Lab Report

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

From the science community perspective, the CRISPR-Cas system could reduce or even eliminate many of the difficulties researchers face when gene editing such as cost, duration and accuracy. Prior to CRISPR-Cas, gene editing was performed in “big labs” with experts

For many years biomedical researchers like myself have been trying to create more proactive ways to amend the genome for living cells. In more recent fieldwork studies there has been a new state of the art instrument based on bacterial CRISP in close works with protein 9 often referred to as CAS9 from the streptococcus progenies have possibly unlocked new data. The CRISP/CAS9 tries to manipulate the function of the gene using homologous recombination and RNA interference, but is set back because it can only provide short term restriction of the genes function and it’s iffy off- target effects.

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.

The author gives a brief history of past genome editing but thoroughly explains the history and mechanism of the CRISPR technology. She elaborates on how the technology has already been used to cure diseases and speculates on its future uses and regulation.

Duchenne Muscular Dystrophy (DMD) is a lethal genetic X-linked disease results from the mutation in the reading frame of the dystrophin protein, and it affects mostly boys in their muscle and cardiopulmonary function. Although there are no effective treatments to cure DMD patients right now, scientists consistently explore more methods to come up with the practical treatments. One of the most popular and valid approaches is a gene-editing therapeutic method – CRISPR/Cas9 Genome Editing. It adapts from the natural systems in bacteria, and it can generate targeted gene modifications to target specific DNA sequence. Then it introduces shifts within exons to restore the reading frame, so it can express a partial functional dystrophin protein.

Duchenne Muscular Dystrophy is a degenerative X-linked recessive disorder usually resulting in death in the late third decade. Mutation of Dystrophin gene at Xp21 disrupts the mRNA reading frame resulting in absent dystrophin protein in muscle cells. Currently no therapy can counteract the disease effectively. Exon skipping with oligonucleotide administration restores the reading frame of the mRNA to produce truncated but functional dystrophin and requires repeated administration which can cause drug accumulation toxicity. Mesoangioblast Stem Cell therapy has shown safety but limited efficacy due to problems of migration and engraftment in patient skeletal muscles. The approach of Cell Mediated Exon Skipping in this project will aim to draw from the strengths of previous strategies while reducing their individual limitations.

Every few years, advancements in technology alter the way scientists do their work. Recently CRISPR-Cas9, a RNA useful for working organisms in the animal kingdom has proven itself beneficial on a gene-editing platform. After performing many abortive attempts to manipulate gene function, including homologous recombination and RNA interference, scientists have finally had a breakthrough with CRISPR-Cas9.

Granted there have been other gene editing techniques used before, but by far CRISPR has been reported to have the most potential to revolutionize different areas where the method is applicable. The fields that researchers believe these modification resources will be the most beneficial, include but are not limited to medicine for curing and preventing diseases, creating socially ideal children, and perhaps aiding in the decrease of world hunger. While these goals do seem quite optimistical, scientists have high hopes for what CRISPR will be able to accomplish with time. Currently the system is just now being tested out on living organisms, with the ambition of figuring ways to genetically terminate diseases (2).

Genetic diseases and illnesses have been of much concern for many years, leaving many deceased or with a poor quality of life. Due to the implication of modern medicine and other techniques used for treatments, mortality rates have decreased and the average life expectancy has increased. Unfortunately, every individual responds differently to the type of treatment they need, which is why the implication of personalized medicine is forthcoming. A certain technique that has been distinguished and commended by researchers today is known as clustered regulatory interspaced short palindromic repeats, or CRISPR. CRISPR is associated with Cas9, and it is a popular genome editing technique which can be programmed to target specific areas of DNA and

CRISPR is versatile in that any target sequence can be modified by simply altering the gRNA sequence. In addition, multiple genes can be edited at the same time with great specificity (Cong 88-89). The convenience and accessibility of CRISPR resourced have also allowed thousands of laboratories worldwide to study CRISPR in different ways, which has broadened the horizons of its biomedical and clinical implications (Collins et al 259). Overall, the ease and simplicity of CRISPR technology has allowed for a rapid increase in the understanding of genome editing, which will allow CRISPR to revolutionize how certain conditions will be treated.

Crispr uses its protein Cas9 to precisely snip out a piece of DNA at any point within the genome and then neatly stitch the ends back together. This way of editing is effortless and has a deep appeal. This article goes in depth on how Crispr works.

Gene editing used to be a relatively painful and laborious process until the development of CRISPR. In short (and seriously abridging the complex science), CRISPR stands for “Clustered Regularly-Interspaced Short Palindromic Repeats” and are segments of genetic code that, paired with an enzyme such as “Cas9,” have the potential to modify the genes of nearly every organism. The development of CRISPR is to genetics what the development of word processors was to writers; I type, delete, copy, and paste words in this essay (much like what CRISPR can do with genes) elegantly on