| scholarly article | Q13442814 |
| P50 | author | Alain Fischer | Q2636403 |
| Marina Cavazzana | Q15972056 | ||
| Salima Hacein-Bey-Abina | Q36332714 | ||
| P2860 | cites work | Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease | Q28143064 |
| LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 | Q28210584 | ||
| Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy | Q28215190 | ||
| Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution | Q28251498 | ||
| Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1 | Q28290118 | ||
| Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery | Q29014419 | ||
| Gene therapy for human genetic disease? | Q34227501 | ||
| Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy | Q35011497 | ||
| Gene therapy: twenty-first century medicine | Q36161193 | ||
| Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients | Q36812729 | ||
| Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor. | Q37149903 | ||
| T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. | Q37182935 | ||
| How I treat ADA deficiency | Q37398309 | ||
| Cancer fears cast doubts on future of gene therapy | Q40603071 | ||
| Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning | Q44044895 | ||
| Regulators split on gene therapy as patient shows signs of cancer | Q45855411 | ||
| Safety panel backs principle of gene-therapy trials | Q45857146 | ||
| Gene therapy: shining hopes dented - but not dashed | Q45857292 | ||
| Cancer risk prompts US to curb gene therapy | Q45859108 | ||
| Harmful potential of viral vectors fuels doubts over gene therapy | Q45862715 | ||
| Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients | Q45866834 | ||
| T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. | Q45866838 | ||
| Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector | Q45880148 | ||
| Gene therapy for immunodeficiency due to adenosine deaminase deficiency. | Q45882856 | ||
| Gene therapists hopeful as trials resume with childhood disease | Q47363139 | ||
| NIH open-access plans draw fire from both sides | Q47864428 | ||
| Cancer fears cast doubts on future of gene therapy | Q59072653 | ||
| Immunological reconstitution of sex-linked lymphopenic immunological deficiency | Q70099373 | ||
| Outcry greets US plan to test smallpox vaccine on children | Q78515999 | ||
| A tragic setback | Q78516025 | ||
| Second cancer case halts gene-therapy trials | Q95796374 | ||
| P433 | issue | 6 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 457-460 | |
| P577 | publication date | 2010-06-01 | |
| P1433 | published in | Nature Immunology | Q1071725 |
| P1476 | title | 20 years of gene therapy for SCID. | |
| P478 | volume | 11 |
| Q41343817 | 3D hotspots of recurrent retroviral insertions reveal long-range interactions with cancer genes. |
| Q89076163 | A novel λ integrase-mediated seamless vector transgenesis platform for therapeutic protein expression |
| Q53205248 | A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD). |
| Q35632772 | Advances in Gene Delivery Systems |
| Q38161559 | Agammaglobulinemia: causative mutations and their implications for novel therapies |
| Q27014770 | Cardiovascular gene therapy for myocardial infarction |
| Q36354454 | Cellular calibrators to quantitate T-cell receptor excision circles (TRECs) in clinical samples |
| Q35144895 | Chromatin landscapes of retroviral and transposon integration profiles |
| Q35539617 | Clinical applications of gene therapy for primary immunodeficiencies |
| Q26801506 | Cloning humans? Biological, ethical, and social considerations |
| Q37873887 | Concise review: Transplantation of human hematopoietic cells for extracellular matrix protein deficiency in epidermolysis bullosa |
| Q38085989 | Curing HIV: lessons from cancer therapy |
| Q62491300 | Cyclodextrin-scaffolded glycotransporters for gene delivery |
| Q38912667 | Delivering Nucleic-Acid Based Nanomedicines on Biomaterial Scaffolds for Orthopedic Tissue Repair: Challenges, Progress and Future Perspectives |
| Q36268546 | Development of a diverse human T-cell repertoire despite stringent restriction of hematopoietic clonality in the thymus. |
| Q38341431 | Emerging regenerative approaches for periodontal reconstruction: a systematic review from the AAP Regeneration Workshop |
| Q37858878 | Ex vivo gene transfer and correction for cell-based therapies |
| Q28076919 | Flow Cytometry, a Versatile Tool for Diagnosis and Monitoring of Primary Immunodeficiencies |
