Mechanistic modelling of enzyme‐restoration effects of new recombinant liver‐targeted proteins in acute intermittent porphyria-Reference-Cited by-同舟云学术

Mechanistic modelling of enzyme‐restoration effects of new recombinant liver‐targeted proteins in acute intermittent porphyria

Published:2022-03-21 Issue:14 Volume:179 Page:3815-3830
ISSN:0007-1188
Container-title:British Journal of Pharmacology
language:en
Short-container-title:British J Pharmacology

Author:

Vera‐Yunca Diego¹²^ORCID,Córdoba Karol M.²³,Parra‐Guillen Zinnia P.¹²^ORCID,Jericó Daniel²³,Fontanellas Antonio²³⁴,Trocóniz Iñaki F.¹²^ORCID

Affiliation:

1. Pharmacometrics & Systems Pharmacology, Department of Pharmaceutical Technology and Chemistry, School of Pharmacy and Nutrition University of Navarra Pamplona Spain

2. IdiSNA, Navarra Institute for Health Research Pamplona Spain

3. Hepatology Program, Center for Applied Medical Research (CIMA) University of Navarra Pamplona Spain

4. Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBERehd), Instituto de Salud Carlos III Madrid Spain

Funder

Instituto de Salud Carlos III

Ministerio de Educación y Formación Profesional

Publisher

Wiley

Subject

Pharmacology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/bph.15821

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4. Acute Intermittent Porphyria: Predicted Pathogenicity ofHMBSVariants Indicates Extremely Low Penetrance of the Autosomal Dominant Disease

5. Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria