Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model

Crigler-Najjar syndrome is a rare disorder of bilirubin metabolism caused by uridine diphosphate glucuronosyl transferase 1A1 (UGT1A1) mutations characterized by hyperbilirubinemia and jaundice. No cure currently exists; treatment options are limited to phototherapy, whose effectiveness diminishes o...

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Principais autores:	Jenny A. Greig, Joanna K. Chorazeczewski, Vivek Chowdhary, Melanie K. Smith, Matthew Jennis, James C. Tarrant, Elizabeth L. Buza, Kimberly Coughlan, Paolo G.V. Martini, James M. Wilson
Format:	Artigo
Jezik:	Inglês
Izdano:	Elsevier 2023-06-01
Serija:	Molecular Therapy: Methods & Clinical Development
Teme:	Crigler-Najjar lipid nanoparticle LNP mRNA UGT1A1 hyperbilirubinemia
Online dostop:	http://www.sciencedirect.com/science/article/pii/S2329050123000177
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