Chapter #9 : CRISPR, Should we use it to edit embryos genome ?

Chapter #9 : CRISPR, Should we use it to edit embryos genome ?

https://soundcloud.com/zoz19-202289416/podcast-presentation

CRISPR is a powerful genome editing  tool that can participate in transforming the field of gene therapy to prevent millions of diseases. CRISPR stands for clustered interspaced short palindromic repeats, which is a specific patterns of DNA sequences, that can be cut by the aid of another enzyme called Caspase 9 to create new genome. How scientists discovered CRISPR? How CRISPR work to prevent diseases? Is it safe to be used on embryos? What are the ethical issues surrounding this technique? Are there any other useful applications for CRISPR? 

All of these questions are answered on the audio link above.

Recources:

https://www.ndsu.edu/pubweb/~mcclean/plsc431/students99/wharam.htm

https://www.musc.edu/humanvalues/pdf/gene-therapy.pdf

http://ghr.nlm.nih.gov/handbook/therapy/ethics

http://thirdspacemedical.com/services/internal-anti-aging/restorative-medicine/diagnostics-and-testing/genetic-fit-test/

Chapter 8: Final thoughts and personal perspective

Chapter 8: Final thoughts and personal perspective

            While doing my research about gene therapy, I realized that the benefits of this technology can be very huge in preventing countless number of diseases. However, ethical issues that arises from gene therapy and the fear of using this technology for genetic enhancement  participated somehow in abandon the use of it. Personally, I think that the benefits of using gene therapy can in some cases outweigh the ethical concerns.  Furthermore, I believe that strict government regulation of the use of gene therapy can solve the fears of using it in the wrong way. For these reasons, I think that we should encourage the use of gene therapy and continue on discovering how to treat more diseases using gene therapy approach.  

         

   Finally, I would like to thank you all for the time you spend reading my blog. I hope I provided you with all information you would like to know about gene therapy.  

Image resource:

https://choward2614.wordpress.com/2013/07/18/a-very-public-thank-you-to-robin-tidwell/

Chapter #7 : Gene therapy has introduced a new way to treat cancer.

Chapter #7 : Gene therapy has introduced a new way to treat cancer.

            When someone have viral infection, the immune system can work very hard to kill the infection. Unfortunately, the immune system can't recognize cancer cells, which makes it ineffective to defeat tumors. Steve Rosenberg from National Cancer Institute was the first scientist to use gene therapy approach to treat patients with cancer. He successfully manipulated the human immune cells to give it the ability to recognize and kill cancer tumors. The author of the article, " Gene Therapy May Bring Cancer Killing-Cells" explains that Dr. Rosenberg was able to transfer a genetically modified T-cells to patients with cancer and succeed in treating  several patients with melanoma. The author mentioned that Dr. Rosenberg explained that cancer cells have their own way to evade the immune response, and by altering T-cells using gene therapy, we can make those cells able to recognize and kill tumors. The only problem that the article discussed regarding using this gene therapy approach is the high costs of this kind of research.

            Personally, I think that the author poorly explained how exactly the T-cells were modified to make it able to recognize the tumor. He also did not mention if this gene therapy performed in T-cells will be passed through offspring or not. If I have cancer, and I am going to pay for gene therapy treatment, I really want my second generation to benefit from this treatment. Rather than sensationalizing people who have cancer  and the public to be more interested in the research, the author could have done a better job giving accurate data on number successful and frailer cases in the study. 

Resources:

http://www.npr.org/templates/story/story.php?storyId=5744500

http://images.1233.tw/somatic-gene-therapy/ 

Chapter # 6: Gene Therapy FAQ

Chapter #6:

Gene Therapy FAQ

1.      What is gene therapy?

Gene Therapy is a way to treat diseases by correcting the faulty gene. A new functional gene is replaced by the faulty or non-functional one.

2.      How many types of gene therapy are there?

There are two types of gene therapy, somatic cell gene therapy, where a normal gene introduced to the body cells like skin, blood, and bone marrow. There is also germline gene therapy, where the new functional gene is introduced to germ cells (egg and sperms).

3.      How does gene therapy work?

Scientists use viruses as the delivery vectors to carry on the desired gene and deliver it to the targeted cell.The DNA for the desired gene is inserted into the genetic material of the virus and then the virus itself is inserted into the human cell. .

4.      Can the virus that is used to carry the corrected gene harm my body?

The viruses that are used to carry the desired genes are engineered so they cannot reproduce inside our body, which minimize any bad effects.

5.      Is there any successful cases of gene therapy?  

There are many successful cases in gene therapy including Heredity blindness, Hemophilia, Fat Metabolism disorder, Cancer, Parkinson’s disease.

