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Example research essay topic: Current Research On Muscular Dystrophy - 576 words

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Duchenne's muscular dystrophy is the result of a defective gene on the X chromosome. This gene is responsible for production of the muscle protein dystrophic. Dystrophin is an integral part of the dystrophic-glycoprotein complex which bears the brunt of the force generated during muscular contraction. When dystrophic is not produced, the dystrophic-glycoprotein complex (DCG) is not present. Absence of the DCG leads to tears in the muscle membrane because the muscle membrane bears the force of muscular contraction alone.

Tears in the muscle membrane allow substances to leak in and out of the muscle fibers at random. This uncontrolled "biochemical traffic" leads to eventual death of the muscle fibers. Most of the current research on Duchenne's muscular dystrophy involves gene therapy. Researchers are attempting to find ways to introduce a healthy dystrophic gene into the afflicted individual. This healthy gene would produce the dystrophic protein thereby regenerating the DGC, which would in turn curb muscle fiber death. Studies with mice have shown that introduction of the dystrophic gene is effective in treating Duchenne's muscular dystrophy.

However, introduction of the dystrophic gene into the body is no easy task. Thus, many scientists are focusing their research on ways to present Viruses have a natural inclination to deposit their genetic material in a cell's nucleus and thus are primary candidates for gene transport. The dystrophic gene is a relatively large gene and therefore must be delivered via an adenovirus. The problem with viral delivery is that the immune system of the recipient recognizes the virus as foreign and destroys both the virus and the protein it is carrying. Researchers at the University of Michigan-Ann Arbor have developed an aden virus that is "gutted" of its own genetic material and consists only of a viral shell. These "gutted" adenoviruses elicit fewer immune responses.

However, it is believed that immunosuppressant drugs, such as FK 506 may be necessary to fully overcome the immune response to adenovirus-based gene All current gene based research has been performed on animals, but this fall, investigators at the University of Ohio-Columbus and the University of Michigan-Ann Arbor will begin a very limited human trial of gene therapy in Duchenne's muscular dystrophy. The major goal of the 24 week study is to establish the safety of the gene transfer procedure. The study involves 12 participants with Duchenne's muscular dystrophy and is waiting for final approval from the Food and Drug Administration. Another focus of research on Duchenne's muscular dystrophy involves the protein Utrophin.

Utrophin is almost exactly like dystrophic, and its potential as a replacement for dystrophic has stirred much interest. Utrophin genes could be introduced into the body via an adenovirus (described above) and "fill in" for the missing dystrophic protein. The major advantage of utrophin over dystrophic is that individuals with the disorder already make utrophin, so their immune systems would accept the protein and not reject it as foreign. Utrophin is coded for on chromosome 6 and is thus unaffected by the defective X chromosome.

Therefore, another method of increasing utrophin would be to manipulate the utrophin genes already present in the muscle fibers to produce more. Utrophin is normally found only at the neuromuscular junction, but to be effective, it must completely surround the muscle fiber. Researchers have found that during fetal life, humans exhibit utrophin around the entire muscle fiber, but as development progresses, the utrophin is replaced with dystrophic. Investigators hope to find the "switch" that creates this change Bibliography:


Free research essays on topics related to: ann arbor, muscle fibers, gene therapy, university of michigan, duchenne muscular dystrophy

Research essay sample on Current Research On Muscular Dystrophy

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