| scholarly article | Q13442814 |
| P356 | DOI | 10.1038/S41573-019-0020-9 |
| P698 | PubMed publication ID | 30858502 |
| P50 | author | Isabelle André-Schmutz | Q45929092 |
| Annarita Miccio | Q47147685 | ||
| Marina Cavazzana | Q15972056 | ||
| P2093 | author name string | Frederic D Bushman | |
| Emmanuelle Six | |||
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| Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. | Q45873054 | ||
| Clinical Manifestations and Pathophysiological Mechanisms of the Wiskott-Aldrich Syndrome. | Q45873782 | ||
| Prostaglandin E2 Increases Lentiviral Vector Transduction Efficiency of Adult Human Hematopoietic Stem and Progenitor Cells | Q45873947 | ||
| Ectopic FOXP3 Expression Preserves Primitive Features Of Human Hematopoietic Stem Cells While Impairing Functional T Cell Differentiation. | Q45874670 | ||
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| Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector | Q45880148 | ||
| Engraftment defect of cytokine-cultured adult human mobilized CD34(+) cells is related to reduced adhesion to bone marrow niche elements | Q45880632 | ||
| Failure of SCID-X1 gene therapy in older patients | Q45881034 | ||
| Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions | Q45883007 | ||
| Retroviral transduction of IL-7Ralpha into IL-7Ralpha-/- bone marrow progenitors: correction of lymphoid deficiency and induction of neutrophilia | Q45887435 | ||
| A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition. | Q47125490 | ||
| Gene therapy comes of age. | Q47554885 | ||
| Generation of adult human T-cell progenitors for immunotherapeutic applications | Q49041421 | ||
| Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety | Q50056661 | ||
| Plerixafor enables the safe, rapid, efficient mobilization of haematopoietic stem cells in sickle cell disease patients after exchange transfusion | Q50145999 | ||
| Tumor necrosis factor (TNF)-mediated activation of the p55 TNF receptor negatively regulates maintenance of cycling reconstituting human hematopoietic stem cells. | Q51063194 | ||
| Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease. | Q52319062 | ||
| Sickle cell disease. | Q52354763 | ||
| Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus. | Q52430688 | ||
| Determinants of response and resistance to CD19 chimeric antigen receptor (CAR) T cell therapy of chronic lymphocytic leukemia. | Q52715364 | ||
| Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? | Q53067119 | ||
| HIV integration site selection: targeting in macrophages and the effects of different routes of viral entry. | Q53625219 | ||
| Improving Lentiviral Transduction of CD34+ Hematopoietic Stem and Progenitor Cells. | Q54239249 | ||
| Signaling through toll-like receptor 7/8 induces the differentiation of human bone marrow CD34+ progenitor cells along the myeloid lineage. | Q54575672 | ||
| Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting. | Q55382255 | ||
| Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia | Q56518421 | ||
| Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans | Q57059969 | ||
| Base editing a CRISPR way | Q57060018 | ||
| Disruption of TET2 promotes the therapeutic efficacy of CD19-targeted T cells | Q58889356 | ||
| Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches | Q59340096 | ||
| Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome | Q59360193 | ||
| Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements | Q61440674 | ||
| IFN-gamma negatively modulates self-renewal of repopulating human hemopoietic stem cells | Q64008444 | ||
| Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency | Q64039159 | ||
| Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia | Q64041242 | ||
| Staurosporine Increases Lentiviral Vector Transduction Efficiency of Human Hematopoietic Stem and Progenitor Cells. | Q64973383 | ||
| TLR agonists induce the differentiation of human bone marrow CD34+ progenitors into CD11c+ CD80/86+ DC capable of inducing a Th1-type response | Q81245709 | ||
| Thalassaemia | Q88678149 | ||
| Editing the Epigenome: Reshaping the Genomic Landscape | Q88933788 | ||
| Somatic mutations precede acute myeloid leukemia years before diagnosis | Q89486407 | ||
| HIV infection results in clonal expansions containing integrations within pathogenesis-related biological pathways | Q89574925 | ||
| Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia | Q24618997 | ||
| LEDGF/p75 functions downstream from preintegration complex formation to effect gene-specific HIV-1 integration | Q24671948 | ||
