The current results show that one can exploit the normal regulation of receptor synthesis to stimulate the single normal gene in FH heterozygotes to produce an increased number of LDL receptors. This stimulation can be achieved by drugs that inhibit HMG CoA reductase in the liver and by maneuvers that cause bile acid depletion. These two therapeutic approaches are most effective when they are combined. It seems reasonable to speculate that such a profound lowering of plasma cholesterol levels will minimize the development of atherosclerosis in FH heterozygotes. In a broader sense, the success of this regulatory manipulation raises the possibility that other genetic diseases may be treated through manipulation of regulatory signals that control the rates of synthesis of gene products.
|Original language||English (US)|
|Number of pages||9|
|Journal||Transactions of the Association of American Physicians|
|State||Published - 1983|
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