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Gene therapy: applications and progress towards the clinic

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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. (Intern Med J 2004; 34: 621−625)
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Keywords: SCID; cellular therapies; gene transfer; retrovirus; stem cell; transduction

Document Type: Research Article

Publication date: 01 November 2004

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