The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism

INTRODUCTION: Inborn errors of metabolism (IEMs) often result from single-gene mutations and collectively cause liver dysfunction in neonates leading to chronic liver and systemic disease. Current treatments for many IEMs are limited to maintenance therapies that may still require orthotropic liver...

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Detalles Bibliográficos
Autores principales: Thompson, Whitney S., Mondal, Gourish, Vanlith, Caitlin J., Kaiser, Robert A., Lillegard, Joseph B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7676758/
https://www.ncbi.nlm.nih.gov/pubmed/33224636
http://dx.doi.org/10.1080/21678707.2020.1791082

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