This website uses cookies to ensure you have the best experience. Learn more

Gene Therapy For Cystic Fibrosis Essay

1009 words - 5 pages

9.1 Gene Therapy
Gene Therapy is a popular target for gene mutations as it combats the source of the disease rather than the surpressing the symptoms. However this therapy has many limitations. The review paper of T.R. Flotte and Beth L. Laube [71] have published facts about gene therapy which will be shortly reviewed in the following paragraphs.
To begin with there are two major pathways for gene therapy: Transduction and Transfection. For Transduction one needs a virus which transfers a piece of DNA, in this case the CFTR gene into target cells. In Transfection on the other hand a plasmid is inserted in the eukaryotic cell via transformation of eg. E. Coli. This can also be performed in ...view middle of the document...

However, recent research by Fisher K.J. et al [72] suggests that deleting the whole viral genome is the next step in Ad virus modification. The now called “High Capacity Ad Vectors” have the benefit of enhanced expression duration and are less prone to be attacked by human immune system. Unfortunately the humoral immune system against the capsule of the virus still prevails [71] and the responsible antibodies cannot be suppressed without increasing the risk of inducing tolerance of wild-type Ads. [73]
9.1.2 Non-viral Infection
To avoid the problems with virus infections an alternative pathway with cationic liposomes has been developed. These are special complexes which consist of DNA and lipid. [71] It can form a large complex where on one side it interacts with the DNA and at the other hydrophobic part it interacts with the cell membrane or enhances the condensation of the complex. [74] Even though its side effects are lower than virus transfection, the low efficiency remains a problem. On the other hand its low inflammatory response makes repeated dosage possible. Other molecular conjugates which consist of protein and DNA complexes are tested as an alternative. Using proteins as a ligand which triggers receptor mediated endocytosis, offers great results in in vivo experiments. [71]
9.1.3 Augmentation and Gene Targeting
The last few paragraphs have shown how to introduce DNA into a cell but one also has to differentiate how the mutated gene should be influenced. In Augmentation the entire CFTR locus is being replaced by a functioning one by a bacterial artificial chromosome (BAC) as practiced in the study of Auriche et al. [87]. This approach has the advantage that the exact mutation does not have to be specified to show good results in in vitro experiments. Yet a problem they are facing refers to the regulation of the newly inserted gene. Needless to say a gene does not only contain genetic material but also regulators of expression which are artificially added in augmentation but they...

Find Another Essay On Gene Therapy for Cystic Fibrosis

Caring for Children with Cystic Fibrosis

2560 words - 11 pages taking these medications and treatments. When children are first diagnosed with cystic fibrosis, they are typically very young. From the beginning, there are many medications and treatments that needed to be taken and done. Sometimes, trying to get children to take medications and treatments is like pulling teeth. The medications for cystic fibrosis are extremely important. If cystic fibrosis patients miss medications or treatments, things may be

Development of Mouse Models for Cystic Fibrosis

1601 words - 7 pages Cystic fibrosis (CF) is a common, lethal, autosomal recessive disorder caused by mutations in the CFTR gene, with the most common mutation (ΔF508) occurring on ∼70% of CF chromosomes. Dysfunction of the CFTR protein, which acts as an apically localized epithelial chloride ion channel, results in the classical manifestations of CF: salty sweat, pancreatic insufficiency, intestinal obstruction, male infertility, and severe pulmonary disease, with

The Symptoms, Causes and Remedies for Cystic Fibrosis

1287 words - 6 pages protein cause secretions such as mucus and digestive juices to be abnormally thick and sticky” (Masel). More than 1100 mutations of this gene have been identified to date (Fitzgerald). This genetic disease is inherited meaning it is passed on from parent (mom and dad) to child but both mom and dad must have the mutated gene in order for this to occur. Cystic Fibrosis does not have gender or racial specifications. However, Caucasians of Northern

Gene Therapy for Multiple Sclerosis, Cancer, and Sickle Cell Diseases

731 words - 3 pages , it is class of diseases characterized by out-of control cell growth. Currently there is around 100 different types of cancer. In current times some forms of cancer are already curable but some are only curable up to certain stages. Currently in the word there is around 13.5 million diagnosed with cancer. It will benefit the whole world if gene therapy is able to cure cancer. Many people lives will be saved and we will be done with this dreaded

Gene Therapy

1035 words - 5 pages aims to annihilate the problem altogether (Learn Genetics: Gene Therapy). Cystic fibrosis also happens to be a perfect candidate for gene therapy since it only occurs on a single gene (Learn Genetics: Gene Therapy) and cells that have the cystic fibrosis gene present will not affect cells that do not (About Cystic Fibrosis). Also, since the lungs can be so easily accessed through the air one breathes, the research shows that it is easy to inject the

