SCIDS and Gene Therapy

11 Comments

1. Jada G. says:

   March 29, 2014 at 8:32 pm

   This article is referring to the bubble boy disease (SCID). My only experiences to this is watching Bubble Boy when I was a little younger but didn’t fully understand what it was. I feel bad about testing on little kids with gene therapy and making it worse on them. “Four young patients were diagnosed with leukaemia after two years after receiving a similar treatment.” (1). I see that continuing studies and testing newer methods have kept kids and adults alive after using forms of gene therapy. As stated in the article Immune System Kills Cancer by Kate Yandell “gene therapy has rid three adult patients with acute leukemia.” In my opinion, “doing something that’s going to advance medical practice and maybe help other children” (2) was the right route to take even though it was not necessarily safe. The family of Nina decided to go along with the therapy hoping of her cure. “We had hoped, but couldn’t have predicted that the response would be so profound and rapid,” Renier Brentjens, lead author of the paper and an oncologist at Memorial Sloan-Kettering Cancer Center is an inspirational quote from the article Immune System Kills Cancer because it gives hope to everyone dealing with some form of this disease.
   Other Article: http://www.the-scientist.com/?articles.view/articleNo/34857/title/Immune-System-Kills-Cancer/

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2. Brian Broecker says:

   April 6, 2014 at 11:43 am

   Gene Therapy
   After reading the article “Bubble Kid’ success puts gene therapy back on track”, I have reflected on what it means to be born healthy. After reading about how Nina suffered an immune system genetic defect, I thought about how lucky I am to not have the choice, live in a bubble for the rest of my life or die at a young age. I do not have any family or personal experience on this topic, so I decided to read an article about the pros and cons of gene therapy. In the article I chose, “Aspects of Gene Therapy” http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/eric.htm,
   they listed the pros and cons of gene therapy. I have agreed and disagreed with the article about how gene therapy will be used in the future. I believe that gene therapy should be used to save the lives of others. In the articles it discussed how in the future, other countries might exploit this opportunity by creating or allowing a “super” army of enhanced humans. I do not believe any country would put their citizens through this type of testing, experimenting and finally the growth of a new and invincible army. I believe if this process was developed sooner and at a cost effective price, there might be a cure for all harmful disease such as cancer and AIDS as well as gene defects. I think that gene therapy is useful in fields of biology and medicine. This new process has saved the lives of countless people, including five-week-old baby Nina.

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3. Sawyer Scheitler says:

   April 6, 2014 at 9:55 pm

   This article was about gene therapy and the development of gene therapy as well as the success and failures of gene therapy in the past. I personally do not know anybody who has any disease requiring gene therapy but I have done multiple projects on stem cell transplants as well as leukemia. I do know how awful and severe it is that in the past, gene therapy has caused leukemia. Leukemia is a life threatening disease that affects people of all ages. I was so relieved to read that the problem has been fixed and that getting leukemia is no longer a concern when it comes to gene therapy. I did read another article from http://www.medpagetoday.com/MeetingCoverage/ASHHematology/43340 which talks specifically about how gene therapy has gotten safer and how leukemia is no longer an issue with gene therapy. I thought this article was very informative and I did not know a lot of the things I read, especially of SCID disease and retrovirus. I know that we will keep improving our research and, in the future, will have even more success with gene therapy.

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4. Colby Koch says:

   April 6, 2014 at 10:05 pm

   I have never experienced this disease or met anyone with it. I found this article interesting though so I found an article that further explains this disease. In this article it explains the 4 major parts of immunodeficiency. http://pedsinreview.aappublications.org/content/14/6/226.abstract

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5. Jackson Williams says:

   April 7, 2014 at 7:16 am

   I have never known anybody that has had this disease. It seemed peculiar that they failed with this so many times. I liked the timeline in the article and how it showed some failures nut also showed the successes. http://www.scid.net/

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6. Zach Messaglia says:

   April 7, 2014 at 1:02 pm

   This article is similar to the one I emailed you earlier this week not because they are the same disease but because they are both rare. Today in our society we are donating to the major disease charities not the diseases and genetic defects no one knows about. In the article I learned about people who have these rare disease and the actions they take to raise awareness about their disease in the scientific community for example the people who have fibrodysplasia ossificans progressiva (FOP) raised money in order to hold a conference with the scientific community to share their scientists findings on the disease. Also not only does curing these rare diseases help those who have it but also those who suffer from other diseases. For example since the scientists who are researching FOP discovered the gene that causes the people who have FOP to keep growing could activate this gene in people who have austeo perosis to cause them to gain bone mass. In your article it covers how they now use gene therapy to cure this disease and the scientists who study FOP also are on the track to use this same method.
   Zimmer, Carl. “The Girl Who Turned to Bone.” Atlantic. The Atlantic, 22 May 2013. Web. 7 April 2014.

