mRNA-based therapy proves superior to the standard of care for treating hereditary tyrosinemia 1 in a mouse model

Hereditary tyrosinemia type 1 is an inborn error of amino acid metabolism characterized by deficiency of fumarylacetoacetate hydrolase (FAH). Only limited treatment options (e.g., oral nitisinone) are available. Patients must adhere to a strict diet and face a life-long risk of complications, includ...

Descripción completa

Detalles Bibliográficos
Autores principales: Cacicedo, Maximiliano L., Weinl-Tenbruck, Christine, Frank, Daniel, Wirsching, Sebastian, Straub, Beate K., Hauke, Jana, Okun, Jürgen G., Horscroft, Nigel, Hennermann, Julia B., Zepp, Fred, Chevessier-Tünnesen, Frédéric, Gehring, Stephan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9357842/
https://www.ncbi.nlm.nih.gov/pubmed/35949297
http://dx.doi.org/10.1016/j.omtm.2022.07.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9357842/
https://www.ncbi.nlm.nih.gov/pubmed/35949297
http://dx.doi.org/10.1016/j.omtm.2022.07.006

Ejemplares similares