RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

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-aminolev...

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Bibliographische Detailangaben
Hauptverfasser: Yasuda, Makiko, Gan, Lin, Chen, Brenden, Kadirvel, Senkottuvelan, Yu, Chunli, Phillips, John D., New, Maria I., Liebow, Abigail, Fitzgerald, Kevin, Querbes, William, Desnick, Robert J.
Format: Artigo
Sprache:Inglês
Veröffentlicht: National Academy of Sciences 2014
Schlagworte:
Biological Sciences
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4040563/
https://ncbi.nlm.nih.gov/pubmed/24821812
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1406228111
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