| Gene transfer in humans using a conditionally replicating lentiviral vector | Q24675343 | ||
| Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences | Q24798872 | ||
| Inflammatory modulation of HSCs: viewing the HSC as a foundation for the immune response | Q27003372 | ||
| Demand-adapted regulation of early hematopoiesis in infection and inflammation | Q27011458 | ||
| Inflamm-Aging of Hematopoiesis, Hematopoietic Stem Cells, and the Bone Marrow Microenvironment | Q28074446 | ||
| Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease | Q28143064 | ||
| Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy | Q28215190 | ||
| Efficacy of gene therapy for X-linked severe combined immunodeficiency | Q28288838 | ||
| Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1 | Q28290118 | ||
| Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo | Q28383769 | ||
| BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis | Q28607483 | ||
| Dynamic variation in cycling of hematopoietic stem cells in steady state and inflammation | Q28742707 | ||
| In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector | Q29547503 | ||
| Chimeric antigen receptor T cells for sustained remissions in leukemia | Q29617588 | ||
| Efficacy and toxicity management of 19-28z CAR T cell therapy in B cell acute lymphoblastic leukemia | Q29617590 | ||
| Transcription start regions in the human genome are favored targets for MLV integration | Q29618456 | ||
| HIV-1 integration in the human genome favors active genes and local hotspots | Q29618457 | ||
| Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand? | Q30364488 | ||
| Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells | Q30501325 | ||
| Retroviral DNA integration: viral and cellular determinants of target-site selection | Q33247467 | ||
| HTLV-1 integration into transcriptionally active genomic regions is associated with proviral expression and with HAM/TSP | Q33325922 | ||
| Clinical course of patients with WASP gene mutations | Q33349914 | ||
| Stem-cell gene therapy for the Wiskott-Aldrich syndrome | Q33392766 | ||
| Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome | Q33404960 | ||
| Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity | Q33413888 | ||
| Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients | Q33671980 | ||
| LEDGF hybrids efficiently retarget lentiviral integration into heterochromatin | Q33731166 | ||
| Infection mobilizes hematopoietic stem cells through cooperative NOD-like receptor and Toll-like receptor signaling | Q33857117 | ||
| Conversion of danger signals into cytokine signals by hematopoietic stem and progenitor cells for regulation of stress-induced hematopoiesis | Q33993410 | ||
| A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells | Q34074917 | ||
| Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease | Q34094502 | ||
| WASP: a key immunological multitasker | Q34100506 | ||
| Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer | Q34123303 | ||
| TET genes: new players in DNA demethylation and important determinants for stemness | Q34155544 | ||
| Gammaretroviral vectors: biology, technology and application | Q34223822 | ||
| Transplantation outcomes for severe combined immunodeficiency, 2000-2009. | Q34279715 | ||
| The BET family of proteins targets moloney murine leukemia virus integration near transcription start sites | Q34342339 | ||
| Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. | Q34356904 | ||
| Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy | Q34356914 | ||
| Cyclic GMP-AMP synthase is an innate immune sensor of HIV and other retroviruses | Q34362864 | ||
| Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease | Q34449867 | ||
| Nuclear architecture dictates HIV-1 integration site selection. | Q34465264 | ||
| Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells | Q34473359 | ||
| Gene therapy of chronic granulomatous disease: the engraftment dilemma | Q34473374 | ||
| HIV latency. Proliferation of cells with HIV integrated into cancer genes contributes to persistent infection | Q34502840 | ||
| Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. | Q34508425 | ||
| HIV infection and lymphoma | Q34585452 | ||
| HIV latency. Specific HIV integration sites are linked to clonal expansion and persistence of infected cells | Q34672568 | ||
| The host genomic environment of the provirus determines the abundance of HTLV-1-infected T-cell clones | Q34707243 | ||
| A modified γ-retrovirus vector for X-linked severe combined immunodeficiency | Q34763502 | ||
