Fatal Familial Insomnia Essay

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The Genetic Disorder Fatal Familial Insomnia

Fatal familial insomnia is a genetic disorder. It manifests itself by many symptoms due to the degeneration of a certain part of the brain, the thalamus. The disease also results in the formation of amyloid plaques. This is the build up of a waxy substance made of proteins associated with polysaccharides. The disease is a result of a mutation of a normal protein that is associated with brain tissue. This is the prion protein. In the case of fatal familial insomnia, the mutation occurs 178 amino acids into the normal protein. Were an asparagine should be, an aspartic acid is instead. This disease is an autosomal dominant, which means that both sexes are affected and there are no carriers. If an …show more content…

The first stage is progressive insomnia, the trade mark of fatal familial insomnia. The first stage develops over approximately four months and includes a collection of psychiatric problems such as panic attacks and bizarre phobias. The second stage includes hallucinations, panic, agitation and sweating and lasts about five months. The third stage lasts about three months and is total insomnia with weight loss. The individual at this point looks much older and may experience incontinence. The fourth stage is around six months long and is recognized as dementia, total insomnia and sudden death after becoming …show more content…

This treatment involves the insertion of the correct gene into an affected individual altering his/her gene expression making it what it should be for the expression of the correct protein. In order for this to happen, early diagnosis of an individual must be accomplished, possibly by the mentioned biotechniques above. This is so that the defective gene may be repaired before the onset of the disease. In order for this to be possible, the corrective gene must be isolated. Furthermore, the corrective gene must be good for transfer as well as a the proper vector to effectively execute the transfer. Because there is no cure for this illness, gene therapy may be the only answer if it is one day successful.
References

Guilleminault, C., Lugaresi, E., at el. Fatal Familial Insomnia: inherited prion diseases, sleep, and the thalamus. Raven Press, 1994. pp.15-20.

Klug, W.S., Cummings, M.R. Concepts of Genetics. Prentice-Hall, Inc., 1994. pp. 425-427.

Medori, R., Tritschler, H-J., at el. "Fatal Familial Insomnia, a Prion Disease with a
Mutation at Codon 178 of the Prion Protein Gene" New England Journal of
Medicine, 326 (7): 444-449 (1992).

Petersen, R.B., Tabaton, M., at el. "Analysis of the prion Protein gene in Thalamic
Dementia" Neurology, 42 (10): 1859-1863 (1992).

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