Closer to a Cure for Cystic Fibrosis Essays

Closer to a Cure for Cystic Fibrosis Essays

Length: 1399 words (4 double-spaced pages)

Rating: Powerful Essays

Open Document

Essay Preview

Overcoming Proteasomes: One Step Closer to a Cure for Cystic Fibrosis

Take a deep breath and consider how easy it was to do so. Now picture struggling and
gasping for air everyday; normal, easy tasks to the average person prove to be quite challenging
to a patient of cystic fibrosis. Scientists have always been daunted by this fatal genetic disease
that affects the body by excreting a “thick mucus” in the lungs, making breathing difficult and
blocking the “ducts” leading from the pancreas, causing “poor digestion of food” (1). Until
recently, there was nothing that scientists could do to provide a long-term cure, but even though
scientists cannot cure patients one hundred percent, they can increase their life expectancy.
Despite having discovered the cystic fibrosis gene’s location in 1989, numerous underlying
obstacles prevent complete success (2). The primary obstacle that prevents scientists from
making gene therapy an effective cure is the placement of the healthy genes into long-term cells,
the cells that remain long enough to be replicated. The replacement of the healthy gene into
long-term cells is necessary because these cells make the new cells, thereby distributing the
healthy DNA throughout the body. The properly functioning gene is attached to a vector or
“carrying molecule” that will transport “the therapeutic gene to the patient’s target cells” (2).
Currently, the most common vector is a virus because it can easily capture the gene and deliver it
into the cell; the virus infiltrates the corrupt cells and places the healthy gene into the nucleus
which then transforms the corrupt cell into one which operates properly (2). The only difficulty
is that the body’s natural immune system provides many b...

... middle of paper ...

2. Institute NHGR. Gene Therapy [Internet]. 2007 [2007 September 18]; Available from
3. Stefano Ferrari DMG, Eric WFW Alton. Barriers to and new approaches for gene therapy
and gene delivery in cystic fibrosis. Advanced Drug Delivery Reviews 2002;64: 1373-1393.
4. J Kim C-PC, KG Rice. The proteasome metabolizes peptide-mediated nonviral gene
delivery systems. Gene Therapy 2006; 12: 1681-1690.
5. Neil Campbell, and Lawrence Mitchell (1999). Biology. New York, Addison Wesley
Longman, Inc.
6. Institute NHGR. Learning About Cystic Fibrosis [Internet]. 2007 [2007 September 18];
Available from: .
7. U Griesenbach DG, and EWFW alton. Gene therapy progress and prospects: cystic
fibrosis. Gene Therapy 2007; 13: 1071-1077.

Need Writing Help?

Get feedback on grammar, clarity, concision and logic instantly.

Check your paper »

Life-Threatening Disease: Cystic Fibrosis Essay

- Cystic Fibrosis is an autosomal recessive disease that affects 30,000 people in the U.S alone and near 70,000 worldwide. The life-threatening disease primarily affects the digestive system and the respiratory system. It is the cause of a dysfunctional CFTR gene, which disrupts the exocrine functions of the pancreas, biliary tree, bronchial glands and the sweat glands. The gene on chromosome 7 is in charge of moving the salt in and out of the cells within the body. When the cells are not capable for the efficient flow of salts a different kind of mucus is produced....   [tags: symptoms, signs, digestive problems]

Powerful Essays
580 words (1.7 pages)

Cystic Fibrosis : A Genetic Disease Essay

- Cystic Fibrosis Cystic Fibrosis is a genetic disease that affects many people today. It takes over a person’s whole life to survive this disease. To survive, people who suffer with Cystic Fibrosis need constant care of this disease. Cystic fibrosis is disorder that is inherited and causes severe damage to the lungs and digestive system. Cystic fibrosis changes the cells that make mucus, sweat and digestive juices. These fluids that are secreted are normally thin and slippery. A defective gene causes the secretions to become thick and sticky....   [tags: Cystic fibrosis, Genetics, Digestion, Pneumonia]

Powerful Essays
809 words (2.3 pages)

Symptoms And Treatment Of Cystic Fibrosis Essay

- Your child is under weight, has greasy, smelly diarrhea, a chronic cough and seems to always be getting pneumonia. He wheezes when he breathes and has impaired exercise ability. He seems to never being growing to the average size of his peers and his skin is very salty (5). The doctor’s first instinct should be to test your child for a disease called cystic fibrosis. Cystic fibrosis is a disease, which causes the mucus in your body to be thicker in some areas. The most affected areas are the lungs and digestive system....   [tags: Digestion, Cystic fibrosis, Pancreas, Cell]

Powerful Essays
1245 words (3.6 pages)

