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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Detalles Bibliográficos
Autores principales: Greig, Jenny A., Chorazeczewski, Joanna K., Chowdhary, Vivek, Smith, Melanie K., Jennis, Matthew, Tarrant, James C., Buza, Elizabeth L., Coughlan, Kimberly, Martini, Paolo G.V., Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10017950/
https://www.ncbi.nlm.nih.gov/pubmed/36936447
http://dx.doi.org/10.1016/j.omtm.2023.02.007

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10017950/
https://www.ncbi.nlm.nih.gov/pubmed/36936447
http://dx.doi.org/10.1016/j.omtm.2023.02.007

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