Genetic Disorders ( Genetic Disease Foundation ) Essay

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

In theory, CRISPR is an extreme method of gene editing and gene editing has been around for years on end. It has been used on plants to make them a more sustainable crop. CRISPR technology has not yet been perfected to be accessible to everyone. Yet the first clinical crispr trial happened in October of 2016, Michael Le Page tells us that in the first clinical trial of CRISPR being used "Doctors removed immune cells from the blood of a person with lung cancer, used CRISPR to disable a gene called PD-1 and then returned the cells to the body," the results of the trial are said to not be released until 2018 (Le Page 1).

Humans have been genetically engineering organisms for nearly 10,000 years using traditional methods of modification—among these methods include selective breeding and crossbreeding. Though effective, these methods were unreliable and were only able to change certain traits. A lack of control over our genetic material proved to be a clear hindrance to our species; when harnessed, advancements in other fields of knowledge would be immeasurable. Once seen as an impossible task, scientists have been able to exploit genes and take control of them. CRISPR-Cas9 is a system that allows scientists to cleave off sections of DNA and artificially modify them by inserting a mutation into the place of the old DNA. This is exceptionally precise, whilst

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

Genome editing is still relatively new in the science world. It was only fairly recently that we gained the first ability to fix our DNA (Rajan). Genome editing is changing the DNA, which gives us the ability to change it for the better – which is not an easy thing to do (What Is Genome Editing?). In order to change the DNA, clustered regularly interspaced short palindromic repeats (CRISPR) are manipulated to improve the DNA (Hornblower, Reis, Robb, and Tzertzinis). A fracture is made in the DNA to modify it, in order to adjust the DNA to try to rid it of genetic diseases and abnormalities (What Is Genome Editing?). Then, a new sequence can be added into the existing DNA and be repaired (What Is Genome Editing?). This is the basic idea of the difficult genome editing. While complicated, once more progress is made and it becomes more successful, this process could work on a variety of diseases (What Is Genome Editing?).

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.

Modifying genomes hasn’t been perfected, and is far too great of a risk to use on society. No one knows what could happen to the population after man starts messing with mother nature. Scientists who are trying to destroy genetic diseases could create new problems or end up killing its test subjects later down the line. The possibilities for disasters are endless. People also claim that modifying humans is the fastest and most efficient way to getting rid of physical and mental health problems, but according to Molecular biologist Ellen Jorgensen, Using CRISPR isn’t what people see it to be. Ellen explains that using CRISPR is not actually easy at all. To use the technology, damaging the gene is crucial. Once the gene is damaged, and the cells start to repair, scientists can then manipulate the cells to repair the way they want it to, and not naturally. This is a complicated process, and it not in any way

The reading shows disease and inheritance in an entirely new light. It introduces the idea that genetically inherited diseases may have been selected for, which means that they must provide certain evolutionary advantages. It reorients the reader’s perspective about a disease like hemochromatosis, which has the potential to be incredibly harmful and even deadly, establishing that it may have once provided protection from the bubonic plague, making it an advantageous trait. This brings other genetic diseases into question, examining why diseases that appear to be harmful have not been eliminated from the gene pool. The idea that a disease that is harmful and dangerous in modern times could have once been a beneficial adaptation is very interesting.

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

There are many genetic diseases and disorders in the world these days, some of which are stranger or more unique then others. One such unique genetic disease is Congenital Insensitivity to Pain with Anhidrosis or CIPA for short. This particular genetic disease causes people who have to it to not be able to feel pain or even differentiated between hot or cold. As well as be unable to sweat, because of the anhidrosis. This genetic condition is also known as hereditary sensory and autonomic neuropathy type IV, but in this paper it will only be referred to as CIPA.

In the first human altering, scientists used a germ line editing technology called CRISPR. This new technology is believed to be a new approach to gene therapy. This technology is used to weaken deadly blood diseases. The CRISPR is created to point out the material in the genes and ultimately stop certain genes at the DNA level. This technology was tested by inserting 86 embryos and waited 2 days for the results. Through this experiment only 71 survived, with these 71, 54 were analyzed. Out of the 54, only 28 were correctly spliced. However only a few contained the replacement genetic material. The CRISPR was used by multiple biologist as a search and substitution tool for altering DNA only 3 years after the first development. With using all the technology and by altering human genes this procedure cost $12,400 dollars. Although they have used it to experiment genes in the DNA, the technology used in this process is not completely safe and still in the beginner stage. A scientist, Huang, has studied the ability of the technology CRISPR, and how they alter genes called HBB. However they stopped using this technology because it was not proven 100%

Wiskott-Aldrich Syndrome (WAS) is a genetic defect characterized by immunodeficiencies and an inability to properly form blood clots. It affects 4 out of 1 million live births worldwide. Patients with WAS show symptoms such as eczema, easy bruising, susceptibility to infection, autoimmune disorders, and in many cases, lymphoma in later stages of life. Wiskott-Aldrich Syndrome was discovered in 1954 by Dr. Robert Anderson Aldrich while examining one of his patients. His findings were similar to that of Dr. Alfred Wiskott, who documented the disease in 1939, but did not know its cause or what the disease was. He noted that the three brothers of the family he studied had WAS, but not the sisters. In 2006, German researchers studied the family members of the brothers from Dr. Wiskott’s documentations and saw that the Wiskott-Aldrich gene was mutated.

Gene editing is not a new concept, however the recent development of the CRISPR/Cas9 gene editing technique has greatly simplified the process of gene editing for scientists. This powerful new technique has many applications in biomedical research and has the potential to treat genetic diseases [2]. All cells in the human body contain a copy of the human genome, which contains almost 25 000 genes. Each molecule of Deoxyribonucleic acid (DNA) is made up of two complimentary strands twisted into a double helix that is held together by hydrogen bonds. Genes are a blue print for the human body, giving it instructions on how to create essential proteins [3]. Due to major advances in technology, scientists and researchers have identified thousands of genes that code for certain diseases. Changing genes in cells is not an easy process, however it can be simplified by using the CRISPR-Cas9 system.

The development of this technique can be the key to cure many diseases. According to GeneScript “CRISPR/Cas9-mediated gene editing is a powerful technique that allows you to create knock-in/out mutations in any gene and any cell.” In addition, according to New Englang Biolabs “Three types of CRISPR mechanisms have been identified,