Gene Therapy

Gene therapy

Each of us carries about half a dozen defective genes. We remain blissfully unaware of this fact unless we, or one of our close relatives, are amongst the many millions who suffer from a genetic disease. Genes are the building blocks of inheritance. Passed from parent to child, they contain instructions for making proteins. If genes don't produce the right proteins or don't produce them correctly, a child can have genetic disorder. Gene therapy is a recombinant DNA process in which cells are taken from the patient, altered by adding genes, and replaced in the patient, where the genes provide the genetic codes for proteins the patient is lacking.

I chose a news article that focuses on gene therapy study that shows significant improvement in Parkinson's disease. New evidence indicates that the first gene therapy for Parkinson's disease has achieved measurable success. Brain scans of patients receiving the treatment confirmed significant changes. Doctors participating in the study injected a adeno-associated virus (AAV) vector to deliver an inhibitory gene (glutamic acid decarboxylase or “GAD”) to the subthalmic nucleus (STN) of the brain. In Parkinson’s disease, STN activity is abnormally increased, largely due to a deficit in GABA (gamma-aminobutyric acid). GABA is the major inhibitory neurotransmitter in the brain. Increasing GAD causes more GABA to be synthesized, thus helping to calm the STN over-activity. The procedure was performed on only one side of the brain, enabling the untreated side to serve as a study control (1).

Gene therapy technologies are now making it possible to provide treatments that 20 years ago were hardly dreamed possible. These new technologies bring a whole new era in medicine. The answers to many medical problems people thought were incurable are now becoming possible. Like other new forms of therapy, gene therapy raises ethical and social