Current progress on gene therapy for primary immunodeficiencies

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

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

(2012) F. Candotti et al.   BLOOD

Body Weight-Dependent Pharmacokinetics of Busulfan in Paediatric Haematopoietic Stem Cell Transplantation Patients

(2012) Imke H. Bartelink et al.   CLINICAL PHARMACOKINETICS

Gene Therapy for Primary Immunodeficiencies

(2012) Christine Rivat 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

Alpharetroviral Self-inactivating Vectors: Long-term Transgene Expression in Murine Hematopoietic Cells and Low Genotoxicity

(2012) Julia D Suerth et al.   MOLECULAR THERAPY

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

In vivo genome editing using a high-efficiency TALEN system

(2012) Victoria M. Bedell et al.   NATURE

FLASH assembly of TALENs for high-throughput genome editing

(2012) Deepak Reyon et al.   NATURE BIOTECHNOLOGY

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

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

(2011) R. Sokolic et al.   BLOOD

Polyclonal fluctuation of lentiviral vector-transduced and expanded murine hematopoietic stem cells

(2011) T. Maetzig et al.   BLOOD

Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy

(2011) George Silva et al.   CURRENT GENE THERAPY

Characterization of the Human Artemis Promoter by Heterologous Gene Expression In Vitro and In Vivo

(2011) Megan M. Multhaup et al.   DNA AND CELL BIOLOGY

Treatment of canine leukocyte adhesion deficiency by foamy virus vectors expressing CD18 from a PGK promoter

(2011) T R Bauer et al.   GENE THERAPY

Optimal conditions for lentiviral transduction of engrafting human CD34+ cells

(2011) N Uchida et al.   GENE THERAPY

Gene Therapy for Canine Leukocyte Adhesion Deficiency with Lentiviral Vectors Using the Murine Stem Cell Virus and Human Phosphoglycerate Kinase Promoters

(2011) Michael J. Hunter et al.   HUMAN GENE THERAPY

Purine metabolism, immune reconstitution, and abdominal adipose tumor after gene therapy for adenosine deaminase deficiency

(2011) Eyal Grunebaum et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Gene therapy for primary immunodeficiencies: Looking ahead, toward gene correction

(2011) Itai M. Pessach 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

Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning

(2011) Marshall W Huston et al.   MOLECULAR THERAPY

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

Correction of SCID-X1 Using an Enhancerless Vav Promoter

(2010) E. Almarza et al.   HUMAN GENE THERAPY

Self-Inactivating Alpharetroviral Vectors with a Split-Packaging Design

(2010) J. D. Suerth et al.   JOURNAL OF VIROLOGY

A Ubiquitous Chromatin Opening Element (UCOE) Confers Resistance to DNA Methylation–mediated Silencing of Lentiviral Vectors

(2010) Fang Zhang et al.   MOLECULAR THERAPY

Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression

(2010) Samantha L Ginn et al.   MOLECULAR THERAPY

Gene Therapy of Canine Leukocyte Adhesion Deficiency Using Lentiviral Vectors With Human CD11b and CD18 Promoters Driving Canine CD18 Expression

(2010) Michael J Hunter et al.   MOLECULAR THERAPY

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

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

How I treat ADA deficiency

(2009) H. B. Gaspar et al.   BLOOD

Adenosine and the Auditory System

(2009) Srdjan Vlajkovic et al.   Current Neuropharmacology

Primary immunodeficiencies

(2009) Luigi D. Notarangelo   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

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

Analysis of Lentiviral Vector Integration in HIV+ Study Subjects Receiving Autologous Infusions of Gene Modified CD4+ T Cells

(2009) Gary P Wang et al.   MOLECULAR THERAPY

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

(2009) Francesco Marangoni et al.   MOLECULAR THERAPY

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

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

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

Transcription Factor Binding Sites Are Genetic Determinants of Retroviral Integration in the Human Genome

(2009) Barbara Felice et al.   PLoS One

Hematopoietic-Specific Lentiviral Vectors Circumvent Cellular Toxicity Due to Ectopic Expression of Wiskott-Aldrich Syndrome Protein

(2008) Miguel G. Toscano et al.   HUMAN GENE THERAPY

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