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Quantitative Systems Pharmacology Model of hUGT1A1‐modRNA Encoding for the UGT1A1 Enzyme to Treat Crigler‐Najjar Syndrome Type 1

Crigler‐Najjar syndrome type 1 (CN1) is an autosomal recessive disease caused by a marked decrease in uridine‐diphosphate‐glucuronosyltransferase (UGT1A1) enzyme activity. Delivery of hUGT1A1‐modRNA (a modified messenger RNA encoding for UGT1A1) as a lipid nanoparticle is anticipated to restore hepa...

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Detalles Bibliográficos
Autores principales:	Apgar, Joshua F., Tang, Jian‐Ping, Singh, Pratap, Balasubramanian, Nanda, Burke, John, Hodges, Michael R., Lasaro, Melissa A., Lin, Lin, Miliard, Bjorn L., Moore, Kristi, Jun, Lucy S., Sobolov, Susan, Wilkins, Anna Katharina, Gao, Xiang
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2018
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6391595/ https://www.ncbi.nlm.nih.gov/pubmed/29637732 http://dx.doi.org/10.1002/psp4.12301

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