Cas9 Therapeutics: A Genetic Analysis

CRISPR is a new gene-modifying tool that has the potential to treat numerous medical conditions by editing genes that are responsible for certain diseases. This technology is based on the ability of bacteria to destroy the DNA of invading viruses. Studies have suggested that this new technology can be applied to human cells, although the idea of chopping up regions of the human genome can be unethical and could even be harmful. In order for the treatment to be administered to a patient, a small piece of RNA and an enzyme that makes a cut in the DNA are delivered to the cells. A biotechnology company, known as Editas Medicine, located in Cambridge, MA, is already designing treatments for conditions of the blood and the eye using CRISPR. For

magine, 20 years from now, sitting in a cold doctor's office deciding the genes of your unborn baby, what color hair, eyes, speed of metabolism, height would you even know what to pick? Impossible you might say but in this day and age technology is growing ever so rapidly that picking the genetic makeup of your baby is closer than you might think. The technology is called CRISPR. This technology doesn't only have the ability to change physical traits, but genetic traits specifically genetic abnormalities and diseases. 20 years ago, no one would have ever thought we would have the answer to, in theory, cure every genetic disease from sickle cell anemia to cystic fibrosis. However, with great scientific breakthroughs comes questioning and

Genome editing is a huge leap forward in science and medicine. Because of recent advances in technology, the study of genes and induced ‘point’ mutations have led to the discovery and advancement of methods previously used in order to mutate genes. The development of Clusters of Regularly Interspaced Short Palindromic Repeats (CRISPRs) and CRISPR associated system 9 protein (Cas9) technology is a hugely significant leap forward as this is a tool that could potentially be used for the research into and hopefully the treatment of a range of medical conditions that are genetically related. Cystic fibrosis (Schwank, G. et al, 2013), haemophilia and sickle cell disease are an example of some of the conditions that have the

With modern technology comes the breakthrough of the decade by altering the human genes. This altering gene invention is called CRISPR/Cas9. However, this invention in the beginning stages of altering genes, began with rats until perfection. The process began early with the embryo stages to edit the genes. With the introduction of CRISPR surrounds a lot of controversy. Some people believe editing genes is playing with the hands of God and refuse to believe in CRISPR. With the article, “Let’s Hit Pause Before Altering Humankind”, by David Baltimore believes CRISPR is a tool with no good intentions. With this information the article should not be published with being against CRISPR.

CRISPR has been garnishing a lot of media attention recently and it is not just popular among the scientific community but also the general public. Several online news outlets and scientific journals have been talking about the significance CRISPR-Cas could have for the field of genetics and science as a whole. I even came across a Youtube video from The Verge, a tech channel that normally does reviews on new smartphones and laptops talking about CRISPR [15]. So why is CRISPR gaining so much attention both from the scientific community and the general public? The answer lies in the potential this technology possesses.

CRISPR stands for Clustered Regularly-Interspaced Short Palindromic Repeats, so it’s simple to see why it was abbreviated. Whilst the topic of genetic modification with humans has been relatively untouched, and in many cases unheard of, in the general public, when we learnt about genetic modification, we learnt more about the way that it works rather than how far we can go with this new technology. What can we do with this, how much can we modify our own genes and to what extent can we do so?

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeat, referring to the repeating DNA sequences found in the genomes of microorganisms. CRISPR technology allows scientists to make precise changes in genes by splicing and replacing these DNA sequences with new ones. Through these changes, the biology of the cell is altered and possibly affects the health of an organism. The possibilities are endless as this offers opportunities in curing deadly diseases, modifying genes, and changing humanity as we know it. Although bioengineering has been around since the 1960s, CRISPR is significant because of the comparative low costs and the ease of the procedure to

CRISPR Cas-9 is a system that changes genes and shows a further promise for treatment for Duchenne Muscular Dystrophy (DMD). Doing this will hopefully avoid ethical dilemmas. DMD effects nearly 1 in 5,600-7,700 people between the ages of 5 and 24 for males in the U. S. CRISPR stands for “Clustered Regularly Interspaced Short Palindromic Repeats”. These are DNA segments found in microorganisms that repeat itself in open spaces. CRISPR attacks any virus that comes into your body and cuts the strand of virus on the DNA out. Researchers have tried to cure DMD in mice by getting therapy through the back of the eye, one of the ways CRISPR therapy works, which seems to work best.

