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By Boris Salinas CRISPR-Cas9, a gene editing tool with the ability to edit DNA and potentially shift the course of evolution. CRISPR stands for, Clustered Regularly Interspaced Short Palindromic Repeat. What does this mean? In every genome there exists a certain repeating pattern of DNA sequence which are also regularly interspaced, these are called REPEATS. In between REPEATS there exists a genome sequence called SPACERS, these SPACERS are not identical in any shape or form. So, what does this mean? And what does it do? The initial intention for the progression of CRISPR research was to innovate gene therapy and develop cures for genetic diseases. Unlike bacteria or viruses which medicine can treat directly or alleviate symptoms until the immune system kills all the remaining bacteria or virus. Genetic diseases are not identified as a threat by the immune system since it is a part of the blueprint by which you are built. To treat genetic disease, you would quite literally have to alter DNA and reprogram the whole organism. As you may imagine this is a very difficult thing to do. The creation of CRISPR-Cas9 was derived from a protein called Cas9. Which is so to say the “immune system” of a cell. Cas9 has the ability to memorize viral DNA and later alter the DNA of the bacteria in which it resides to create a resistance against the virus. It was later discovered by an American Biochemist, Jennifer Doudna, that this protein could be directed or programmed. This meant that with the use of the Cas9 protein, the possibility of editing genomes however is needed becomes a possibility. This will alter the course of evolution. CRISPR-Cas9 is an extremely powerful tool. Currently this technology is only about 18 years old. In November 2018, reports from China marked the first time in history that humans edited the genetic code of a future generation. These two subjects were Twin Girls who are now immune to HIV. In a sense, with this new technology we as a species exist in an era which could be considered the end of the beginning.
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By: Boris Salinas The U.S. Food and Drug Administration has finally approved a treatment that gives children with a rare genetic disease that causes premature aging more time to enjoy life. Hutchinson-Gilford progeria syndrome (progeria for short), a disease that causes teenagers to die due to heart attack, stroke, or heart failure. Most of these children are expected to die before achieving the age of 15. Zokinvy is the first and only approved treatment for progeria which was approved on November 20, 2020. In a clinical trial, which included 62 children receiving the drug, as well as 81 who would not take the drug. Zokinvy was proven to increase the life span by 3 months on average after the first three years of treatment. For those children who received Zokinvy for up to 11 years, their life spans were extended by about 2 years and 6 months. Progeria is caused by a mutation of the LMNA, or lamin A gene. Lamin A is the framework of the nucleus that holds the cell together. Researchers currently believe that the mutation of the lamin A gene causes the defective lamina A protein to destabilize the nucleus. This instability is the cause of premature aging in progeria patients. Children with progeria have an elevated amount of progerin, generated by a single nucleotide polymorphism. The protein is unable to pass through the cell membrane and cannot be recycled for new proteins, causing cells to age prematurely, which then causes stiffness in connective tissue and blood vessels. Everyone’s bodies make progerin, the amount is then elevated as a person begins to age. However, children with progeria create an abnormal amount of it. At birth, these children look like any other, symptoms appear later around the age of two. Over their lifetimes, these children will lose body hair and body fat, gain joint stiffness, cardiovascular disease, and many other symptoms that are associated with accelerated aging. Zokinvy was created by a company called Eiger Biopharmaceuticals from Palo Alto, California. This drug is meant to lower the amount of progerin that is produced, as well as lower the amount that is accumulated in cells. These oral pills do not fully block production, and the amount digested is limited due to side effects such as vomiting, diarrhea, infections, nausea, decreased appetite, fatigue, upper respiratory infection, abdominal pain, muscle and joint pain, and electrolyte abnormalities. With the approval of Zokinvy by FDA, researchers can now focus on other drugs and therapeutics that could further extend the lives of children that suffer from progeria. The possibility of using gene therapy to fix the mutation that causes the disease are also being investigated. |
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The opinions and views expressed in this blog are those of the authors and do not reflect the views, opinions or standpoints of Youth4Better. Any content published in this blog is not with the intent of harm towards any religion, ethnicity, race, organization, club, society, individual, or anyone or anything. |