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7. Zach Messaglia says:

   April 7, 2014 at 1:07 pm

   (continuing from my previous comment)
   Like Colby I have had no experience nor have I had either disease. I have though felt like a friend to the people interviewed in my article thanks to the author.

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   • Zach Messaglia says:

     April 7, 2014 at 1:37 pm

     Here is my article link
     http://www.theatlantic.com/magazine/archive/2013/06/the-mystery-of-the-second-skeleton/309305/

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8. Charlie Rohen says:

   April 7, 2014 at 9:28 pm

   I have no personal experience with this disease nor have I ever met anyone who has it. It would be very lonely and embarrassing to live in a bubble for 12 years and never be able to get out of the plastic case around me. I think it would be terrible to live inside a case and know that I was going to die at a very young age because of my condition. This article gave me more information on the bubble boy case and helped me see how sad it truly was to be David Vetter. I think that gene therapy could become a promising cure in the future, but like almost all of science, there has to be failure before there is success.

   http://www.cbsnews.com/pictures/bubble-boy-40-years-later-look-back-at-heartbreaking-case/

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9. Chris Rodriguez says:

   April 8, 2014 at 7:21 pm

   Blindness is also gene mutation, and occurs in the X chromosome. In this web article: http://abcnews.go.com/Health/man-saved-blindness-gene-therapy/story?id=21578118, a man named Nick Tuftnell goes through gene therapy to have hopefully restored his sight. At the University of Oxford, six people participated in the study, and each went through gene therapy. This gene therapy was to treat the deterioration of the ocular cells. They injected working genes into the eye, so the genes could start to repair the cells by patching the missing genetic information. Through this, they found that the best way to fix the cells is to place the missing genes in before they lose their visual acuity.
   Similarly, in Nina’s story the researchers used lentiviruses to do treat the Bubble Boy disease. This disease is caused by the IL2RG gene. Apparently only males with this condition inherit this gene. They are not able to fight off infections. The lentiviruses are used as a place holder to temporarily fix the DNA. These genes could be modified into the correct sequences and this lead to improvements in health. The gene was corrected when researchers removed the CD34+ cell which is a stem cell in the bone marrow. Years later, the patients still had healthy cells with some developing side effects. For more information, visit this web article: http://health.usnews.com/health-news/family-health/cancer/articles/2010/07/21/gene-therapy-shows-promise-with-bubble-boy-disease.

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10. Ryan Osman says:

    April 14, 2014 at 3:30 pm

    SCID and Gene Therapy

    SCID was the first disease to be treated with gene therapy in over 20 years. SCID is caused by a faulty gene adenosine deaminase, which dispatches a toxic molecule from white blood cells, which builds up and kills cells that fight infections. Gene therapy is when you insert a working gene into a person with the faulty version, and the product of the two should overcome the deficit. Usually scientists use viruses, because they are very durable, survive, and insert their genes into the host’s genome, but they are still dangerous because they can activate nearby genes and trigger cancer. An alternative for this is lentiviruses, which are much more safe than using viruses.
    Overall, I think that gene therapy can be very useful. It has a lot of potential, and can be used for many more purposes. “It’s a principle that could be applied to other diseases where you want a protein or enzyme to be released into the blood” said Maria Limberis, at the University of Pennsylvania. This means that there is a lot of potential, and can help many more people with different diseases. Also it has more refined, has much better results, and has helped many people already. “All of the hard work has come to a point where gene therapy could become a more routine modality of medicine” said Inder Verma, at the Salk Institute. Last, another way to make sure there is a lower morbidity rate, is to use chemotherapy to help after using gene therapy. “In ADA-SCID, there is not such a powerful survival advantage, and so we think that using a small amount of [chemotherapy] will allow us to engraft a larger number of stem cells to allow lymphoid development to occur” said Dr. Gaspar from University College London.

    Outside source: http://www.medscape.com/viewarticle/748672

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