Gene Therapy

Gene therapy is the replacement of a defective gene in an organism suffering from a genetic disease; recombinant DNA techniques are used to isolate the functioning gene and insert it into cells (More than 300 single-gene genetic disorders have been identified in humans; a significant percentage of these may be amenable to gene therapy).

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.