title: New tools in regenerative medicine: gene therapy.
abstract: Gene therapy aims to transfer genetic material into cells to provide them with new functions. A gene transfer agent has to be safe, capable of expressing the desired gene for a sustained period of time in a sufficiently large population of cells to produce a biological effect. Identifying a gene transfer tool that meets all of these criteria has proven to be a difficult objective. Viral and nonviral vectors, in vivo, ex vivo and in situ strategies co-exist at present, although ex vivo lenti-or retroviral vectors are presently the most popular.Natural stem cells (from embryonic, hematopoietic, mesenchymal, or adult tissues) or induced progenitor stem (iPS) cells can be modified by gene therapy for use in regenerative medicine. Among them, hematopoietic stem cells have shown clear clinical benefit, but iPS cells hold humongous potential with no ethical concerns.
authors.affiliation: Department of Immunology, Universidad Complutense de Madrid, Spain.
authors.forename: Miguel
authors.initials: M
authors.lastname: Muñoz Ruiz
authors.forename: José R
authors.initials: JR
authors.lastname: Regueiro
mesh_terms: D002986:Clinical Trials as Topic; D015316:Genetic Therapy / Q000295:instrumentation / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics / Q000378:metabolism; D006801:Humans; D044968:Regenerative Medicine / Q000295:instrumentation* / Q000379:methods*; D013234:Stem Cells / Q000166:cytology / Q000502:physiology; D019076:Transgenes