Each day I step into AP Biology class, I learn so many bizarre things that are going on in the world and all around us. Change. I consistently see and hear about change and advancements. From designer babies to a small change in our DNA sequence, we are now able to do things to change our appearances or even find a cure to diseases just by the current biotechnological advancements.
Have you heard about gene therapy? Gene therapy is the introduction of a normal gene into a patient, usually to treat diseases caused by a single defective gene. Scientists are treating patients with diseases by adding a new allele in strands of RNA which are placed in a retroviral vector. The vector is then inserted in somatic cells, such as bone marrow cells, so the normal allele is inserted in chromosomes and is reproduced. One genetic disease gene therapy treats is severe combined immunodeficiency (SCID). SCID patients show a very weak immune system, which makes them susceptible to all kinds of illnesses. Since bone marrow reproduces throughout our entire lives, includes stem cells, and even produces our blood and immune system cells, bone marrow cells are a prime candidate to help SCID patients. Gene therapy has been used to treat Leber’s congenital amaurosis (LCA), which is a retinal degenerative disease that causes vision loss, a blood disorder, and even a degenerative disease of the nervous system.
One of the awesome things that AP Biology course equips the students are the several guest speakers that come in and share their insight about biology. Recently, Mana Anvar, an MS student at UCSF, shared her story working in UCSF’s Baraban Lab. There she studied pediatric epilepsy on zebrafish models in order to gain more understanding and research on Dravet Syndrome. One big connection that I learned in class that she mentioned is CRISPR-Cas9, which essentially edits a genetic sequence. In this lab, she was able to use CRISPR-Cas9 to edit or remove the gene that causes Dravet Syndome in zebrafish to observe seizure activity. She performed several tasks such as genotyping, sequencing, cannabinoid drug screening, and tracking. To experiment and understand how the drug could essentially help individuals with Dravet Syndrome, she analyzed and tracked the distance, movement, and velocity of the zebrafish treated with the drug. Anvar not only explained her work relating to biology research, but she also inspired me to pursue the subject because biology is interdisciplinary. Biology never fails to amaze me.
All of these advancements are pretty awesome to think and learn about, but of course, there are ethical concerns. We must think about the extent to which we use these technologies and gene editing. This is a great transition into our class conversation on bioethics! Keep that in mind and as the Amoeba Sisters say, stay curious ✌️.
Works Cited
McGovern Institute, director. Genome Editing with CRISPR-Cas9. YouTube, YouTube, 5 Nov. 2014, http://www.youtube.com/watch?v=2pp17E4E-O8&t=89s.
NationwideChildrens, director. SMA Type 1: How Gene Therapy Works. YouTube, YouTube, 7 Dec. 2017, http://www.youtube.com/watch?v=iBmyXr_o1hU.
“The Effects of Lighting on Zebrafish Development.” Powers Scientific, 6 Aug. 2015, powersscientific.com/effects-of-lighting-on-zebrafish/.
Arabella Krystienne's AP Biology Blog 