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.

Limitations of Gene Therapy

Original Paragraph:
The basis of gene therapy is find a gene that is not functioning right and to insert a healthy portion into that gene. To find these genes, scientists must perform genetic tests or genetic screening to see if the gene that causes for example, cystic fibrosis, is present. This genetic testing is producing much controversy and raising many ethical and legal problems. Many believe that this is an invasion of privacy. They believe that if prenatal tests are performed that these could lead to an increase in the number of abortions. Many people that hear a positive test for a disease are now are faced with a dilemma. That dilemma is in finding adequate insurance to cover the treatments, which could be gene therapy, or living out their lives knowing that they carry a gene for a disease. This positive test has placed them in a high risk group that they may have not been in if they had not been tested. This high risk tag makes insurance coverage almost impossible to find. The insurance that will cover them may be extremely expensive. So, choosing to have the test may be a question of whether they think their current insurance will cover them or not. Many young couples may find out that they are carriers of a disease, and now must decide if they want to have a child that could be born with a genetic disease. Still another problem is regulation of the uses of gene therapy. Our society is obsessed with the idea of youth and beauty. If scientists could identify the gene that contributes to youth or beauty, then the technique of gene therapy could be monopolized by the cosmetic industry to enhance beauty or to "turn back the clock." The problem with this is whether baldness, height, or beauty should be enough of a reason to require gene therapy. Federal regulation will have to come into play in deciding whether a big nose is as important of a genetic problem as cystic fibrosis and which requires gene therapy.

Key Ideas:

• Gene therapy produces too much controversy and it's raising legal and ethical issues.
• A lot of people think that genetic testing is an invasion of privacy.
• They believe that if they found out about their baby's genetics, it might lead to a number of abortions.
• When people hear that they have positive results for the disease, they are often faced with dilemma.
• The dilemma is that they're not sure whether to get an insurance to help cure the disease with treatments (maybe gene therapy), or living their life with that certain disease.
• If they are positive, then it will put them in a high risk if they have not been tested.
• This high risk will cause the coverage to be hard to find.
• The insurance will sure be expensive.
• So they should decide if they actually want to get tested or not.
• Many couples might find out that the baby they're carrying has a disease, and they have to decide if they want the baby to be worn with the disease.
• Since the society today is infatuated by youth and beauty, they would be able to have an abortion because the baby's not perfect.
• The issue is that the height, beauty or weight isn't a reason to have gene therapy.
• The federal regulation will have a role in this when it comes to deciding if it's actually a problem or not.

Source in MLA: Ndsu.edu, (2014). Gene Therapy. [online] Available at: http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/brandi.htm [Accessed 15 Oct. 2014].

Paraphrase:
Gene therapy produces too much controversy and it's raising legal and ethical issues. A lot of people think that genetic testing is an invasion of privacy. They believe that if they found out about their baby's genetics, it might lead to a number of abortions. When people hear that they have positive results for the disease, they are often faced with dilemma. The dilemma is that they're not sure whether to get an insurance to help cure the disease with treatments (maybe gene therapy), or living their life with that certain disease. If they are positive, then it will put them in a high risk if they have not been tested. This high risk will cause the coverage to be hard to find. The insurance will sure be expensive. So they should decide if they actually want to get tested or not. Many couples might find out that the baby they're carrying has a disease, and they have to decide if they want the baby to be worn with the disease. Since the society today is infatuated by youth and beauty, they would be able to have an abortion because the baby's not perfect. The issue is that the height, beauty or weight isn't a reason to have gene therapy. The federal regulation will have a role in this when it comes to deciding if it's actually a problem or not.

Figure 1: This pictures explains how genetic screening works.
Source: http://www.theage.com.au/ffxImage/urlpicture_id_1047145003009_2003/03/12/genetics2.jpg

     In Figure 1, this picture is talking about how genetic screening works, since I talked about it earlier on how some couples find out about their baby's genetic diseases through genetic screening. So in this picture, it's saying that the birth mother is given fertility drugs, then multiple eggs are produced and then are fertilized to make embryos. Then the embryos are analyzed in the lab for any genetic limitations or a tissue that doesn't work well. After that, they inject only healthy embryos to the uterus and the mother finally gives birth to a healthy baby.

Introduction of Gene therapy

Original Paragraph:

Gene therapy is likely to be most successful with diseases caused by single gene defects. The first successful gene therapy on humans was performed in 1990 by researchers at the National Institutes of Health. The therapy treated a four-year-old child for adenosine deaminase (ADA) deficiency, a rare genetic disease in which children are born with severe immunodeficiency and are prone to repeated serious infections.

