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Phenylalanine hydroxylase mRNA rescues the phenylketonuria phenotype in mice

Phenylketonuria (PKU) is an inborn error of metabolism caused by a deficiency in functional phenylalanine hydroxylase (PAH), resulting in accumulation of phenylalanine (Phe) in patients’ blood and organs. Affected patients encounter severe developmental delay, neurological deficits, and behavioral a...

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Tallennettuna:
Päätekijät: Maximiliano L. Cacicedo, Christine Weinl-Tenbruck, Daniel Frank, Maria Jose Limeres, Sebastian Wirsching, Katja Hilbert, Mansure Abdollah Pasha Famian, Nigel Horscroft, Julia B. Hennermann, Fred Zepp, Frédéric Chevessier-Tünnesen, Stephan Gehring
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Frontiers Media S.A. 2022-10-01
Sarja:Frontiers in Bioengineering and Biotechnology
Aiheet:
phenylketonuria (PKU)
phenylalanine hydroxylase (pah)
metabolic diseases
mRNA-based therapy
sapropterin
Linkit:https://www.frontiersin.org/articles/10.3389/fbioe.2022.993298/full
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