Progress and problems with the use of viral vectors for gene therapy.
Gene therapy has a history of controversy. Encouraging results are starting to emerge from the clinic, but questions are still being asked about the safety of this new molecular medicine. With the development of a leukaemia-like syndrome in two of the small number of patients that have been cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.
Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA.
Clare E
CE
Thomas
Anja
A
Ehrhardt
Mark A
MA
Kay
D000256:Adenoviridae / Q000235:genetics; D003673:Defective Viruses / Q000235:genetics; D000229:Dependovirus / Q000235:genetics; D015316:Genetic Therapy / Q000639:trends*; D005822:Genetic Vectors; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D009369:Neoplasms / Q000628:therapy*; D012190:Retroviridae / Q000235:genetics; D018139:Simplexvirus / Q000235:genetics; D014780:Viruses / Q000235:genetics*