Gene therapy is a not a new field and has been evolving for decades. Even with the best efforts of researchers around the globe, there has only been limited success in gene therapy mainly with the common diseases like cancer. Will gene therapy overcome the diseases that have no other cures?
Gene therapy is an experimental technique that treats or prevents diseases through the use of genes. Scientists have focused on diseases that are caused by single-gene defects such as sickle cell anaemia, muscular dystrophy, haemophilia and cystic fibrosis. A gene can't easily be directly inserted into your cells, therefore it's delivered using a carrier called a vector. There are unsolved problems with ...view middle of the document...
There have been 25 published clinical trials for CF whereas non-viral approaches have accounted for nine of these trials. Regardless of the challenges, mainly getting the gene into the cells more efficiently and to make gene expression last longer, the development of better new viruses and liposome's will see the challenges of CF gene therapy overcome.
Haemophilia is a blood disorder which impairs the body's ability to control blood clotting or coagulation, this causes increased bleeding and usually affects males. Symptoms are internal or external bleeding episodes which lead to a severely increased risk of the bleeding being prolongued from common injuries, sometimes the bleeds may be spontaneous and without any real cause for it. Deficiency in coagulation factor VIII is the most common cause of haemophilia. Life expectancy varies with the severity and if the patients receive adequate treatment. Life expectancy varies from not reaching maturity, to a lifespan of approximately 50-60 years. On 10 December 2011, successful treatments of haemophilia B was reported by a team of British and American investigators using gene therapy. In October 2013, the Royal Free London NHS Foundation Trust reported that after they had treated 6 patients with haemophilia in 2010 and early 2011, and that 2 years later all of the patients were still able to produce blood plasma clotting factor this was reported by scientists at University College London and elsewhere reported on 10 December in the New England Journal of Medicine and on 11 December at the American Society of Hematology meeting in San Diego, California.. This shows that gene therapy has made progress towards overcoming haemophilia where other treatments could only prolongue a patient's life. The World Federation of Haemophilia created an infographic to highlight some of the issues facing people with haemophilia (fig.3).
Figure.4 summarises clinical trials performed with various vector systems to treat haemophilia A and which contributed to the understanding of the challenges that face gene therapy with regards to haemophilia in general. Recorded clinical failures include the death of three research subjects when cytotoxic T-cells became dangerous, where infused T-cells avoided tolerisation mechanisms and then attacked healthy tissue.
Diabetes mellitus (DM) is a chronic condition that is associated with extremely high levels of glucose in the blood. Diabetes is due to the cells of the body don't respond to the insulin that is produced properly or due to the pancreas not producing enough insulin. There are three main types of diabetes mellitus:
• Type 1 DM is from the body failing to produce insulin.
• Type 2 DM is from insulin resistance, where cells fail to use the insulin properly, possibly causing a complete insulin deficiency.
• Gestational diabetes takes place when a high blood glucose level is developed in pregnant women without a previous diabetes diagnosis....