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Can Germ line Gene Therapy be used as a treatment for Huntingtons Disease?

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Can Germ line Gene Therapy be used as a treatment for Huntington's Disease? Target Audience: Biology A-Level Graduates Report Outline Gene Therapy has been used to reduce the effects of many diseases, but so far, very few solutions have arisen for Huntington's disease. I will be exploring the effectiveness of germ line therapy compared to its risks and morality. Problem Huntington's disease is a genetic condition where there is a mutation in the DNA sequence on the dominant gene 'huntingtin'. The mutation is repetitive so there is more than just one chromosome effected by this mutation. This means the offspring of a Huntington's disease carrier has a 50% chance of developing the disease. Huntington's disease is a neurodegenerative genetic disorder which means the neurones in the brain can lead to a cognitive decline over time. Because this process takes years before it shows any signs of effect, most people don't realize they have Huntington's disease until they reach mid-life. The gene 'huntingtin' codes for the Huntington protein, commonly found around areas of the brain. When a mutation occurs, this protein is no longer produced and instead a different form of protein is produced. ...read more.


They found that providing new genetic instructions which counteracted the production of the mutated protein and a supplement of Huntington's protein, lowered the effects of the disease on mice. "Gene therapy in these models successfully attenuated the symptoms of Huntington's disease and increased life span." (2) Benjamin F. Biaggini, Professor of Biological Sciences. Benefits and Risks One of the most important questions to take into consideration is whether the 'benefits outweigh the risks'. Gene therapy has a wide variety of balanced benefits and risks. This is what makes it such a controversial subject. The main benefit of gene therapy is that it can effectively reduce the symptoms of diseases, diseases such as Huntington's Disease, cystic fibrosis, sickle cell anaemia and many others. This could be a massive medical breakthrough Another benefit is that if germ-line therapy research is continued, we could be able to completely wipe out diseases by removing the gene before it starts to replicate. This is similar to stem cell research as it is working with early embryos to eradicate the disease before it is too late. However, gene therapy is not 100% safe especially with the stage it is at now. ...read more.


Alternatives There are also many effective alternatives to gene therapy which are arguably more cost-effective than gene therapy. Perhaps one of the most popular alternatives is stem cell research. This research has been competing with gene therapy for cures to diseases since the 90's. Stem cell research offers the chance to create a clone of a human being which does not have Huntington's disease. This also has a lot more research needed to be done before it is deemed acceptable by society. Another alternative is drug therapy. This therapy is already being used by most patients suffering from Huntington's disease. Drugs are very cost efficient and simple, but concern has arisen over the effectiveness on patients and these drugs cannot fully eradicate the symptoms of Huntington's disease. Physiotherapy is a kind of treatment that also seems to have good effects on patients. Sufferers can take part in mental and physical exercises which have proven to delay the effects of Huntington's disease. In my opinion, Gene therapy remains the most important way of treating Huntington's disease, although it may require more study and analysis, the future of gene therapy for cures looks promising, and with more finance being invested in research, this could be a medical breakthrough for many genetic diseases. ...read more.

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A sound outline of the arguments surrounding the use of gene therapy but lacking in some detail of the technical processes, and of sociological debates surrounding the issue.

Marked by teacher Jacqui Punter 21/02/2012

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