| Analysis of lentiviral vector integration in HIV+ study subjects receiving autologous infusions of gene modified CD4+ T cells | Q34956211 | ||
| Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy | Q35011497 | ||
| Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice | Q35112505 | ||
| Toll-like receptors on hematopoietic progenitor cells stimulate innate immune system replenishment | Q35113561 | ||
| Prostaglandin E2 enhances human cord blood stem cell xenotransplants and shows long-term safety in preclinical nonhuman primate transplant models. | Q35140717 | ||
| Chromatin organization at the nuclear pore favours HIV replication | Q35196941 | ||
| Decade-long safety and function of retroviral-modified chimeric antigen receptor T cells | Q35202343 | ||
| Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report | Q35615894 | ||
| Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis. | Q35618854 | ||
| Bone marrow homing and engraftment of human hematopoietic stem and progenitor cells is mediated by a polarized membrane domain | Q35802208 | ||
| Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency | Q35850728 | ||
| Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. | Q35913748 | ||
| Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts | Q35944232 | ||
| Characterization of the chicken beta-globin insulator | Q35956059 | ||
| Clinical Studies of Ex Vivo Expansion to Accelerate Engraftment After Umbilical Cord Blood Transplantation: A Systematic Review | Q36249572 | ||
| Primary Immunodeficiency Diseases: an Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency 2015. | Q36319898 | ||
| Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. | Q36370817 | ||
| Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction | Q36370822 | ||
| Immunosurveillance by hematopoietic progenitor cells trafficking through blood, lymph, and peripheral tissues | Q36580764 | ||
| In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia | Q36802562 | ||
| Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients | Q36812729 | ||
| Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line. | Q37066362 | ||
| In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases | Q37089345 | ||
| Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome | Q37089728 | ||
| Integration target site selection by a resurrected human endogenous retrovirus | Q37135161 | ||
| Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia | Q37281429 | ||
| Restoration of NET formation by gene therapy in CGD controls aspergillosis | Q37372812 | ||
| Prostaglandin E2 regulates vertebrate haematopoietic stem cell homeostasis | Q37416219 | ||
| Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy | Q37453594 | ||
| Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients | Q37578901 | ||
| Genome-wide analyses of avian sarcoma virus integration sites | Q37583468 | ||
| IL-1 receptor blockade restores autophagy and reduces inflammation in chronic granulomatous disease in mice and in humans | Q37627905 | ||
| Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells | Q37717887 | ||
| Biosafety features of lentiviral vectors | Q38073294 | ||
| Modulating target site selection during human immunodeficiency virus DNA integration in vitro with an engineered tethering factor | Q38309659 | ||
| Cellular innate immunity and restriction of viral infection: implications for lentiviral gene therapy in human hematopoietic cells | Q38390745 | ||
| Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction | Q38718308 | ||
| CAR T-Cell Therapy: Progress and Prospects | Q38741433 | ||
| CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells | Q38758476 | ||
| Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia | Q38768398 | ||
| Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects | Q38791734 | ||
| Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency | Q38874124 | ||
| In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells | Q39042960 | ||
| 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. | Q39691004 | ||
| Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. | Q39835313 | ||
| Lentiviral vectors escape innate sensing but trigger p53 in human hematopoietic stem and progenitor cells. | Q40139019 | ||
| P433 | issue | 6 | |
| P304 | page(s) | 447-462 | |
| P577 | publication date | 2019-06-01 | |
| P1433 | published in | Nature Reviews Drug Discovery | Q45998 |
| P1476 | title | Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges | |
| P478 | volume | 18 |
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