| Q38592859 | From Genomics to Gene Therapy: Induced Pluripotent Stem Cells Meet Genome Editing |
| Q64064618 | Gene correction for SCID-X1 in long-term hematopoietic stem cells |
| Q34163207 | Gene therapy finds its niche |
| Q42686916 | Gene therapy for Fanconi anemia: one step closer to the clinic. |
| Q36584648 | Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis |
| Q33413888 | Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity |
| Q35670173 | Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation |
| Q38073152 | Gene therapy for heart failure: where do we stand? |
| Q33881920 | Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. |
| Q38364511 | Gene therapy for primary immunodeficiencies. |
| Q31131479 | Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector |
| Q38607706 | Gene therapy returns to centre stage |
| Q48493269 | Genetics and bioethics: how our thinking has changed since 1969. |
| Q27499229 | Genetics of SCID |
| Q28740951 | Genome-wide target profiling of piggyBac and Tol2 in HEK 293: pros and cons for gene discovery and gene therapy |
| Q38061148 | Haematopoietic stem cell transplantation for SCID patients: where do we stand? |
| Q37945718 | Hematopoietic stem cell expansion and gene therapy |
| Q27023434 | Hematopoietic-stem-cell-based gene therapy for HIV disease |
| Q37974988 | Homologous recombination-based gene therapy for the primary immunodeficiencies |
| Q37850449 | In vivo nucleic acid delivery with PEI and its derivatives: current status and perspectives |
| Q37995010 | Induced pluripotent stem cells and severe combined immunodeficiency: merely disease modeling or potentially a novel cure? |
| Q30409845 | Intravenous injection of a foamy virus vector to correct canine SCID-X1. |
| Q42211507 | Introducing PeptoPlexes: polylysine-block-polysarcosine based polyplexes for transfection of HEK 293T cells. |
| Q64093236 | Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease |
| Q34729193 | Long-lasting production of new T and B cells and T-cell repertoire diversity in patients with primary immunodeficiency who had undergone stem cell transplantation: a single-centre experience. |
| Q45879168 | Lymphopoiesis in transgenic mice over-expressing Artemis |
| Q28293733 | MicroRNAs as therapeutic targets for lung cancer |
| Q38220597 | Moonlighting adenosine deaminase: a target protein for drug development |
| Q36945734 | New frontier in regenerative medicine: site-specific gene correction in patient-specific induced pluripotent stem cells |
| Q45879745 | Nonmyeloablative conditioning generates autoantigen-encoding bone marrow that prevents and cures an experimental autoimmune disease |
| Q26861654 | Nucleic acid therapy for lifespan prolongation: present and future |
| Q35839684 | Physical Characterization of Gemini Surfactant-Based Synthetic Vectors for the Delivery of Linear Covalently Closed (LCC) DNA Ministrings |
| Q33399943 | Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome |
| Q37649782 | Primary Immune Deficiency Treatment Consortium (PIDTC) report |
| Q37055021 | Reduced T cell receptor excision circle levels in the colonic mucosa of microscopic colitis patients indicate local proliferation rather than homing of peripheral lymphocytes to the inflamed mucosa |
| Q45874266 | Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework. |
| Q35749668 | Sampling protein motion and solvent effect during ligand binding |
| Q45886049 | Severe combined immunodeficiencies and related disorders |
| Q35086128 | Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. |
| Q36429491 | Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity |
| Q37870053 | Stem cell self-renewal: lessons from bone marrow, gut and iPS toward clinical applications |
| Q93167184 | Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID |
| Q35224339 | Targeted plasmid integration into the human genome by an engineered zinc-finger recombinase |
| Q55365935 | The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells. |
| Q38698470 | The functional relationship between hematopoietic stem cells and developing T lymphocytes |
| Q41957410 | Translating the lessons from gene therapy to the development of regenerative medicine |
| Q42231996 | Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells. |
| Q37118845 | Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs |
| Q82893669 | [MicroRNAs as therapeutic targets for lung cancer] |
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