Wednesday, 4 April 2012



So we all know that there are some diseases that modern medicine cannot cure. One method that is becoming very popular is gene therapy. Basically, what they do is try to fix the gene that has gone haywire, and this theoretically would prevent the spread of these disease causing genes.(Campbell, N. A., Reece J.B., 2002.) Notice that this method attempts to treat the origin of the disease, rather than its symptoms. How scientists usually go about in performing gene therapy involves the injection of a genetically modified vector virus that is able to carry the introduced genes to the target cell's genome. The idea behind this process is that the genome would accept this new encoded gene, and will then be able to reproduce new normal genes. This is just one of the methods for gene therapy. Other methods include genetic manipulation or deactivation of the defected gene. (Seppa, N. , 2012)

Gene therapy is still at an experimental stage, because its effects are not permanent and further research is needed. One reason is that the cell isn't able to duplicate the foreign gene, because of the rapidly dividing nature of many cells prevents the gene from receiving long term benefit, and multiple rounds of therapy would be needed. Also, the introduced virus cannot be controlled by the body.(Seppa, N. 2012)

Recently, there has been some success treatment of Parkinson's disease with the use of gene therapy with a group involving 65 people. Half underwent a placebo surgery, while the other half received gene therapy (Antonini, A. 2011). The gene therapy involved an injection of an inactive virus carrying a GAD gene into the brain, because this area of the brain becomes over reactive in people with Parkinson's disease the muscle movement signals are blocked, due to the lack of GABA neurotransmitters. The introduction of the new gene begins new production of these neurotransmitters, which improves muscle movement.

Six months after the treatment, 23.1% got better in the group that received gene therapy compared to the 12.7% in the placebo group (Antonini, A. 2011). Also, no one had side effects from the vector virus. Although people got better, it wasn't considered a big clinical effect.  Further study is needed on larger groups, and on larger doses of gene therapy. Much more research is needed to help scientist discover how to utilise the vector virus so that the introduced genes can be continuously duplicated.

There are good and bad things about gene therapy. Obviously, the good thing is that gene therapy can potentially improve disabilities and save lives. The bad things are the ethical issues surrounding humans manipulating gene, acting like god. There are also unanswered questions about whether the transferred virus would make us sicker. For all we know, the insertion of this genetically modified gene could damage other cell function. But because we don't know this, much more research is needed. Aside from all of these unanswered questions, gene therapy can potentially cure the incurable.

By Sam Banh

Gene therapy for Parkinson's passes the ultimate test - health - New Scientist. 2012. Jabr, F. Gene therapy for Parkinson's passes the ultimate test - health - 17 March 2011 - New Scientist. Available at: [Accessed 18 March 2012]

Gene Therapy For Parkinson's Advances - Science News. 2012. Seppa, N. Gene Therapy For Parkinson's Advances - Science News. [ONLINE] Available at: [Accessed 15 March 2012]

Antonini, A. A, 2011. Movement disorders: Towards New Therapies in Parkinson's Disease. Movement disorders: Towards New Therapies in Parkinson's Disease, 1/11, 7-9

Campbell, N. A., Reece J.B., 2002. Biology. 6th ed. San Francisco USA: Pearson Eucation, Inc

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