Final Web Blog Essay

My Essay's Research Question:

• How can the application of gene therapy help cure Alzheimer's disease?

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"Gene Therapy: Good or Bad?" by Halya

   The first gene therapy on humans that was successful happened in 1990 in the ‘NIOH’ (National Institutes of Health). The first person who received this treatment was a 4 year old child who had adenosine deaminase deficiency (usually called ‘ADA’), which is a genetic disease where children are born with critical failure in the immune system that is used to protect infections. They are believed to have serious infections continuously. Gene therapy has been trying to treat diseases like an immunodeficiency disease such as SCID, cystic fibrosis, Canavan's disease and Gaucher's disease since 1990. SCID is a disease where children has a shortage of natural defenses against infections and will only be able to live in lonely environments, which means that the only way it can be cured is by doing gene therapy.

     Alzheimer's disease is a disease where a person loses their memory progressively. Most of the people who has this disease are middle-aged or old. There are currently no cures for this disease, unfortunately. It was said that approximately 44 million people are living with Alzheimer's disease all around the world. If there's no cure to Alzheimer's disease, there will most likely be an increase to almost 76 million. In 2050, there is a possibility that the disease will affect 135 million people. But by using gene therapy, it might cure it.

     Scientists have successfully switched off a gene that was assumed of the cause of Alzheimer's disease, by using a different and latest technique to transport the drugs straight to the subject’s brain. They tried testing this new technique out on mice. They used small particles usually called 'exosomes' that are let out by a number of cells, and are directly sent to the mice's brain. Exosomes can be used to transfer gene therapy to certain specified genes in the subject’s brain. One of the genes are BACE1, that is related with Alzheimer's disease. Exosomes come across as delivering special carriages to brain cells, so that the technology has a few embryonic applications. They have not been tested on human genes because they might have some technical errors during the process.

    Ex vivo gene therapy is a type of gene therapy that can be used to treat Alzheimer's disease. Ex vivo gene therapy are cells that are genetically modified in vitro to show the gene we want to use. Biological micropumps act as an effect for secretion of the protein you need, where they are implanted in the brain. The advantages of treating Alzheimer's disease with gene therapy is that they can target a certain cell type for the production of genes that are going to be used before placing the cells in the subject's brain. Another benefit is safety because it's rare for a virus to pass through since the viruses are not genetically modified by the cells. But, there are always limitations to benefits. One of the disadvantages are that to prolong and genetically modified in vitro, the subject's cells must be able to divide, or else a specific postmitotic cell populations cannot be the target for ex viro gene therapy. The other disadvantage is that surgically transplanting a living tissue is a much easier process than gene therapy.

     Gene therapy potentially creates empathy with the society as it has many elements of ethics such as body shape, certain disorders or disabilities and also the fact that because it is an expensive treatment, it would apparently be available specially for the rich people. It would also be unacceptable to judge which traits are normal or abnormal from the human's perspectives. And finally, once such therapy is being applied in general, it's still doubtful if we are allowed to use it for the future generation.

     In conclusion, I think gene therapy is good for people who are affected with deadly disease. I also think that it has a great technique to help cure people who really need it. But, for people who are just normal, like almost every other human being, should just stick with what God gave them because in God's perspective we are already perfect. We are also 'perfect' in our own way.

Resources:

• Ama-assn.org, (2014). Gene Therapy. [online] Available at: http://www.ama-assn.org/ama/pub/physician-resources/medical-science/genetics-molecular-medicine/current-topics/gene-therapy.page
• Nhs.uk, (2011). New method of drug delivery to the brain - Health News - NHS Choices. [online] Available at: http://www.nhs.uk/news/2011/03March/Pages/new-method-of-drug-delivery-to-the-brain.aspx 
• Brightfocus.org, (2014). Alzheimer's Facts & Statistics. [online] Available at: http://www.brightfocus.org/alzheimers/about/understanding/facts.html
• Medscape.com, (2014). Growth Factor Gene Therapy for Alzheimer Disease: Gene Delivery of NGF. [online] Available at: http://www.medscape.com/viewarticle/446200_3
• Ncbi.nlm.nih.gov, (2014). Ex vivo gene therapy for Alzheimer's dise... [Clin Neurosci. 1995-1996] - PubMed - NCBI. [online] Available at: http://www.ncbi.nlm.nih.gov/pubmed/8914793
• Genetics Home Reference, (2014). What are the ethical issues surrounding gene therapy?. [online] Available at: http://ghr.nlm.nih.gov/handbook/therapy/ethics

Book Sources:

• Claybourne, A., Moncrieff, S., Reiss, M., Brooks, F. and Seay, C. (2003). The Usborne internet-linked introduction to genes & DNA. Tulsa, Okla.: EDC Pub.

Video 1: This video shows the studies of gene therapy for people suffering from Alzheimer's disease. Also, how it can help cure it.

Source: https://www.youtube.com/watch?v=jg8nQi-LebI

      In this video, they were talking about how a couple dealt with Alzheimer's disease; what they did and how they coped through their journey. Richard, the husband who has Alzheimer's, was the very first patient in a certain hospital to get gene therapy for his disease. They injected a virus straight to his brain by injecting it through his skull. This virus will help the person cope with the disease better and prevent him from dying from Alzheimer's.