Genome editing, or gene editing, is a type of genetic engineering in which DNA is inserted, deleted or replaced in the genome of a living organism using engineering ucleasus, or “molecular scissors” in the layman's terms. The technology at head of this procedure is the CRISPR-Cas9,the most versatile and precise method of gene editing and also is what made gene editing the hot topic it is in the medical world. As a result, of this breakthrough medical scientists have the ability to cure sickness, by eliminating genes that cause disease doctors can treat a wide range of illness

A gene editing method called CRISPR, Science’s 2015 Breakthrough of the Year, is an easy and inexpensive way to cause the “DNA-cutting enzyme” to cut out specific parts of the DNA strand, therefore changing the genetic composition of an organism. Another way to use the CRISPR is to use it to deliver regulatory factors to successfully turn genes on or off and any level in between, which can help save lives by turning off deadly genes. CRISPR is currently being used for biomedical purposes, such as repairing genetic defects or creating tissue-based treatments. This genetic editing method could be used and is being used to change the very animals and plants that we know today by creating allergen-free peanuts or virus resisting pigs.

CRISPR-cas9 allows researchers to edit part of the genome by inserting, deleting, or altering sections of the DNA sequence. The CRISPR-cas9 system consists of two key molecules: Cas9 and single-guide RNA (sgRNA). The enzyme Cas9 is ‘guided’ by sgRNA to the DNA sequences where it cuts the two strands of DNA at the specific location in the genome. Endogenous DNA repair mechanisms try to repair the breaks in both strands which then leads to indels that can cause mutation in the target gene. This process is shown in Figure 1.1. To determine whether CRISPR–Cas9 can be used to understand gene function in human preimplantation development, the POU5F1 gene that encodes the development regulator OCT4 was selected as the target. It was predicted that

CRISPR/Cas9 is a type of treatment that is common during gene therapy. This kind of therapy engages an enzyme complex called CRISPR/Cas9. It is found in bacteria. It works together with Ca9, and it is an enzyme that cuts DNA, so pretty much this is like scissors that can cut DNA. How does CRISPR/cas9 work? Well, In the beginning, CRISPR/Cas9 complex looks around the cell 's DNA till it spots and connects to a sequence that correlates to the CRISPR. This was all said by Biochemist John Reidhaar-Olson. So in other terms, the Enzyme will scan the DNA in the cell until it finds its match, Then it will attach itself to that sequence of the DNA. A university in Japan injected CRISPR/Cas9 into human embryos to fix a fatal blood disease called Thalassaemia. They got the Embryos from a local clinic. None of the embryos could be born because they were fertilized with two sperms. In the end, the Embryos were found not to have the fatal blood diseases anymore. Thus showing how useful CRISPR/Cas9 is when doing gene therapy. (Tanya Lewis)

Currently, one of the most exciting applications for CRISPR/Cas9 systems is that it has the potential to cure hereditary diseases. A significant amount of current medical research is heading in that direction with hopes that scientists can develop a

The last few decade, technology has made so much progress that today humans are allowed to do things that never even crossed their mind, modifying the plants and animals and all that progress is for the benefit of humans. Technology has made a great impact in business, communication, education, healthcare, etc. Today in the healthcare, medical research has become extremely advanced, and every disease seemingly has a cure or at least a prevention; it has advanced with the discovery of many drugs and medications that have helped prevent many deadly diseases and disorders at also has advanced with a medical technique that can help cure many diseases. A revolutionary genome-editing tool known as CRISPR- Cas9 is based on something that bacteria do naturally. About a decade ago, scientists realized that bacterial cells possess a defense system that recognizes and chops up the DNA of invading viruses. This revolutionary genome tool could be able to eliminate cancer, malaria, cure blindness, edit human, etc. According to crazy-things-we-can-do-with-crispr-cas9 published by Jay Bennett in Jan 26, 2016 people are already using the tool to make changes to organisms that could revolutionize agriculture, lead to new treatments for diseases with no cure, or even modify human embryos.

Crispr is short for Clustered Regularly Interspaced Short Palindromic Repeats. It has been utilized to remove and modify various harmful genes found in multiple organisms. According to the United States Library of Medicine and National Institutes of Health, This has been beneficial to science because it allows a closer look into the key roles gene manipulation plays in examining reproductive process of mice and other species (Young, Aitken & Ikawa, 2015). It has been seen as a viable source for preventing and possibly curing diseases including Cystic Fibrosis via genome editing. The Cystic Fibrosis Foundation classifies this detrimental disease as