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Journal of Hepatology|December 19, 2020
Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4-/- mouse model of PFIC3Guangyan Wei, Jingsong Cao, Pinzhu Huang, et al.American Journal of Human Genetics|August 22, 2023
GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individualsMenno Ter Huurne, Benjamin L Parker, Ning Qing Liu, et al.Science Translational Medicine|January 10, 2024
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduriaSonam Gurung, Oskar Vilhelmsson Timmermand, Dany Perocheau, et al.Nature Communications|May 26, 2021
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage diseaseJingsong Cao, Minjung Choi, Eleonora Guadagnin, et al.Journal of Inherited Metabolic Disease|December 4, 2023
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduriaSonam Gurung, Saketh Karamched, Dany Perocheau, et al.Autophagy|January 12, 2008
Guidelines for the use and interpretation of assays for monitoring autophagy in higher eukaryotesDaniel J Klionsky, Hagai Abeliovich, Patrizia Agostinis, et al.Pageof 3