Cysstic Fibrosis 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

Once the complex was bound to the DNA, a cut would be made to eliminate and destroy the invaders. 83% of archaeal genomes and 45% of bacterial genomes (Shabbir, M. et al, 2016) were shown to be able to successfully utilize the CRISPR Cas9 system. These are very promising statistics, so it is no wonder that there has been such an advancement in the past few years to bring this technology to eukaryotic cells, mammalian cells and eventually human cells.

The CFTR gene encodes a protein that is present in epithelial tissues in lungs, sweat glands, and pancreas, and helps

Cystic fibrosis is known to be one of the most common and deadly diseases in Caucasians, affecting 1 in 2500 children. This percentage results in 30,000 individuals within the United States to be diagnosed with CF. There are over 1900 mutations of this gene that cause a wide variety of severities within this disease. (McCance, Huether, Brashers, & Rote, 2010) Due to its complex mutation and unknown cause, only treating the symptoms of CF have been the main treatment protocol to this disease. Current treatments are cumbersome and expensive providing patients with life expectancy only into their twenties, but usually younger in most cases. There has been specific progress towards a cure involving gene therapy providing hope for a cure to

A blood sample through a heel prick test is immediately taken after birth. If this test proves to be positive, a sweat test will be done to measure the amount of salt in the sweat. Most babies who have CF are now diagnosed within the first two months of

Cystic fibrosis (CF) is an inherited autosomal recessive disorder that affects the lungs and digestive system most often. In the United States some 30,000 children and adults have CF. There are approximately 1,000 new cases of cystic fibrosis diagnosed each year in the US with 70% of patients diagnosed with CF by the age of two, 40% of patients with CF are 18 or older. In the 1950's most children with CF did not survive to attend elementary school, but in 2006 the median age of survival was 37 years (Cystic Fibrosis Foundation, 2007).

common life-shortening disease. More that 1,000 mutations in the CFTR gene have been found in

Cystic fibrosis is a recessive genetic disease caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This mutation causes problems with the apical membrane CFTR protein which when working to its full potential regulates the sodium and chloride intake in epithelial cells (Eitan Kerem et al. 2005). It affects about 70,000 people in the world and in the UK it is said to affect 1 in every 2,500 children. The disease causes the apical membrane CFTR protein to not be able to transport the sodium and chloride it needs to, this can have a huge effect on multiple organ systems, mainly the respiratory system. The most common clinical problems Cystic Fibrosis causes are pulmonary damage

i. Cystic Fibrosis (CF) affects those that are homozygous recessive for the F508del mutation in the CFTR (Cystic Fibrosis transmembrane conductance regulator) gene

Cystic fibrosis (CF) is an autosomal recessive disease that affects the cystic fibrosis trans membrane conductance regulator (CFTR) gene located on Chromosome 7, in a persons DNA. Autosomal means that the gene for CF is not carried on the sex chromosomes and both male and females are affected by the mutation. The disease is a recessive disease meaning that it requires 2 abnormal genes to be expressed; only one gene would mean that the person is a carrier of the disease. This is because the normal CFTR gene dominated the abnormal CFTR gene. For the disease to be present and show in the person, they must have inherited 2 abnormal CFTR genes, one from each parent. Being a ‘carrier’ does not mean that you have CF but it does mean that you can pass the defective CFTR genes onto your children, possibly leading the off-spring being affected by the CFTR genes. It is the most common fatal genetic disease in the US today with an average life span of 25-30 years. (Ndsu.edu, Cystic Fibrosis and Gene Therapy Ndsu.edu,. (2014). Cystic Fibrosis and Gene Therapy. Retrieved 14 August 2014, from http://www.ndsu.edu/pubweb/~mcclean/plsc431/st). CF is a genetic disorder that affects the digestive and respiratry systems. The CFTR gene causes a protein to not work properly blocking the movement of NaCl (Sodium Chloride) in and out of the cell, causing an abnormally sticky and thick mucus to be produced on the out side of the cells. This mucus clogs the lungs leading to infection. It also blocks

CRISPR-Cas9 is a unique technology that can be used to edit the human genome (1). Compared to other techniques of editing DNA, CRISPR and Cas9 are cheaper, faster, and more accurate. CRISPR-Cas9 gives geneticists as well as medical researchers the ability to edit specific parts of the genome by the removal, adding, or altering of certain sections of DNA sequences (1). CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats and is a part of bacterial defense system (2). The two key molecules of CRISPR-Cas9 are Cas9 (an enzyme that can cut DNA at specific locations) and guide RNA (a small piece of RNA sequence that guides where the Cas9 should cut) (1). CRISPR is a crucial component

Cystic Fibrosis is caused by a genetic defect in Chromosome 7. Chromosome 7 encodes the cystic fibrosis transmembrane conductance regulator, also known as CFTR. There are over 1,000 mutations of this gene causing cystic fibrosis, with each mutation manifesting as a different variation of disease onset and clinical presentation. The most common mutation is the loss of phenylalanine residue at deltaF508. The abnormal functioning CFTR causes impaired chloride transport and more viscous secretions. The defect causes dehydrated secretions in the respiratory tract and gastrointestinal tract. Being dehydrated, these secretions become more difficult to move throughout the body. Along with impaired

There still needs much to be done such as researching the appropriate risks and benefits for the clinical trials (Sciences Engineering medicine (20--). It is rapidly growing and increasing by making heritable genome editing of the embryo, egg, and sperm in the hopeful future (Sciences Engineering medicine (20--) It is very likely to be many years before CRISPR-cas9 is used in human beings. The main attention is drawn towards animal models or isolated human cells (CRIPSR 20--). The purpose of this for the goal to treat diseases in humans. Another plan for the future is to focus on eliminating “off-target” effects, where the CRISPR-cas9 entity cuts different gene to one the one intended to be edited (CRIPSR 20--). In our world today, the

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,

A common form of gene therapy is to take a genetically engineered virus, or a viral vector, like the Herpes simplex virus (HSV) or Adenovirus. These viruses purposes are to infect cancer cells while leaving the healthy cells in the body alone (Cross). Viruses are modified to rid the virus of the toxicity, but maintain the virus’s high gene transfer capability (El-Aneed). The genes are modified by deleting parts or all of the coding regions in the viral genome (Thomas). An RNA guided editing tool called CRISPR-Cas9 (clustered regulatory interspaced short palindromic repeats) is capable of editing multiple genes simultaneously (Xiao-Jie). CRISPR-Cas9 is a cost effective tool in gene therapy and while it is relatively new, with just being introduced for mammalian use in 2013, it is already proving to be very useful. This type of gene therapy is where problems arise with a patient’s low immune system and high risk of infections, the biological system can create an infection in the patient.