Since 1990, gene therapy had been tested in human clinical trials for treating such diseases as severe combined immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease, and Gaucher's disease. In 2003, more than 600 gene therapy clinical trials were under way in the United States but only a handful of these are in advanced stages. SCID, in which children lack natural defences against infection and can only survive in isolated environments, remains the only disease cured by gene therapy.

Key Ideas:

• Gene therapy is most successful with diseases caused by single gene defects.
• The first gene therapy on humans that was successful happened in 1990 in the National Institutes of Health
• The patient that received this treatment was a four-year-old child who had adenosine deaminase (ADA) deficiency, which is a genetic disease where children are born with critical immunodeficiency and are likely to have serious infections repeatedly.
• Gene therapy has been trying to treat diseases such as immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease and Gaucher's disease since 1990.
• SCID is a disease where children lack natural defences against infection and can only live in lonely environments, which means that the only way it can be cured is by doing gene therapy.

Sources in MLA:

• 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 [Accessed 7 Oct. 2014].

Paraphrasing:

  Gene therapy is most successful with diseases caused by single gene defects. The first gene therapy on humans that was successful happened in 1990 in the National Institutes of Health. The patient that received this treatment was a four-year-old child who had adenosine deaminase (ADA) deficiency, which is a genetic disease where children are born with critical immunodeficiency and are likely to have serous infections repeatedly. Gene therapy has been trying to treat diseases such as immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease and Gaucher's disease since 1990. SCID is a disease where children lack natural defences against infections and can only live in lonely environments, which means that the only way it can be cured is by doing gene therapy.

What is Gene Therapy?

a.     In brief describe about this application (what is it all about?)

•      "Gene therapy is an experimental process that uses genes to treat or prevent disease."
•      "Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one."
•      "It is an experimental treatment that is still being developed, but it has the potential to restructure treatment for all kinds of genetic conditions." 

b.     What are some benefits of this application?

•      There's a chance of life for people who were born with a genetic disease or is developing cancer.
•      It can be effective when it's delivered to the tissue with the correct procedure.
•      You can avoid the side effects from the drugs used in the process.
•      It fixes the problem; where it is happening.
•      It has the possibility of preventing or even eliminating hereditary diseases.

c.     What are some future improvements which still need to be done to make this application perfect (limitations)?

•      To find a gene that's not functioning right, they need  to see it through genetic screening, and genetic screening created a lot of disagreements and it raised many ethical and legal problems.
•      "If prenatal tests are performed that these could lead to an increase in the number of abortions."
•      Many young couples want to see if their baby has genetic diseases. When they're checking with genetic screening, they will find out the flaws of their babies, which can lead to abortion.
•     It is quite difficult to deliver the genes to the tissue or system.
•     It can be expensive because the process might take time and it's difficult to do.
•     Scientists are unsure of the long term effects of gene therapy.

d.     What issues (social, economic, ethical, political or cultural) are currently related to the use of this application worldwide?

•      Ethical, social and economic issues are currently related to the use of the application of gene therapy.

Bibliography:

- Better Health Channel, (2013). Gene therapy - Better Health Channel. [online] Available at: http://www.betterhealth.vic.gov.au/bhcv2/bhcarticles.nsf/pages/Gene_therapy

- 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

- Genetics Home Reference, (2014). What is gene therapy?. [online] Available at: http://ghr.nlm.nih.gov/handbook/therapy/genetherapy

- ScienceDaily, (2014). Gene therapy. [online] Available at: http://www.sciencedaily.com/articles/g/gene_therapy.htm

- Ndsu.edu, (1996). Gene Therapy. [online] Available at: http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/brandi.htm

- Genetherapyinfo.webs.com, (n.d.). Gene Therapy - Advantages vs Disadvantages. [online] Available at: http://genetherapyinfo.webs.com/advantages-vs-disadvantages

 Figure 1: This video explains how gene therapy works in a simple way. Source: https://www.youtube.com/watch?v=bLI1Gfb0ynw

     In Figure 1, this video is talking about the process of gene therapy. It's talking about how the doctors and scientists are basically "hacking" your genes. It also talked about the first human to ever have gene therapy and how gene therapy have been helping kids. According to this video, the man said that gene therapy have also been used to treat dogs who has Type 1 Diabetes. How this works is that the scientists injects two items to the dog's muscles. Then it appears that the gene therapy works; they're still able to live after.