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

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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Bibliographic Details
Main Authors: Maximiliano L. Cacicedo, Christine Weinl-Tenbruck, Daniel Frank, Sebastian Wirsching, Beate K. Straub, Jana Hauke, Jürgen G. Okun, Nigel Horscroft, Julia B. Hennermann, Fred Zepp, Frédéric Chevessier-Tünnesen, Stephan Gehring
Format: Article
Language:English
Published: Elsevier 2022-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hereditary tyrosinemia type 1
fumarylacetoacetate hydrolase
nitisinone supplementation
mRNA-based therapy
mouse model
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000985

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http://www.sciencedirect.com/science/article/pii/S2329050122000985

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