标题
Gene therapy for severe combined immunodeficiencies and beyond
作者
关键词
-
出版物
JOURNAL OF EXPERIMENTAL MEDICINE
Volume 217, Issue 2, Pages -
出版商
Rockefeller University Press
发表日期
2019-12-13
DOI
10.1084/jem.20190607
参考文献
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注意：仅列出部分参考文献，下载原文获取全部文献信息。- Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice
- (2019) Hye-Sook Kwon et al. BLOOD
- Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome
- (2019) Katelyn E. Masiuk et al. Cell Stem Cell
- Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives
- (2019) Luigi Naldini EMBO Molecular Medicine
- A New Class of Medicines through DNA Editing
- (2019) Matthew H. Porteus NEW ENGLAND JOURNAL OF MEDICINE
- Lentiviral gene therapy corrects platelet phenotype and function in Wiskott-Aldrich patients
- (2019) Lucia Sereni et al. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
- Inflammatory consequences of inherited disorders affecting neutrophil function
- (2019) Mary C. Dinauer BLOOD
- The γc Family of Cytokines: Basic Biology to Therapeutic Ramifications
- (2019) Warren J. Leonard et al. IMMUNITY
- Long-term ex vivo haematopoietic-stem-cell expansion allows nonconditioned transplantation
- (2019) Adam C. Wilkinson et al. NATURE
- Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors
- (2019) Julian Grünewald et al. NATURE
- Engineering a haematopoietic stem cell niche by revitalizing mesenchymal stromal cells
- (2019) Fumio Nakahara et al. NATURE CELL BIOLOGY
- 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
- Somatic genetic rescue in Mendelian haematopoietic diseases
- (2019) Patrick Revy et al. NATURE REVIEWS GENETICS
- Gene correction for SCID-X1 in long-term hematopoietic stem cells
- (2019) Mara Pavel-Dinu et al. Nature Communications
- Benefit Corporation: a path to affordable gene therapies?
- (2019) Alain Fischer et al. NATURE MEDICINE
- B-cell differentiation and IL-21 response inIL2RG/JAK3SCID patients after hematopoietic stem cell transplantation
- (2018) Alexandra M. Miggelbrink et al. BLOOD
- T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations
- (2018) Sujal Ghosh et al. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
- Transfer of gene-corrected T cells corrects humoral and cytotoxic defects in patients with X-linked lymphoproliferative disease
- (2018) Neelam Panchal et al. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
- Reply to ‘Comment on: Evidence of innate lymphoid cell redundancy in humans’
- (2018) Eric Vivier et al. NATURE 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
- SCID genotype and 6-month post-transplant CD4 count predict survival and immune recovery: a PIDTC retrospective study
- (2018) Elie Haddad et al. BLOOD
- 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
- T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency
- (2018) Erik L. Clarke et al. Genome Medicine
- Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries
- (2018) Juliana Folloni Fernandes et al. JOURNAL OF CLINICAL IMMUNOLOGY
- Base editing: precision chemistry on the genome and transcriptome of living cells
- (2018) Holly A. Rees et al. NATURE REVIEWS GENETICS
- Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency
- (2017) Divya Punwani et al. HUMAN GENE THERAPY
- Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation
- (2017) Valentina Capo et al. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
- Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency
- (2017) Kit L. Shaw et al. JOURNAL OF CLINICAL INVESTIGATION
- 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
- Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice
- (2016) T. Soheili et al. BLOOD
- Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency
- (2016) Maria Pia Cicalese et al. BLOOD
- Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
- (2016) Maria Sessa et al. LANCET
- Evidence of innate lymphoid cell redundancy in humans
- (2016) Frédéric Vély et al. NATURE IMMUNOLOGY
- Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
- (2016) Suk See De Ravin et al. Science Translational 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
- Severe combined immunodeficiencies and related disorders
- (2015) Alain Fischer et al. Nature Reviews Disease Primers
- HLA-haploidentical stem cell transplantation after removal of + T and B cells in children with nonmalignant disorders
- (2014) A. Bertaina et al. BLOOD
- Successful RAG1-SCID gene therapy depends on the level of RAG1 expression
- (2014) Karin Pike-Overzet et al. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
- 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
- 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
- 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 adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans
- (2012) F. Candotti et al. BLOOD
- Thymocytes may persist and differentiate without any input from bone marrow progenitors
- (2012) Laetitia Peaudecerf et al. JOURNAL OF EXPERIMENTAL MEDICINE
- Thymus-autonomous T cell development in the absence of progenitor import
- (2012) Vera C. Martins et al. JOURNAL OF EXPERIMENTAL MEDICINE
- In vivo T-cell dynamics during immune reconstitution after hematopoietic stem cell gene therapy in adenosine deaminase severe combined immune deficiency
- (2011) Silvia Selleri 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
- 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
- Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial
- (2010) G. P. Wang et al. BLOOD
- 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
- 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
- Clinical and immunologic consequences of a somatic reversion in a patient with X-linked severe combined immunodeficiency
- (2008) C. Speckmann et al. BLOOD
- 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
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