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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...

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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

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