In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

Conventional therapy for hereditary tyrosinemia type-1 (HT1) with 2-(2-nitro-4-trifluoromethylbenzoyl)−1,3-cyclohexanedione (NTBC) delays and in some cases fails to prevent disease progression to liver fibrosis, liver failure, and activation of tumorigenic pathways. Here we demonstrate cure of HT1 b...

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Detalles Bibliográficos
Autores principales: Nicolas, Clara T., VanLith, Caitlin J., Hickey, Raymond D., Du, Zeji, Hillin, Lori G., Guthman, Rebekah M., Cao, William J., Haugo, Benjamin, Lillegard, Annika, Roy, Diya, Bhagwate, Aditya, O’Brien, Daniel, Kocher, Jean-Pierre, Kaiser, Robert A., Russell, Stephen J., Lillegard, Joseph B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9411607/
https://www.ncbi.nlm.nih.gov/pubmed/36008405
http://dx.doi.org/10.1038/s41467-022-32576-7

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