Duchenne Muscular Cystrophy Research Paper

Ben has Duchenne Muscular Dystrophy (DMD). DMD is a degenerative disease of the muscles. When someone has this disease their muscles do not produce enough dystrophin to stay together. This causes the muscles to deteriorate over time. With proper care, the rate of muscle degradation can be slowed down. Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. Muscle weakness can start as early as age three. It first affects the hips, pelvic area, thighs, and shoulders. This disease is still fatal and will be until further studies and research are done to find ways to cure this disease.

Imagine how it would be like if you can’t run and have to be in a wheelchair at a young age. You would face lot’s of difficulties like having to get up and walking. A rare disease called Duchenne Muscular Dystrophy can do this to you. Duchenne is a disease with rapidly worsening muscle weakness. It is not very well know. Boys are more likely to have it them girls because boys don’t inherit a flawed dystrophin gene. This gene protects you from Duchenne.

Duchenne muscular dystrophy is a genetic disease that pertains only to males caused by a defective gene and normally leads to many problems affecting a child’s leg movement. This disease got its name from the neurologist, Guillaume Benjamin Amand Duchenne. He was a very consistent doctor who followed many patients medical records. He diagnosed one of his patients with muscular dystrophy and then the disease picked up his name, due to his founding.

Duchenne Muscular Dystrophy, referred to as DMD, is the most severe form of all muscular dysrophies. It is rapidly progressive and occurs primary in boys. DMD is caused by a recessive mutation in the X chromosome. It can be inherited by either parent however, it can also be present with no family members having the mutation. DMD is caused by a lack of dystrophin. Dystrophin is a protein found in muscles that enables the muscle tissues to repair themselves.

Reviewing the ethical dilemmas on this topic listed earlier, those with some type of religious background will most likely have a harder time accepting this new area of scientific development. Whereas, those who do not involve religious beliefs in their view point may see CRISPR as a revolutionary scientific discovery that will significantly benefit the greater good. Allowing CRISPR to be further research, and hopefully one day used, can greatly impact billions of people in such an amazing way. Living with an incurable disease or having the potential to develop one later on in life can be detrimental to not only the individual, but the individual’s family. Eliminating terminal genetic diseases will not only relieve people of pain and

CRISPR Cas9 technology should be used to modify human and embryonic DNA because if we have the technology of CRISPR Cas9 and all of the advantages it entails to help people who want to have healthy children and to stop the inheritance of deadly genetic diseases, then we should. I believe the evidence supports my claim because CRISPR has so many advantages in knocking out the diseased gene to stop the passing down of the diseased gene. Although it is still in the research stage, the number of risks including the off target mutations and the generation of mosaics have been very low and close to none which helps the argument against CRISPR of it being unsafe and unexpected. The evidence states

Crispr is based on a natural system used by the bacteria to protect themselves from the infection by viruses. When the bacterium detects the presence of a viral DNA, it produces 2 types of short RNA. One of which contains a sequence that matches that of the invading viral DNA. These 2 RNAs form a complex protein called Cas9. Cas9 is a nuclease, a type of enzyme that can cut DNA. When the matching sequence, known as the guide RNA, finds the target within the viral genome, the Cas9 cuts the target DNA, disabling the virus. But this procedure can be used for any DNA sequence at a precise location by changing the guide RNA to match the

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

One argument that I agree with only because it would be un-humane not to; is that by using CRISPR on babies who are prone to inheriting medical diseases will be relieved of these potential issues by cutting the mutated DNA out of their genome. At first sight it is hard to say that anyone would disagree with this argument, because who would not want people to be free of impairments? But after further investigation, it actually doesn’t play out that way. For instance, many genes are pleiotropic or have multiple effects on the organism . This means that by selecting for a certain gene to be deleted or over expressed there can be multiple unseen effects on the organism. The classic example is Sickle Cell Anemia. Sickle Cell Anemia is an unwanted disease which causes red blood cells to become misshaped which is harmful to human health. However, in people who live in Africa it is actually beneficial because it prevents malaria. So if we were to “cut” the DNA sequence that was responsible for Sickle Cell Anemia with the use of CRISPR for those who live in Africa where malaria is prevalent could actually cause death and more serve medical issues.

The problem with this is that parents could have the opportunity to decide almost anything for the child, even in its fetus state, which could lead to certain genetic traits being lost. Fortunately, the DNA editing of a fetus is illegal in many countries. Another problem is referred to as the “Gene Dive”. This is the stopping or passing on of a certain gene. The problem is unless this is kept under control in the wild havoc could be let loose on entire ecosystems. If one trait is not passed on, it could spread rapidly if the animal or plant reproduces without a controlled environment. The last, and personally the most troubling, problem is that CRISPR is available to anyone at the moment. Sets can be bought online as we speak. No single company owns CRISPR at the moment so laws are hard to enforce when anyone can access it.

There’s really no reason to stop scientists from doing gene editing, but researchers need to be careful and no one is yet ready to do germline editing that could be passed on to future generations. Gene editing is a wonderful thing that could change the world, but I don’t think we are ready for it yet.

The entrepreneur Barry Schuler once said “By being able to write a genome and plug it into an organism, the software, if you will, changes the hardware.” This compares the process of gene editing with the simplicity of constructing a computer. Through recent years of history, people have developed more advanced technology for genome editing in children while they are still in embryo. One of which is through using CRISPR-Cas9 as a pair of scissors to cut off parts of the gene people don’t want to have in their children. In brief, scientists believe that they can use this method to get rid of many illness in kids through further experiments, including some of the common ones such as muscular dystrophy, cystic fibrosis, and cancer. At the same time, the geneticist George M. Church also has high hope for future medical benefits from this advanced technology in saying, “The goal of

Ethical questions have been raised in regard to the development of the new drug CRISPR-Cas9. CRISPR-Cas9 is a form of genetic engineering that allows for precise modification of DNA. CRISPR-Cas9 works by injecting a modified protein into the body to work on DNA like scissors by snipping the unwanted section of genes. CRISPR-Cas9 creates enormous possibilities for genetic modification, and the advancement of human health. The creation of new technology creates new benefits; however, it also presents significant risk. The use of such drug raises ethical questions such as who will have access to treatment and who will it be developed for. Further ethical questions are raised when considering not only how the modification of genes will affect

Back in the 1970s recombinant DNA technology turned a new page of biology. And what an important page it was-for the first time scientists could manipulate DNA molecules and study genetics up close. The progress of technology led to the development of new genome engineering techniques which gave scientists the tools to study genetics as it happens- straight from the organism of interest. One of these tools is called CRISPR and is promising to transform not only the world of biology, but much, much more.

The pioneer in the primary characterization of the CRISPR system was Francisco Mojica. In 1993, he was the first researcher to notice a pattern in a set of palindromic sequences. He was eventually able to correlate them with the genomes of certain bacteriophage. Following a more thorough investigation, he was able to confirm his hypothesis, and determine that the system was a function of the bacterial immune response. An unusual protein was located in the CRISPR locus by Alexander Bolotin in 2005, this protein was suspected to take part in nuclease activity. Following this new lead, scientists decided to focus their investigation on integrating the CRISPR system into human genome