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Cystic Fibrosis Gene Therapy – A Cure For This Terrible Disease?
Cystic fibrosis is a genetic disease that affects certain glands in the body, including those responsible for producing mucus and sweat. It is an inherited disease, meaning that this disease is not contracted from some outside source but is rather the effect of an abnormality in some part of a person’s genes. This disorder is caused by the absence of a gene known as the cystic fibrosis trans-membrane conductance regulator.
This gene plays an important role in the creation of sweat, digestive juices, and mucus. CF has been known to affect the lungs, gastrointestinal tract, liver, and pancreas. In the lungs, cystic fibrosis causes overproduction of mucus, resulting in inflammation of the affected tissues. This leads to symptoms such as coughing, weakness, and excessive phlegm production. Eventually, bacteria take refuge in the thick mucus and begin to multiply, avoiding the body’s immune system thanks to the protection of the mucus, and lead to secondary infections such as pneumonia.
When it affects the digestive system, cystic fibrosis causes production of thickened secretions from the pancreas, which is responsible for producing important digestive enzymes. These thick secretions block the passage from the pancreas to the digestive tract, and also cause irreversible damage to the pancreas itself. The prevention of digestive enzymes from reaching the digestive tract leads to a lack of absorption of important minerals and nutrients from food, and can result in malnutrition.
Current Treatments for Cystic Fibrosis
Most current treatments for cystic fibrosis deal solely with the management of symptoms of the disease. Most of the treatments commonly used on patients are not cures, and simply help to remove mucus secretions and prevent bacterial infections from taking root in the lungs. Scientists have, however, come up with a new and revolutionary treatment that should be able to provide a lasting cure for this disorder when it is fully developed. This treatment is known as gene therapy.
Gene Therapy & Its Role in Treating Cystic Fibrosis
Since cystic fibrosis results from errors in the genes, scientists seek to correct those errors by replacing the erroneous genes in the affected cells with new, correct genes. This is known as gene therapy. The ideal solution is to be able to insert correct genes into all of the cells affected by the gene for cystic fibrosis. Scientists have discovered that it is extremely difficult to get affected cells to take up the modified genes, however, and thus treatments have thus far proven to be relatively ineffective.
New methods of delivering the corrected genes to the affected cells hold great promise, however, and various methods have already entered testing phases. Once scientists perfect the method of cystic fibrosis gene therapy, patients will then have a proper cure for the disease, as opposed to simply preventing the decline of organ function for as long as possible.
Gene therapy faces serious opposition from some sectors however; as there are people who believe that introducing any form of gene into the human body is unethical. Such protests have yet to have any effect on the progress of research into gene therapy methods, but there is the chance that the scientific community will have to deal with such accusations sometime in the future.
Article Source: http://EzineArticles.com/?expert=Didier_De_Coster
Tags: cystic fibrosis, digestive enzymes, digestive juices, gastrointestinal tract, glands in the body, mucus secretions, revolutionary treatment, secondary infections, thick mucus, thick secretionsStem Cell Healing and Gene Therapy – The Miracle Units to Rid Genetic Diseases
Gene is the structural and functional unit of life. These basic entities are the vehicles that carry entire genetic information encoding for every function of the body. This it does so by encoding for specific amino acids. Several amino acids team up to build proteins. It is these proteins which will then be an integral part of hormones, enzymes and other essential components which will regulate the body mechanisms. In most cases one gene codes for one amino acid and several amino acids make for a single protein. Thus in the built up of a single protein which can serve as enzyme or hormone is made up by the instructions coming from a group of inter-related genes.
It is essential to understand that in the event of any alteration in the structure of gene, defective commands will be given causing the formation of wrong structure of protein. The alteration takes place by way of mutation caused due to natural aging process, environmental factors, radioactive components, ultra violet rays and others of the kind. This altered protein will not be similar to the protein desired and needed by the body to perform the required function. This means the metabolism is faulty and not taking place as required. This is externally manifested by way of a disease.