Essay on The Cystic Fibrosis Transmembrane Regulator Gene

- Cystic fibrosis is a genetically inherited disease due to a mutation in the CFTR gene. This disease causes life changing health complications that affect many organs within the human body. There is always ongoing research for this disease to discover better treatment options that are more effective and possibly find a cure to reverse the mutated gene. The Cystic fibrosis transmembrane conductance regulator gene (CFTR) is responsible for providing the instructions to help make the protein that produces mucus in the body....   [tags: Cystic fibrosis, Genetics, Mutation, Asthma]

Powerful Essays
1836 words (5.2 pages)

The Effect Of Cystic Fibrosis Essay

- The effects of cystic fibrosis can be broken down to a micro-perspective investigation in both the genotype, the encoding of the DNA, and the phenotype, the expression of DNA to trait. A precursor to the expression of cystic fibrosis, is through the inheritability pattern of cystic fibrosis from both parents. Cystic fibrosis is an autosomal recessive disease, meaning in the DNA of each parent, each had one copy of these recessive trait which is not expressed through them. However, with both parents being carriers, the offspring has a 25% chance of inheriting cystic fibrosis, a 25% chance of inheriting non-mutated genes and a 50% chance of being a carrier of the cystic fibrosis gene (Mayer-Ha...   [tags: Mutation, DNA, Gene, Cystic fibrosis]

Powerful Essays
992 words (2.8 pages)

Essay about A Brief Note On Cystic Fibrosis ( Cf )

- Cystic Fibrosis Introduction Cystic fibrosis (CF) is an inherited chronic disease that must be carried by both parents in order to for the person to suffer from it. When two parents who are carriers have a child there is a 25 percent chance of having a child with CF and when one parents has CF and one parent is a carrier, there is a 50 percent chance of having a child with CF This disease affects organs such as the liver, lungs, pancreas, and intestines. This disease disrupts the body’s salt balance leaving too little salt and water on the outside of cells which makes the thin mucus that protects the linings of the airways, digestive system, reproductive system, and other organs and tissues...   [tags: Cystic fibrosis, Pulmonology, Asthma]

Powerful Essays
1312 words (3.7 pages)

Essay on Cystic Fibrosis : A Lung Disease Caused By A Gene

- Cystic Fibrosis Cystic Fibrosis is a lung disease caused by a mutated gene. This gene called the CFTR gene causes a protein to not allow salt or water to flow through a cell properly. This produces a thick, sticky mucus to collect in the lungs and other organs. In order for a person to get this disease, the parents both have to be carriers of this disease. Usually a person is diagnosed within the first year of life. In the United States 1 in 2,500 to 3,500 Caucasian Americans, 1 in 17,000 African-Americans, and 31,000 Asian-Americans will have this disease....   [tags: Cystic fibrosis, Pulmonology, Asthma]

Powerful Essays
1397 words (4 pages)

Taking a Closer Look at Gene Therapy Essay

- Gene Therapy Genetic disorders have been plaguing people for ages and causing death. As of today in the year 2013 new information and research, and something called gene therapy, hope now exists for these less fortunate individuals. Gene therapy is a technique for correcting defective genes responsible for disease development. Research Scientist realizes it has been around for a while now and is getting more advanced with time. Experiments which is a ongoing process pertaining to gene therapy. Ethical issues are something that has been accompanying the procedure since it has been used....   [tags: genetic disorders, ethical issues]

Powerful Essays
942 words (2.7 pages)

Genetic Defects in Cystic Fibrosis Transmembrane Conductance Regulator Essay

- Abstract: The main goal of this paper is to explain what Cystic Fibrosis is and also to explain what the causes of Cystic Fibrosis are. Cystic Fibrosis is caused by a mutation in a gene called Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). Cystic Fibrosis is known as one of the most common life-shortening disease. More that 1,000 mutations in the CFTR gene have been found in people with Cystic Fibrosis. Most of these mutations change single protein amino acids in the CFTR protein and it deletes a small amount of DNA from the CFTR gene....   [tags: Biology Medical CFTR Cystic Fibrosis]

Powerful Essays
717 words (2 pages)

Cystic Fibrosis Essay

- Imagine drowning, lungs filling with water that swallows the air and suffocates those caught in it. Now, imagine drowning in a hospital bed surrounded by doctors and family members who can only stand by and watch the inevitable. This is the fate of a person with Cystic Fibrosis. Cystic Fibrosis is a disease that forces a person to drown in mucus that fills their lungs while it wreaks havoc on the body. This chronic disease causes devastating health problems, has no cure, and forces patients to endure painful temporary treatments....   [tags: Chronic Disease, Health Problems, Treatments]

Powerful Essays
1776 words (5.1 pages)