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...

Ful tanımlama

Kaydedildi:
Detaylı Bibliyografya
Yayımlandı:Br J Pharmacol
Asıl Yazarlar: Parra‐Guillen, Zinnia P., Fontanellas, Antonio, Jiang, Lei, Jericó, Daniel, Martini, Paolo, Vera‐Yunca, Diego, Hard, Marjie, Guey, Lin T., Troconiz, Iñaki F.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: John Wiley and Sons Inc. 2020
Konular:
Research Papers
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC7312321/
https://ncbi.nlm.nih.gov/pubmed/32133631
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/bph.15040
Etiketler: Etiketle
Etiket eklenmemiş, İlk siz ekleyin!

Benzer Materyaller