Cellular gene therapy.
During the last decade, molecular genetic techniques have been used increasingly to transfer human genes into mammalian cells, to correct and enhance cell function, and finally to treat human disease. Despite the current obstacles to developing even the simplest therapeutic strategy, gene therapy promises to have an almost unlimited future. The ability to collect specific blood cells in large numbers, to manipulate their expansion, growth, and differentiation in vitro, and also to cryopreserve these cells for later use has been central to the early developments in gene therapy. This article reviews the major concepts involved in blood cell-based gene therapy, a model for all somatic cell gene therapy.
Lee
J H
JH
Department of Transfusion Medicine, Warren G. Magnuson Clinical Center, National Institutes of Health, Bethesda, Maryland, USA.
Klein
H G
HG
D000818:Animals; D015704:CD4 Antigens / Q000235:genetics / Q000627:therapeutic use; D002478:Cells, Cultured / Q000637:transplantation; D002648:Child; D003673:Defective Viruses / Q000235:genetics; D030342:Genetic Diseases, Inborn / Q000235:genetics / Q000628:therapy; D015316:Genetic Therapy / Q000331:legislation & jurisprudence / Q000379:methods; D005822:Genetic Vectors; D015658:HIV Infections / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007114:Immunization / Q000379:methods; D016219:Immunotherapy, Adoptive; D051379:Mice; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D012190:Retroviridae / Q000235:genetics; D012449:Safety; D016511:Severe Combined Immunodeficiency / Q000201:enzymology / Q000235:genetics / Q000628:therapy; D014162:Transfection
D015704:CD4 Antigens