Progress and problems with the use of viral vectors for gene therapy

CE Thomas, A Ehrhardt, MA Kay - Nature Reviews Genetics, 2003 - nature.com
CE Thomas, A Ehrhardt, MA Kay
Nature Reviews Genetics, 2003nature.com
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.
Abstract
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.
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