Description

Gene therapy is a gene or gene product that can be selectively delivered to a specific cell/tissue with minimal toxicity. Gene therapy is 'the use of genes as medicine. It involves the transfer of a therapeutic or working gene copy into specific cells of an individual in order to repair a faulty gene copy. Thus it may be used to replace a faulty gene, or to introduce a new gene whose function is to cure or to favorably modify the clinical course of a condition. The scope of this new approach to the treatment of a condition is broad, with potential in the treatment of many genetic conditions, some forms of cancer and certain viral infections such as AIDS. Direct targeting of cancer cells with gene therapy has the potential to treat cancer on the basis of its molecular characteristics. But although laboratory results have been extremely encouraging, many practical obstacles need to be overcome before gene therapy can fulfill its goals in the clinic.