Gene Therapy refers to a technique in which a healthy DNA is inserted in a person’s cells in order to cure a disease or to replace a mutated gene. Either a healthy gene is inserted directly or a gene encoding for a therapeutic protein is used.
In Preventive Gene therapy, a gene is inserted at a young age before the defected gene is expressed. A DNA carrier or a vector is used to transfer the new DNA in the body,these vectors can be viruses or a non-viral method like insertion of naked DNA and electroporation can be used. Uptil now, the diseases that have been shown to be cured using gene therapy include Cystic fibrosis, SCID, haemophilia, Parkinson’s disease, Myeloma and Leukemia.
Most of the genetic disorders cured by the gene therapy are caused by one mutated gene, as those with multiple mutated genes are still very difficult to cure even with the latest techniques of Gene Therapy. Although gene therapy has a tremendous potential for good, it has a very limited use due to the disadvantages associated with the process right now.
Our immune system attacks any invader it detects and destroys it, so making sure that the DNA would reach its required site in the cell becomes very difficult.
Another disadvantage is the short term activated DNA or genes. For a disease to be cured completely, the inserted DNA must stay activated for a long times, which doesn’t happen in most of the cases, means a person would need multiple rounds of therapy before seeing any significant improvement.
The vector or the viral particles also pose danger to human body, as they can regain their virulence and result in serious viral infections and may also lead to toxification. Beside that, the inserted DNA can easily get damaged or mutated during rapid cell division and can cause cancer.
However, despite all the disadvantages, gene therapy will soon become a reality making life disease free by helping scientists discover new ways of defeating Cancer, AIDS, Diabetes and other dreadful ailments.
This video will explain you how gene therapy works.
[Video Credit: DNews]