Gene therapy: applications and progress towards the clinic.
Gene therapy was originally conceived as an approach to the treatment of genetic disease, to repair or replace a faulty gene. Subsequently, gene therapy clinical trials have been undertaken for a wide range of conditions, particularly cancer and AIDS. Overall, the results from gene therapy have been disappointing. The reasons include the following: (i) low gene transfer efficiencies and (ii) shortcomings in the identification and manipulation of appropriate target cells, including progenitor cell populations required for the maintenance of long-term effects. Today, the immense potential of gene therapy remains, but more basic research is required to improve technical aspects of this form of cellular therapy.
Trent
R J A
RJ
Department of Molecular and Clinical Genetics, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia. rtrent@med.usyd.edu.au
Alexander
I E
IE
D001315:Australia; D002986:Clinical Trials as Topic; D018014:Gene Transfer Techniques / Q000639:trends*; D015316:Genetic Therapy / Q000379:methods / Q000639:trends*; D006801:Humans