Article
RNAi-mediated silencing of hepatic <i>Alas1</i> effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice
12 May 2014
Abstract excerpt
The acute hepatic porphyrias are inherited disorders of heme biosynthesis characterized by life-threatening acute neurovisceral attacks. Factors that induce the expression of hepatic 5-aminolevulinic acid synthase 1 (ALAS1) result in the accumulation of the neurotoxic porphyrin precursors 5-aminolevulinic acid (ALA) and porphobilinogen (PBG), which recent studies indicate are primarily responsible for the acute...
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