Novel mRNA therapy restores GALT protein and enzyme activity in a zebrafish model of classic galactosemia

Messenger RNA (mRNA) has emerged as a novel therapeutic approach for inborn errors of metabolism. Classic galactosemia (CG) is an inborn error of galactose metabolism caused by a severe deficiency of galactose‐1‐phosphate:uridylyltransferase (GALT) activity leading to neonatal illness and chronic im...

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Detalles Bibliográficos
Autores principales: Delnoy, Britt, Haskovic, Minela, Vanoevelen, Jo, Steinbusch, Laura K. M., Vos, Esther Naomi, Knoops, Kèvin, Zimmermann, Luc J. I., Noga, Marek, Lefeber, Dirk J., Martini, Paolo G. V., Coelho, Ana I., Rubio‐Gozalbo, Maria Estela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9541528/
https://www.ncbi.nlm.nih.gov/pubmed/35527402
http://dx.doi.org/10.1002/jimd.12512

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9541528/
https://www.ncbi.nlm.nih.gov/pubmed/35527402
http://dx.doi.org/10.1002/jimd.12512

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