Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model
Jenny A. Greig, Joanna K Chorazeczewski, Vivek Chowdhary, Melanie K. Smith, Matthew Jennis, James C. Tarrant, Elizabeth L. Buza, Kimberly A. Coughlan, Paolo G.V. Martini, James M. Wilson
Abstract
mRNA may represent a potential treatment for Crigler-Najjar syndrome.
Topics & Concepts
GlucuronosyltransferaseBilirubinPharmacologyKnockout mouseEndocrinologyInternal medicineMedicineJaundiceChemistryEnzymeBiochemistryMicrosomeReceptorNeonatal Health and BiochemistryHeme Oxygenase-1 and Carbon MonoxideErythrocyte Function and Pathophysiology