Another alternative strategy to gene therapy is exon skipping which differs from gene therapy in that it focuses on the gene transcript rather than the gene itself (McClorey et al., 2005). Exon skipping prevents the transcription of the exon containing the mutation. Exon skipping is a process by which synthetic DNA molecules, antisense, are utilized to create a bridge by which the dysfunctional parts of the gene can be skipped over (Partridge, 2010). This intervention has been found to be particularly useful when treating Duchenne MD in laboratory animals and experts are hoping that this will correlate to human subjects as well.
Cell therapy has also shown promise in the treatment of MD. In this approach cells can be taken either from a donor that has healthy formations of the mutated gene or by utilizing repaired cells of the individual with MD (Cossu and Sampaolesi, 2004). The use of the individuals own genes has been made possible through the cloning process and these genes can then be introduced into the system of the MD patient. Recent studies have shown an 11% improvement in the positive muscle fibers surrounding the area of injection (Cossu and Sampaolesi, 2004). This approach is not without its challenges as well including the availability of donor cells as well as the failure of the donor cells to disperse throughout the muscle resulting in a concentration of cells in a particular area. Attempts to overcome this barrier have utilized stem cells and cells taken from bone marrow which have been proven to have regenerating factors in skeletal muscles (Cossu and Sampaolesi, 2004).
Pharmacological interventions have also shown promise in the treatment of MD these medications have primarily been utilized to stop the progression of the dystrophic process and have taken the form of protease inhibitors, anti-inflammatory drugs, calcium blockers and drugs that impact the metabolism of proteins and lipids (Cossu and Sampaolesi, 2004). Despite ongoing research, pharmacological interventions have not proven to be particularly effective in the reduction of symptomolgy producing only modest results in clinical trials (Cossu and Sampaolesi, 2004). However, there is data to suggest that the use of corticosteroid treatments can reduce the loss of ambulation by 2 to 4 years and will decrease the risk of the development of skeletal defects as well as slowing the onset of respiratory and cardiac issues (Cossu and Sampaolesi, 2004). Yet one must also...
Physical activity is key in slowing the progressive nature of the disease since inactivity will encourage quicker deterioration of the muscle tissue. Beyond the individual maintaining an effort of physical activity, various treatments, such as physical therapy, speech therapy, respiratory therapy, and the use of wheelchairs, canes, walkers, or braces may be needed. Orthopedic surgery to correct damage done to the bones due to the deterioration of the muscle
Muscular Dystrophy. The writer explores the types, the causes, the symptoms and the research to find a cure. There were six sources used to complete this paper. One would be hard pressed to find an American who has never witnessed the Jerry Lewis telethon for Muscular Dystrophy. It is an annual Labor Day tradition and has been for many decades. While everybody is aware that Lewis does the telethon and
The success was remarkable, according to the researchers: Even muscles that had already lost half of its mass, recovered visible. (Leppanen et al. p5549-65) At the same time, the mice survived for several weeks longer than their untreated counterparts and also developed a healthy appetite again. (Mantovani, p296) The new study is therefore interesting in two respects: First, it demonstrates that the muscle loss at least in animal models in
Researchers at Cornell University discovered that Monarch butterfly caterpillars died when they ate plants dusted with the pollen of Bt corn that was growing in nearby fields, and many scientists worry that with so much insecticide in the corn plants, insects might develop a resistance to it (Dyer 2002). These fears and concerns are echoed by Francis Fukuyama who believes that genetic enhancement will undermine the system of human
Understanding Scoliosis: A Common Spinal Condition Introduction Scoliosis is a common spinal condition characterized by an abnormal lateral curvature of the spine. This condition can affect individuals of all ages, but it is most commonly diagnosed during adolescence when the spine is rapidly growing. The curvature of the spine in individuals with scoliosis can appear as an "S" or "C" shape when viewed from the back. While the exact cause of scoliosis is
During the gene decoding process the double stranded DNA splits up to reveal a single strand from which the base sequence of the gene is copied onto a single stranded nucleic acid known as the messenger ribonucleic acid or mRNA. This implies that we have an exact copy of the gene base in the mRNA except that Urasil (U) replaces the T. base and deoxyribose is replaced by ribose.
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