6.      Is gene therapy available to treat my disorder?

Gene therapy is still in clinical trials, the FDA has not approved to perform gene therapy in humans yet.

Resources: 

http://learn.genetics.utah.edu/content/genetherapy/gtsuccess/

http://ghr.nlm.nih.gov/handbook/therapy

http://www.geogene.com/gene-therapy.html

http://learn.genetics.utah.edu/content/genetherapy/gtchallenges/

http://discovermagazine.com/2009/sep/02-second-coming-of-gene-therapy

https://www.ndsu.edu/pubweb/~mcclean/plsc431/students/bergeson.htm

http://blog.supercoder.com/icd-10-2/cms-offers-assistance-in-transitioning-to-icd-10/

Chapter Five: Arguments For and Against Gene Therapy

Chapter Five: Arguments For and Against Gene Therapy

            While some people think that gene therapy is a great discovery that will cure many diseases, others think it is risky with unknown long terms effects and we should avoid it to avoid any bad consequences. The public debate around the use of gene therapy begin when clinical trials of gene therapy started long time ago. Since gene therapy deals with changing the genetic makeup of the body, there are many ethical and social issues surround the use of gene therapy. 

            In one hand there are many people in the public who holds thoughts supporting the use of gene therapy. One of the strongest public arguments for gene therapy is that it will end suffering from many diseases by finding treatments for these disease. For instance, when a genetic disease is inherited within a family, the family individuals can undergo genetic screening to know if they carry the faulty gene for that disease.  If they do so, they can undergo gene therapy and cure that disease without suffering. Even if this looks a great opportunity to end suffering from certain disease, there are some ethical issues surrounding genetic testing and gene screening. Bioethics think that a parent who test the genetic composition of their fetus might want to abort that child if he tests positive for certain disease. This will increase the rate of apportion. Also, other bioethics think that the high costs of gene therapy may makes it available only for the rich people to use. Another argument for gene therapy, is that it targets the cause of the disease not the symptoms, which makes it the best among other general practices. 

            On the other hands, Some people argue against the use of gene therapy. Since gene therapy deals with changing the genetic makeup of the body, there are many people think that using gene therapy scientists are trying to "play God" by manipulating the genetic composition of the human being. They think that even though it can produce good effects like eliminating diseases, it can also produce devastating effects because we do not know the complexity of the human being enough to play with it and manipulate it. Another argument against gene therapy is the fear that some might use it to enhance physical traits like beauty and intelligence, which will give some people more life opportunities than others. 

Recourses: 

https://www.ndsu.edu/pubweb/~mcclean/plsc431/students99/wharam.htm

https://www.musc.edu/humanvalues/pdf/gene-therapy.pdf

http://ghr.nlm.nih.gov/handbook/therapy/ethics

http://thirdspacemedical.com/services/internal-anti-aging/restorative-medicine/diagnostics-and-testing/genetic-fit-test/

Chapter Four: Gene Therapy Regulations

            When we talk about changing the biological make up of an individual, that must be something very dangerous. Our genetic make up makes us what we are, so changing this genetic composition should be highly regulated to avoid the long term risks. Ross Laframboise in his article "Gene Therapy Regulations: A Proactive Approach", explains the importance of regulating gene therapy researches in order to minimize the risks. He mentions that there are two main organizations now regulating gene therapy researches including US Food and Drug Administration (FDA) which makes sure of the safety during researches, and  Recombinant DNA Advisory Committee (RAC) that is responsible of reviewing gene therapy protocols. Besides these two organizations, the author expresses the importance of the government regulation especially for germline gene therapy. Since germline gene therapy can affect the future generations, we need to take cautions on the specific genetic composition that we are going to change. The author also mentions that some organizations should be responsible of adopting the ethical issues regarding gene therapy to prevent any social problems. 

 

References: 

https://www.ndsu.edu/pubweb/~mcclean/plsc431/students/ross.htm

https://bigkingken.wordpress.com/2011/07/20/pain-relief-from-herpes-virus-not-a-typo/

Chapter Three: A Cure for Colorblindness Using Gene Therapy Approach

A Cure for Colorblindness Using Gene Therapy Approach.

​Using gene therapy approach, scientists at the university of Washington was successfully able to cure colorblindness in monkeys. The article "A cure for 'colorblindness' may be in sight" from the CNN news, explains that Maureen Neitz and Jay Neitz found that monkeys who suffer from color blindness are unable to differentiate between green and red color. Lookinginto their genes, they found that one of the genes that are responsible for encoding these two colors was missed. In fact, this what makes those monkey see things that are green or red relatively similar. In order to cure the monkeys, scientists replaced a new gene to the retina using a weak virus to deliver the gene. Finally, they test the monkeys and announced that their therapy was successful. Although this gene therapy was not tested in humans and the FDA did not give their permission for that, scientists gave those people suffering from colorblindness a hope for cure.  

​Even though the story adequately covers the most important steps in using gene therapy approach to treat diseases including how to find the faulty gene, how to deliver the corrected gene, and finally testing if the therapy actually worked, the story did not adequately covers the long term effectsof this gene therapy. One of the disadvantages of gene therapy is that the long term effects are not known. Therefore, scientists have to study the long term effects of their therapy before saying it worked on monkeys or is going to be working in humans. Another important point that the story did not mention is if this corrected gene will be passed to the second generation. If I am a colorblind person, I need to know if this therapy will affect my offspring or not before I decide to undergo this gene therapy. 

References:

https://www.youtube.com/watch?t=352&v=EgPMc90uXlU

http://www.pri.org/stories/2015-09-20/cure-colorblindness-may-be-sight

http://genetherapyinfo.webs.com/advantages-vs-disadvantages

Chapter 2 : Comparison of the Two Types of Gene Therapy

Chapter 2 : Comparison of the Two Types of Gene Therapy

There are two types of the gene therapy; germline gene therapy, somatic gene thrapy.   In germline gene therapy, the scientists try to treat diseases by inserting a normal gene instead of the faulty one only in germ cells, which are the sperms and eggs. In fact, the modifications of germline genes can be passed through generations, which is very good. Why? Because modifying the abnormal genes for certain diseases  in one generation, will be passed to the second generation and we will be eliminating the disease from occurring and no one will suffer from this disease any more. Even though this is a good opportunity to eliminate diseases entirely from one family, this type of therapy is not being attempted because there are some concerns of the risks that germline gene therapy can make such as altering another normal gene and causing new defect. Another consideration regarding this type of gene therapy is that few people may use this therapy for genetic enhancement such as choosing eye color for their next generation or increasing beauty and intelligence, which will give them more opportunities than others and may raise moral issues.  

            In the second type of gene therapy, somatic cell, scientists try to modify a gene that is in the body cells such as introducing normal genes to skin cells, bone marrow cell, blood cells, and a lot more. Unlike germline gene therapy, this type cannot be passed through generations and it will only affect the individual who is being treated. Even though there are many obstacles facing the scientists in the field, like how to deliver certain gene to the target cell. There are many clinical trials in their late phases which promise that somatic cell  gene therapy approach will be an effective treatment to many human diseases in future.  

 References:

http://www.nejm.org/doi/full/10.1056/NEJM199310143291607

http://www.news-medical.net/health/Gene-Therapy-Types.aspx

Chapter 1: Introduction

Chapter 1 : Introduction To The Gene and Gene Therapy

What is a gene ?

            Genes basically are small molecules that carry our genetic information. Genes consist of many nucleotides that make up our DNA, and are normally found in thread like structure called Chromosomes. Those chromosomes are usually within the nucleus of each human cell. When the cell divide and produce new daughter cells, a complete set of chromosomes can be found in each daughter cell. Inside each cell of our body, there are two copies of each gene; one from each parent. The main function of our genes is that it gives the instructions of  synthesizing proteins and enzymes, which are important for the body functions and metabolisms. Sometimes, abnormal genes arises from mutations, which are alteration of the nucleotide sequence. As a result, the gene produce missing or un-functional proteins that may cause genetic disorders.

What is Gene Therapy ?

            Gene Therapy can correct the genetic defects that mutations may resulted by targeting the abnormal gene and inserting new one to the cell. If mutations in certain gene resulted in abnormal or missing proteins, then the protein will not be able to function normally.  Gene therapy can introduce a normal copy of the gene to make the protein functional. The first successful gene therapy was done by William French Anderson in 1990 at the National Institutes of Health (NIH). He was successfully able to treat a girl who was suffering from Severe Combined Immunodeficiency disease that is known of producing impaired immune function.  Nowadays, there are many patients receiving gene therapy correcting their faulty proteins and making them function normally. However,  gene therapy is a very challenging task.  For instance, scientists have to find ways to transfer a foreign gene to the human call, make the gene expressed, and finally avoid the immune response to this foreign molecule.

             

References :

http://ghr.nlm.nih.gov/handbook/therapy

Pierce, B. A. (2012). Genetics a conceptual approach. New York, NY: Kate Ahr Parker.