Duchenne Muscular Dystrophy Case Study

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

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

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

Nowak KJ, & Davies KE. (2004). Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment. EMBO Reports, 5(9), 872-876. doi:10.1038/sj.embor.7400221

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.

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.

CRISPR Cas9 has many advantages, but it has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate, and more efficient than other existing genome editing methods. CRISPR reduces the time required to modify targeted genes and has improved bioinformatics tools to identify the best sequences to design guide RNAs. CRISPR Cas9 in embryos works extremely well for the generation

(…) Researchers can edit genes by ridding the faulty genes and replacing the lettering(coding) with the CRISPR-Cas9.

The ability to engineer biological systems and organisms has an enormous potential for applications across basic science, medicine and biotechnology. Genome editing is a group of technologies that allow scientists the ability to change an organism’s DNA, which can provide better outcomes for health and disease control compared to natural immunity and mutations. Genome editing (gene editing) allows genetic material to be added, removed or altered at particular locations in the genome. A number of gene editing technologies have emerged in recent years with one of the most versatile and precise methods of genetic manipulation being Crispr-Cas9 (Steve scott,2016). The term Crispr-cas9, (clustered regular interspaced short palindromic repeats)

CRISPR is a new gene editing technology that has recently become popular in the scientific community in addition to the media due to its vast range of applications as well as the controversy that accompanies the topic. Because this is such a touchy subject, it is difficult for the technology to gain traction in the community. It follows that gaining the support of the public is a crucial step if scientists wish to continue the advancement of the technology. One way this can be done is by facilitating communication between communities, specifically, by publishing articles about new information and advancements that emerge, so the public can be educated on the topic of CRSIPR and the field can gain their support.

CRISPR has the possibility of revolutionizing medicine—fighting disease, curing cancer, and eliminating genetic diseases. One way in which CRISPR would fight against cancer, is achieved by removing the immune cells from a cancer patient. The cells would then be genetically modified using CRISPR—targeting the immune cells against the cancer—and then injected back into the patient. Another application to fight cancer involves editing genes to stop cancer cells from multiplying. These uses of CRISPR would be used to effectively fight cancer. Researchers using CRISPR have already reversed the mutations that cause blindness, cystic fibrosis, and hemophilia. Parents that are carriers of some genetic diseases would be able to treat the embryo with CRISPR in order to reverse these genes, resulting in a healthy child. These applications all have incredible outcomes; why are they not being pursued by all medical companies

PURPOSE. The purpose of this study is to examine how CRISPR genome editing technology can be used to change heart muscle dystrophin expression and enable proper muscle function in dystrophic mice. Dystrophic cardiomyopathy is the most common cause of death in patients with DMD (Duchenne muscular dystrophy).

Many researchers around the world have been using the CRISPR-Cas9 gene editing tool to try and fix or alter disease-causing mutations in human embryos. Many of these scientists have found a mutation in these genes known as, MYPBC3. This type of mutation mainly affects young athletes. It can cause the heart muscle to thicken, which is known as a heart condition called, hypertrophic cardiomyopathy. This type of condition is a dominant gene. Meaning, that if a child inherits this mutation, they will have experience some sort of symptom from it. Many scientists hope that using CRISPR-Cas9 will delete this genetic mutation in human embryos.

The CRISPR-Cas system (often referred to CRISPR-Cas9) is defined by four functional components for efficient RNA-guided genome modification. These components are Cas9, RNase III, tracrRNA, and pre-crRNA. The first of the functional units is the Cas nuclease (1,2). Cas protein has been shown to have helicase and nuclease activity (1), and is the catalytic