8/13/2020
VECR Final Project
During VECR, one of my favorite activities was the week when Alicia Morales spoke. She taught us about impostor syndrome, which I felt was really valuable, especially as I look to work in this field someday. Impostor syndrome, as she explained, is when a person feels less experienced and qualified than the facts present, causing self-doubt and anxiety. Her talk, about impostor syndrome, and what it was, as well as the resources she provided for testing our level on the spectrum of impostor syndrome, really helped me to identify with some of the things that I felt, and I really valued that talk. In addition to talking about impostor syndrome, another thing that she talked about was the basics of the immune system. She taught me more about the varied functions of the immune system, as well as helped me to conceptualize the way that CAR T-Cells work, which was the main focus of the VECR program. Another really interesting day in VECR was the day that we discussed bioethics. I found this especially enlightening because, not only did it give me a chance to see other perspectives and rationalizations, the discussion was framed around the current COVID-19 pandemic. The discussions, and examples provided, gave me a lens into the actual decisions that real people are making, and showed me the conviction needed to really do that kind of job.
Slides from Alicia Morales' presentation on modified T-Cells and the immune system
One very interesting study that I was exposed to during the course of VECR was about using CRISPR-Cas9 to genetically edit a small subset of stem cells, with the goal of alleviating the clinical symptoms of hemoglobinopathies, like sickle cell anemia, and thalassemia. The way that this treatment works is by introducing a naturally found mutation that increases the amount of fetal hemoglobin (HbF) produced as an adult. This type of hemoglobin is used to substitute for damaged and defective adult hemoglobin, which is the cause of sickle cell anemia and thalassemia. The mutation responsible for producing extra hemoglobin, HbF, is introduced to a small amount of hematopoietic stem cells, or stem cells responsible for the creation of blood, using CRISPR-Cas9. CRISPR can selectively remove and change parts of DNA, and so, by using CRISPR to introduce the mutation, and then reintroducing the modified stem cells to the body, properly functioning hemoglobin can be restored in larger amounts to the body, reducing the effects of hemoglobinopathies. One of the reasons that this study is so important, is because of the decreased cost, and increased efficiency of the treatment. The only previously viable treatment for diseases like sickle cell anemia was an allogenic transplant, which is limited by complications, and the availability of donors. However, this treatment method used the patient’s own cells, combined with a very focused and targeted edit, to provide lasting results. Even though methods that edit stem cells are currently being tested, this specific method narrows down the edit to a more specific subset of stem cells, drastically decreasing the amount of cells that are edited, and allowing for greater production of these cells. Since this study was conducted on rhesus macaques, and seemed to pose no long-term health risks, it looks like an extremely positive advancement that could be used to help many people. This study was conducted by the lab of Dr. Hans-Peter Kiem, who heads the Stem Cell and Gene Therapy Program at Fred Hutch, as well as being a professor at the University of Washington, and an attending physician for SCCA. This study really interested me because of its potential as a significantly safer and cheaper alternative, as well as its use of CRISPR-Cas9, which I find very interesting of its own right.
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