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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Veröffentlicht in:	Proceedings of the National Academy of Sciences - PNAS Jg. 111; H. 21; S. 7777
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:	Journal Article
Sprache:	Englisch
Veröffentlicht:	United States 27.05.2014
Schlagworte:	5-Aminolevulinate Synthetase - genetics 5-Aminolevulinate Synthetase - metabolism Analysis of Variance Animals Blotting, Western Drug Evaluation, Preclinical Electrophoresis, Polyacrylamide Gel Female Liver - metabolism Mice Mice, Inbred C57BL Particle Size Porphyria, Acute Intermittent - prevention & control Real-Time Polymerase Chain Reaction RNA Interference - drug effects RNA Interference - immunology RNA, Small Interfering - pharmacology RNAi therapeutics heme biosynthetic disorders liver-targeted siRNA
ISSN:	1091-6490, 1091-6490
Online-Zugang:	Weitere Angaben
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