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Article

mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks

19 May 2021

Abstract excerpt

Variegate porphyria (VP) results from haploinsufficiency of protoporphyrinogen oxidase (PPOX), the seventh enzyme in the heme synthesis pathway. There is no VP model that recapitulates the clinical manifestations of acute attacks. Combined administrations of 2-allyl-2-isopropylacetamide and rifampicin in rabbits halved hepatic PPOX activity, resulting in increased accumulation of a potentially neurotoxic heme...

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Share to Heme Oxygenase-1 and Carbon Monoxide
Share to Methemoglobinemia and Tumor Lysis Syndrome
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 34458006
DOI: 10.1016/j.omtn.2021.05.010

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