title: Current progress on gene therapy for primary immunodeficiencies.
abstract: Primary immunodeficiencies have played a major role in the development of gene therapy for monogenic diseases of the bone marrow. The last decade has seen convincing evidence of long-term disease correction as a result of ex vivo viral vector-mediated gene transfer into autologous haematopoietic stem cells. The success of these early studies has been balanced by the development of vector-related insertional mutagenic events. More recently the use of alternative vector designs with self-inactivating designs, which have an improved safety profile has led to the initiation of a wave of new studies that are showing early signs of efficacy. The ongoing development of safer vector platforms and gene-correction technologies together with improvements in cell-transduction techniques and optimised conditioning regimes is likely to make gene therapy amenable for a greater number of PIDs. If long-term efficacy and safety are shown, gene therapy will become a standard treatment option for specific forms of PID.
authors.affiliation: Molecular Immunology Unit, Center for Immunodeficiency, Institute of Child Health, University College London, London, UK.
authors.forename: L
authors.initials: L
authors.lastname: Zhang
authors.forename: A J
authors.initials: AJ
authors.lastname: Thrasher
authors.forename: H B
authors.initials: HB
authors.lastname: Gaspar
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D005822:Genetic Vectors; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D016086:Lentivirus / Q000235:genetics; D016511:Severe Combined Immunodeficiency / Q000628:therapy*; D014161:Transduction, Genetic; D014182:Transplantation, Autologous