Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia

Classic galactosemia (CG) is a potentially lethal inborn error of galactose metabolism that results from deleterious mutations in the human galactose-1 phosphate uridylyltransferase (GALT) gene. Previously, we constructed a GalT(−/−) (GalT-deficient) mouse model that exhibits galactose sensitivity i...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Balakrishnan, Bijina, An, Ding, Nguyen, Vi, DeAntonis, Christine, Martini, Paolo G.V., Lai, Kent
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6952165/
https://ncbi.nlm.nih.gov/pubmed/31604675
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.09.018
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