The natural corollary of this would be that correction in the defective make up of the gene would automatically lead to normal functioning of the body. The current motto is ‘To repair what you can and replace all that what you cannot repair’. This is now being taken care of by the break through in stem cell research and gene therapy. The repair and replacement therapy is taken care of by way of stem cell and gene therapy. It is aimed to restore the original functions and restore back to the disease-free state.
o Stem cells are primarily taken up because they are still at the threshold of adolescence. They are active, vigorous and have the potential of being transformed into mature cells of the desired type such as the nerve cell, blood or any other tissue. The acquire forms similar to the neighboring cells in whose vicinity they fall.
o Practically this is undertaken by first boosting the production of stem cells in the bone marrow and in the connective tissue around blood vessels of the umbilical cord and the placenta. These stem cells are harvested and then injected in the organ that requires the repair. For example stem cells from the bone marrow after harvesting are injected into failing heart helps replenish its lost properties. Stem cells have helped in curing leukemia, Parkinson’s disease, lymphoma, cancers and other rare ailments.
o Another repair and replacement technique is the gene therapy. Genetics have learnt to cut, splice and insert genes using the genetic scissors and genetic glue. Genetic scissors are the enzyme endonuclease and glue is the enzyme ligase which is capable of artificial synthesis in the laboratory.
o The defective gene is cut using endonuclease and removed. Simultaneously the counterpart gene functioning normally is extracted in the similar fashion from another organism particularly from bacterium E.coli. The normal gene is inserted in the diseased organ and fixed using ligase. Soon the normal gene is integrated in the genome which now behaves as normal. The major bonus with this technology is that the incurable genetic disease being transcending hereditarily is cured for ever. The next generation will receive the customary gene and shall be disease free.
o Gene replacement has helped cure diabetes, color blindness, severe combined immunodeficiency (SCID), cosmic enhancement, anti-aging and several such times programmed by genetic coding and timing. Gene and stem cell therapy are the modern elixirs to eternal youth and facilitators to a disease free-ageless body.
Article Source: http://EzineArticles.com/?expert=Rosy_Vohra
Tags: amino acids, body mechanisms, mature cells, natural aging process, nerve cell, radioactive components, stem cell research, stem cells, structure of protein, ultra violet raysRelated posts
Breast Cancer Gene Therapy – How Does it Work?
Scientists have begun to explore gene therapy as a possible candidate for breast cancer treatments. To give you a clue of what this therapy might be, it’s basically the delivery of gene(s) to cancer cells in the body. Injected genes have a direct effect on changing the malign cell behaviour.
The therapeutic genes as they are called are delivered to cancerous cells via vectors (or carrier vehicles). Think of them as computer programmers, who reprogram the bad cells. They basically deliver therapeutic healthy genes to cancerous tissue. This is as close to curing the cause of the malign tumour as modern medicine can get.
However, the greatest constraint in this therapy in general and specifically for breast cancer is an optimal delivery vector. There is extensive research seeking to optimize therapeutic gene delivery to cancerous cells combined with other different available therapies. It is hoped that gene therapy will limit the toxic side effects for the patient undergoing other treatments.
Gene therapy for breast cancer definitely has a big potential, even for today. Either when used in conjunction with another or as a sole form of treatment it can already be applied today. The success rate percentage data is hard to find from good sources, but this therapy is definitely worth of your attention. Through this treatment it is possible to explicitly target cancerous cells only and attempt to kill them. Also, gene carriers will continuously travel in the blood to seek cancer cells.
Genes play a central role in the breast cancer development. Statistics show that hereditary breast cancer makes up approximately 5% to 10% of all types. Some scientists believe that gene therapy is the most effective means of treating this type of cancer and it may accomplish more desirable results than any other available treatment.
Article Source: http://EzineArticles.com/?expert=John_M_Marshall
Tags: breast cancer, breast cancer treatments, cancer cells, cancer development, cancerous cells, carrier vehicles, gene therapy for breast cancer, hereditary breast cancer, therapeutic genes, toxic side effects