Deadly Diseases: Cystic Fibrosis

666 words - 3 pages mutated gene to offspring, especially in the certain population with the high disease prevalence. Cystic fibrosis is symptomatic when two disease carriers have children, who inherit two recessive genes. Cystic fibrosis significantly increase mortality and morbidity, with the life expectancy approximately 30 years. The mucus clogs up the lungs, which leads to breathing difficulties with severe cough and shortness of breath, frequent respiratory

Cystic Fibrosis

1699 words - 7 pages , which affects the chloride ions in the body. It causes thick mucus, which blocks airways in the body, causing infection. The symptoms of Cystic Fibrosis can vary and increasingly worsen over time. It affects mainly the respiratory and digestive systems. Doctors have found many treatments and techniques that help ease the symptoms for the patients; including airway clearance techniques, inhaled medicines, therapy vests, and exercise. Before

Cystic Fibrosis

930 words - 4 pages babies born with cystic fibrosis reach adulthood, and further advances, particularly in the field of gene therapy, may produce even better treatments in the coming years.Cystic fibrosis is caused by a defect in the gene responsible for developing cystic fibrosis transmembrane conductance regulator (CFTR). A protein controls the flow of chloride ions into and out of certain cells. In healthy people, CFTR forms a channel in the plasma membrane so

Overview of Cystic Fibrosis

1316 words - 5 pages vital organs, such as the lungs, pancreas, and intestines. One can determine if he/she has cystic fibrosis by analyzing certain symptoms. Cystic Fibrosis can be diagnosed according to the symptoms the patient shows, and can be treated through specific types of treatments, such as gene therapy. Cystic Fibrosis is a lifelong disease that has a harsh negative affect on many vital organs, some of which include the lungs, pancreas, and intestines

Cystic Fibrosis

968 words - 4 pages Collins and Professor Jack Riordan discovered the exact faulty gene that was responsible for CF in 1985 after cystic fibrosis was narrowed down to chromosome 7. Chromosome 7 was called the cystic fibrosis transmembrane conductance regulator (CFTR). This discovery was one of the most important discoveries for cystic fibrosis research! In the 1990’s, it was discovered that there was dangers of cross-infection amongst people that are have cystic fibrosis

Cystic Fibrosis - 2384 words

2384 words - 10 pages copy of the gene from each parent. This gene mutation is responsible for cystic fibrosis. ("Cystic fibrosis Canada," 2011) This gene is known as the CFTR gene ("," 1996). Reference Figure 3. The “gene makes a protein that controls the movement of salt and water in and out of your body's cells. In people who have cystic fibrosis, the gene makes a protein that doesn't work right. This causes the thick, sticky mucus

Similar Essays

Cystic Fibrosis: A Case For Gene Therapy

752 words - 3 pages Cystic Fibrosis "“ A Case For Gene TherapyCystic Fibrosis is caused by the mutation in the gene, which is located in the long arm of chromosome 7, causing the loss of a single amino acid from a large protein and thus disrupting the binding site for ATP in the protein. Cystic Fibrosis is inherited genetically from one generation to the next due to an autosomal recessive allele. People suffering from Cystic Fibrosis are homozygous for the

Cystic Fibrosis And Gene Therapy Essay

1337 words - 5 pages future looks promising.The aim of this report is to examine Cystic Fibrosis as a genetic disorder, how it affects the lives of those born with it and to investigate gene therapies associated with it. This report also investigates the ethical issues and recent developments in gene therapy as well as the factors that have kept it from becoming an effective treatment for genetic disorders.All of the information presented in this report has been

The Cystic Fibrosis Gene Essay

1262 words - 5 pages copies of the mutated gene into the affected person's airways so it can replace the mutated nucleotides. This known as gene therapy.      A drastically different approach taken in an attempt to cure cystic fibrosis, performed as a method of preventive medicine, involves correcting the disease while the affected "person" is still an embryo. Test tube fertilization from affected parents and gene diagnosis during

Closer To A Cure For Cystic Fibrosis

1299 words - 5 pages commonly used viruses are “Retroviruses, Adenoviruses, Adeno-associated viruses, and Herpes simplex viruses” (2). The primary virus used in gene therapy is the “adenoassociated virus” (AAV), because they are able to “insert their genetic material at a specific site on [the] chromosome” (2). And even though “trials experimenting with gene therapy” as a possible cure for cystic fibrosis have been unable to show that “gene transfer efficiency” is