouse Models of SCID-X1

(2010) Brian Sorrentino   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 safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

20 years of gene therapy for SCID

(2010) Alain Fischer et al.   NATURE IMMUNOLOGY

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

Aryl Hydrocarbon Receptor Antagonists Promote the Expansion of Human Hematopoietic Stem Cells

(2010) Anthony E. Boitano et al.   SCIENCE

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

(2009) Brian D. Brown et al.   NATURE REVIEWS GENETICS

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

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

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

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

(2009) N. Cartier et al.   SCIENCE

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

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

(2008) Susannah I Thornhill et al.   MOLECULAR THERAPY

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

(2008) Gary P. Wang et al.   NUCLEIC ACIDS RESEARCH