What is Gene Therapy?
Gene therapy is a relatively new concept, it is an experimental technique that uses genes to theoretically treat and prevent various types of Illnesses/Genetic Disorders such as Haemophilia, Cancer, Chronic Granulomatous Disorder and Cystic Fibrosis. The process of gene therapy involves the use of a Viral Vector (Most Common Form) other forms of delivery which are non-viral include the injection of naked DNA, sonoporation and magnetofection.
Figure 1 .1 Shows us how an unmodified Virus under normal conditions work.
How It Works?
The viruses DNA is removed and replaced by the needed DNA that corresponds to the specific required gene, which is then to be inserted ...view middle of the document...
A disorder that falls under the required criteria perfectly, this hereditary disorder affects the cells of the lungs, pancreas and small intestines the effect causes abnormal mucus secretions in these areas increasing their vulnerability towards infection causing fatal complications. It is believed that Gene Therapy can be a possible solution in fixing the mutated gene that causes this disorder, this affected gene is CFTR responsible for the regulation of the components of sweat.
Figure1.3 Showing the effect of Cystic Fibrosis
Gene Therapy in 2014: Choroideremia Trial
Choroideremia is a rare inheritable disease that causes blindness it affects around 1 in 50,000 people, there is currently now cure for it. It is caused by defects in the CHM gene on the X chromosome, this causes the protein produced by the CHM not to be present, slowly shutting down (Killing Off) the pigment cells in the retina of the eye one by one. As this disease progresses, the surviving retina gradually shrinks, reducing the victim’s vision.
The aim of the treatment in this trial was to get the gene therapy into the cells in the retina of the eye without causing damage to the eye itself. After six months the patients showed improvements in their vision in dim light and two of the six tested were able to read more lines on an eye chart than before the treatment.
Results have been collected from the first six patients that had undergone the treatment, because of the success of the first six patients, three more patients have recently been tested at a higher dosage.
Professor MacLaren of Oxford University says “It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained for as long as we have been following up the patients, which is two years in one case. The results showing improvement in vision in the first six patients confirm that the virus can deliver its DNA payload without causing significant damage to the retina. This has huge implications for anyone with a genetic retinal disease such as age-related macular degeneration or retinitis pigmentosa, because it has for the first time shown that gene therapy can be applied safely before the onset of vision loss.”
The Trial was a success and is still in progress. Gene therapy definitely seems like a viable solution in the future and with more knowledge about the practical use and manipulation of the technique one will be able to cure other serious diseases.
Figure 1.4 Showing the amount of Gene Therapy trials accepted
They are calling it the future of medicine, what does this entail?
The advantages include:
• It allows windows of opportunity to open for people that are suffering from genetic diseases which are currently untreatable, it might give them a chance to live a normal life without hospitals and pills
• The avoidance of normal drug side affects
• Gene therapy has the potential to ultimately...