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Tay-Sachs, Canavan, GM1 and Sandhoff diseases


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Term Definition
Lentivirus (lentiviral)

Lentivirus represents a genus of slow viruses with long incubation period (months, even years) and a propensity to induce a wide range of pathologies in different animal species. Due to their rather flexible genome and a potential of transducing many forms of nondividing cells, lentiviruses have become one of the most widely used vectors for gene transfer. (http://www.news-medical.net/life-sciences/What-is-Lentivirus.aspx)

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A group of rare genetic disorders that affect the central nervous system by disrupting the growth or maintenance of the myelin sheath that insulates nerve cells. Myelin is commonly referred to as the brain's "white matter", myelin covers nerve cells and ensures the clear transmission of nerve impulses from one part of the body to another. These disorders are progressive, meaning that they tend to get worse throughout the life of the patient.

The word leukodystrophy comes from the Greek words leuko (meaning white), trophy (meaning growth), and dys (meaning ill). If you put these words together, the word leukodystrophy describes a set of diseases that affect the growth or maintenance of the white matter (myelin).

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Linkage analysis

Linkage is the tendency for genes and other genetic markers to be inherited together because of their location near one another on the same chromosomes. Linkage analysis uses the location relationship to determine carrier status of particular genes.

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Large and complicated reddish-brown glandular organ located in the upper right portion of the abdominal cavity; secretes bile and functions in metabolism of protein and carbohydrate and fat; synthesizes substances involved in the clotting of the blood; synthesizes vitamin A; detoxifies poisonous substances and breaks down worn-out red blood cells. Definitions from: WordNet®

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Lorenzo's Oil

Is a combination of two fats extracted from olive oil and rapeseed oil and is specific to adrenoleukodystrophy (ALD), does not repair myelin, and does not have any known effect on other demyelinating disorders. In ALD presymptomatic boys Lorenzo's Oil often (but not always) prevents the onset of the disease by stopping the body from producing the very long chain fatty acids, whose buildup leads to demyelination. For more information visit the Myelin Project.

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Lysosomal storage disease

Lysosomal storage disease is a group of disorders that affect specific enzymes. These enzymes normally break down items for reuse in the cells. If the enzymes are missing or don't work properly the items can build up and become toxic.

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Synonyms - LSD

Organelle structures found within the cytoplasm of cells which contain digestive enzymes; responsible for breaking down the cell of debris into reusable parts.

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