Published in Sci Transl Med on August 24, 2011
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
Therapeutic genome editing: prospects and challenges. Nat Med (2015) 2.42
Gene therapy returns to centre stage. Nature (2015) 1.95
Quantitative assessment of T cell repertoire recovery after hematopoietic stem cell transplantation. Nat Med (2013) 1.86
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood (2012) 1.85
Targeting long non-coding RNA to therapeutically upregulate gene expression. Nat Rev Drug Discov (2013) 1.82
Gene therapies advance towards finish line. Nat Rev Drug Discov (2011) 1.64
The long quest for neonatal screening for severe combined immunodeficiency. J Allergy Clin Immunol (2012) 1.46
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med (2014) 1.42
Retroviral integrations in gene therapy trials. Mol Ther (2012) 1.34
Gene therapy for PIDs: progress, pitfalls and prospects. Gene (2013) 1.22
Post-transplantation B cell function in different molecular types of SCID. J Clin Immunol (2012) 1.18
Hematopoietic stem cell engineering at a crossroads. Blood (2011) 1.10
Gene therapy on the move. EMBO Mol Med (2013) 1.10
IL-7 in human health and disease. Semin Immunol (2012) 1.08
Gene therapy for primary immunodeficiencies. Hum Gene Ther (2012) 1.03
Gene therapy matures in the clinic. Nat Biotechnol (2012) 1.02
Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy. Transl Res (2013) 1.01
Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther (2012) 0.98
Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells Transl Med (2014) 0.97
Thymidine kinase suicide gene-mediated ganciclovir ablation of autologous gene-modified rhesus hematopoiesis. Mol Ther (2012) 0.94
Impact of next-generation sequencing error on analysis of barcoded plasmid libraries of known complexity and sequence. Nucleic Acids Res (2014) 0.90
History and current status of newborn screening for severe combined immunodeficiency. Semin Perinatol (2015) 0.89
Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line. Hum Gene Ther Methods (2012) 0.89
Clinical applications of gene therapy for primary immunodeficiencies. Hum Gene Ther (2015) 0.88
The case for mandatory newborn screening for severe combined immunodeficiency (SCID). J Clin Immunol (2014) 0.88
Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models. Semin Hematol (2013) 0.86
The human application of gene therapy to re-program T-cell specificity using chimeric antigen receptors. Chin J Cancer (2014) 0.85
HSV-sr39TK positron emission tomography and suicide gene elimination of human hematopoietic stem cells and their progeny in humanized mice. Cancer Res (2014) 0.85
Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector. PLoS One (2013) 0.85
Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. Gene Ther (2012) 0.83
"Special exemptions": should they be put on trial? Mol Ther (2013) 0.80
Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning. J Clin Immunol (2015) 0.80
Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection. PLoS One (2012) 0.80
Lentiviral vectors for the treatment of primary immunodeficiencies. J Inherit Metab Dis (2014) 0.79
Development of gene therapy for thalassemia. Cold Spring Harb Perspect Med (2012) 0.79
Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies. Front Immunol (2015) 0.79
A phase I dose escalation study of Ad GV.EGR.TNF.11D (TNFerade™ Biologic) with concurrent chemoradiotherapy in patients with recurrent head and neck cancer undergoing reirradiation. Ann Oncol (2012) 0.79
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand? Hum Gene Ther (2016) 0.79
Current Progress in Therapeutic Gene Editing for Monogenic Diseases. Mol Ther (2016) 0.78
Use of V(D)J recombination excision circles to identify T- and B-cell defects and to monitor the treatment in primary and acquired immunodeficiencies. J Transl Med (2013) 0.78
Retroviral vectors: from cancer viruses to therapeutic tools. Hum Gene Ther (2014) 0.77
T-cell Receptor and K-deleting Recombination Excision Circles in Newborn Screening of T- and B-cell Defects: Review of the Literature and Future Challenges. J Public Health Res (2013) 0.77
Alpharetroviral vectors: from a cancer-causing agent to a useful tool for human gene therapy. Viruses (2014) 0.77
Gene therapy researchers' assessments of risks and perceptions of risk acceptability in clinical trials. Mol Ther (2013) 0.77
Rapid and Efficient Stable Gene Transfer to Mesenchymal Stromal Cells Using a Modified Foamy Virus Vector. Mol Ther (2016) 0.77
Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A. J Genet Syndr Gene Ther (2011) 0.77
Current translational and clinical practices in hematopoietic cell and gene therapy. Cytotherapy (2012) 0.77
Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice. Hum Gene Ther (2014) 0.76
Gene therapy studies in a canine model of X-linked severe combined immunodeficiency. Hum Gene Ther Clin Dev (2015) 0.76
Gene therapy for SCID-X1: focus on clinical data. Mol Ther (2011) 0.76
Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery. Mol Ther Methods Clin Dev (2017) 0.75
Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders. J Mov Disord (2016) 0.75
Evolving Gene Therapy in Primary Immunodeficiency. Mol Ther (2017) 0.75
Recent Advances in Lentiviral Vaccines for HIV-1 Infection. Front Immunol (2016) 0.75
Genetic Engineering and Manufacturing of Hematopoietic Stem Cells. Mol Ther Methods Clin Dev (2017) 0.75
Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy. Mol Ther Methods Clin Dev (2017) 0.75
Stem cells in translation: impression of the ISSCR Regional Meeting in Florence. Stem Cell Reports (2013) 0.75
Effect of fetal bovine serum on foamy and lentiviral vector production. Hum Gene Ther Methods (2013) 0.75
Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy. Mol Ther Methods Clin Dev (2017) 0.75
Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med (2008) 15.30
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med (2006) 9.68
Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med (2009) 8.20
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med (2002) 6.25
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med (2010) 4.76
Impaired response to interferon-alpha/beta and lethal viral disease in human STAT1 deficiency. Nat Genet (2003) 3.95
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
A robust model for read count data in exome sequencing experiments and implications for copy number variant calling. Bioinformatics (2012) 3.10
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest (2007) 2.80
Essential role of the NADPH oxidase subunit p47(phox) in endothelial cell superoxide production in response to phorbol ester and tumor necrosis factor-alpha. Circ Res (2002) 2.79
Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood (2007) 2.79
Effective gene therapy with nonintegrating lentiviral vectors. Nat Med (2006) 2.70
Adaptor protein 3-dependent microtubule-mediated movement of lytic granules to the immunological synapse. Nat Immunol (2003) 2.56
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther (2009) 2.56
Th17 plasticity in human autoimmune arthritis is driven by the inflammatory environment. Proc Natl Acad Sci U S A (2010) 2.43
Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol (2010) 2.35
The Wiskott-Aldrich syndrome. J Allergy Clin Immunol (2006) 2.29
Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency. Medicine (Baltimore) (2010) 2.26
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther (2006) 2.08
Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study. Lancet (2009) 1.96
Adoptive immunotherapy with allodepleted donor T-cells improves immune reconstitution after haploidentical stem cell transplantation. Blood (2006) 1.93
A prospective evaluation of degranulation assays in the rapid diagnosis of familial hemophagocytic syndromes. Blood (2012) 1.92
Impact of thymoglobulin prior to pediatric unrelated umbilical cord blood transplantation on immune reconstitution and clinical outcome. Blood (2013) 1.90
2013 IDSA clinical practice guideline for vaccination of the immunocompromised host. Clin Infect Dis (2014) 1.81
Stable gene transfer to muscle using non-integrating lentiviral vectors. Mol Ther (2007) 1.81
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci Transl Med (2011) 1.81
Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia. Blood (2006) 1.80
Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. Blood (2012) 1.80
The cytoplasm of living cells behaves as a poroelastic material. Nat Mater (2013) 1.80
The leukocyte podosome. Eur J Cell Biol (2005) 1.72
2013 IDSA clinical practice guideline for vaccination of the immunocompromised host. Clin Infect Dis (2013) 1.69
Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood (2010) 1.68
Cytoskeletal remodeling mediated by WASp in dendritic cells is necessary for normal immune synapse formation and T-cell priming. Blood (2011) 1.67
Megakaryocytes assemble podosomes that degrade matrix and protrude through basement membrane. Blood (2013) 1.65
Inhibition of IkappaB kinase by vaccinia virus virulence factor B14. PLoS Pathog (2008) 1.63
Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. Blood (2011) 1.62
Vaccinia virus proteins A52 and B14 Share a Bcl-2-like fold but have evolved to inhibit NF-kappaB rather than apoptosis. PLoS Pathog (2008) 1.57
Wiskott-Aldrich syndrome protein deficiency leads to reduced B-cell adhesion, migration, and homing, and a delayed humoral immune response. Blood (2004) 1.56
Deficiency in the Wiskott-Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment. Blood (2006) 1.56
A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors. Mol Ther (2010) 1.54
Diminished production of anti-inflammatory mediators during neutrophil apoptosis and macrophage phagocytosis in chronic granulomatous disease (CGD). J Leukoc Biol (2003) 1.53
X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options. Blood (2010) 1.51
Autoimmune lymphoproliferative syndrome: molecular basis of disease and clinical phenotype. Br J Haematol (2006) 1.49
Unregulated actin polymerization by WASp causes defects of mitosis and cytokinesis in X-linked neutropenia. J Exp Med (2007) 1.49
Inhibition of calpain stabilises podosomes and impairs dendritic cell motility. J Cell Sci (2006) 1.47
An Artemis polymorphic variant reduces Artemis activity and confers cellular radiosensitivity. DNA Repair (Amst) (2010) 1.46
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol Ther (2008) 1.44
SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease. Blood (2012) 1.44
WIP regulates the stability and localization of WASP to podosomes in migrating dendritic cells. Curr Biol (2006) 1.43
Update on the hyper immunoglobulin M syndromes. Br J Haematol (2010) 1.43