title: Lentivirus technologies for modulation of the immune system.
abstract: Lentiviral vectors (LVV) are important tools for the treatment of immune system disorders. Integration of therapeutic genetic material into the haematopoietic stem cell compartment using LVV can mediate long-term correction of haematopoietic lineages, thereby correcting disease phenotypes. Twenty years of vector development have successfully brought LVV to the clinic, with follow up studies of clinical trials treating primary immunodeficiencies now being reported. Results have demonstrated clear improvements in the quality of life for patients with a number of conditions in the absence of the severe adverse events observed in earlier retroviral gene therapy trials. Growing interest in gene modified adoptive T cell transfer as an alternative strategy has driven further technology innovation, including characterisation of novel viral envelopes. We will also discuss the progression of gene editing technology to preclinical investigations in models of immune deficiency.
authors.affiliation: Molecular and Cellular Immunology, Institute of Child Health, University College London, London WC1N 1EH, UK.
authors.forename: Benjamin C
authors.initials: BC
authors.lastname: Houghton
authors.affiliation: Molecular and Cellular Immunology, Institute of Child Health, University College London, London WC1N 1EH, UK; Department of Paediatric Immunology, Great Ormond Street Hospital NHS Foundation Trust, London WC1N 3JH, UK. Electronic address: C.Booth@ucl.ac.uk.
authors.forename: Claire
authors.initials: C
authors.lastname: Booth
authors.affiliation: Molecular and Cellular Immunology, Institute of Child Health, University College London, London WC1N 1EH, UK; Department of Paediatric Immunology, Great Ormond Street Hospital NHS Foundation Trust, London WC1N 3JH, UK.
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
mesh_terms: D000818:Animals; D006801:Humans; D007107:Immune System; D007153:Immunologic Deficiency Syndromes / Q000276:immunology / Q000628:therapy*; D016086:Lentivirus / Q000235:genetics*