Gene therapy is the insertion of genes into an individual cell and tissue to treat a disease, and herditary diseases in particular. Gene therapy is unique in that as it employs the genetic material.DNA itself as the means of treatment. Gene therapy typically aims to supplement defective mutant allele with a functional one. Although, the technology is still in its infancy, it has been used with some success. Most, if not all, disease have a genetic factor.
The genetic factor can be wholly or partially responsible for the diseases. For examples, in disorders such as systic fibrosis, hemophilia, and muscular dystrophy, changes in a gene directly result in the condition. In other conditions such as high cholesterol and high blood presure, genetic and environmental factors interact to cause disease. Disorders associated with ageing often involve the loss of gene activity in specific type of cells.
Even infections can be related to genes. In fact, they have two sets of genetic genes of the person with the infection.
Gene therapy comprises of two approaches: direct approach to gene therapy involves the delivery of transmembrane protein whose inactivation results in the lung disease cystic fibroses. Another example is (SCID), which affects children, making them highly appropriate genes involves the delivery of functional gene encoding adensine deaminase. Indirect approach is used where disease does by replacing a gene, the protein produced by the thearapeutic gene will produce some indirect benefit in the patient. Cancer is one example of this indirect approach where several trials of gene therapy have already been undertaken. One means of killing the cancer cell is to deliver a gene encoding enzyme that converts a harmless substance known as pro-drug into a lethat drug.
If the gene encoding the enzyme can be deliverd specifically to the cancer cells, the pro-drug will be converted to the lethal drug by the cancer cells, so killing them while the normal cells survive.
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