![]() This study represents the first clinical application of lentiviral-mediated gene therapy and demonstrates the correction of a neurometabolic disease. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. This study describes the development of lentiviral vectors for gene therapy.Ĭartier, N. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Use of retroviral vectors for gene transfer and expression. Improving gene therapy efficiency through the enrichment of human hematopoietic stem cells. Efficient Ex vivo engineering and expansion of highly purified human hematopoietic stem and progenitor cell populations for gene therapy. This is a proof-of-principle study for the correction of bone marrow failure. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia. ![]() Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. This reports one of the first clinical trials for β-thalassaemia using intrabone injection of a drug product, reporting transfusion independence in paediatric patients.Įichler, F. Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent β-thalassemia. This reports a clinical trial for β-thalassaemia reporting transfusion independence. Gene therapy in patients with transfusion-dependent beta-thalassemia. Current status of stem cell mobilization. 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. Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy. Multiparametric whole blood dissection: a one-shot comprehensive picture of the human hematopoietic system. ![]() Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to plerixafor alone. Gene expression profiling identifies significant differences between the molecular phenotypes of bone marrow-derived and circulating human CD34+ hematopoietic stem cells. Differences in cell cycle kinetics of candidate engrafting cells in human bone marrow and mobilized peripheral blood. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. This study is the first demonstration of clinically successful gene therapy with conditioning. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. This study is one of the first demonstrations of clinically successful gene therapy in humans.Īiuti, A. ![]() Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Amendment to clinical research project, project 90-C-195, January 10, 1992. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives. Death after hematopoietic stem cell transplantation: changes over calendar year time, infections and associated factors. Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. The EBMT Handbook: Hematopoietic Stem Cell Transplantation and Cellular Therapies (Springer, 2019). Hematopoietic stem cell transplantation in its 60s: a platform for cellular therapies.
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