Disease pharmacokinetic–pharmacodynamic modelling in acute intermittent porphyria to support the development of mRNA‐based therapies

Background and Purpose Acute intermittent porphyria (AIP) results from haplo‐insufficiency of the porphobilinogen deaminase (PBGD) gene encoding the third enzyme in the haem biosynthesis pathway. As liver is the main organ of pathology for AIP, emerging therapies that restore enzyme hepatic levels a...

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Veröffentlicht in:	British journal of pharmacology 2020-07, Vol.177 (14), p.3168-3182
Hauptverfasser:	Parra‐Guillen, Zinnia P., Fontanellas, Antonio, Jiang, Lei, Jericó, Daniel, Martini, Paolo, Vera‐Yunca, Diego, Hard, Marjie, Guey, Lin T., Troconiz, Iñaki F.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animal models Animals Dosage Drug development Enzymes Heme Hydroxymethylbilane synthase Hydroxymethylbilane Synthase - genetics Liver Mathematical models Mice mRNA Nanoparticles Neurotoxicity Pharmacodynamics Pharmacokinetics Porphyria Porphyria, Acute Intermittent - drug therapy Porphyria, Acute Intermittent - genetics Rabbits Rats Research Paper Research Papers RNA, Messenger
Online-Zugang:	Volltext
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