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Phenylalanine hydroxylase mRNA rescues the phenylketonuria phenotype in mice.
Front Bioeng Biotechnol. 2022 Oct 7;10:993298. doi: 10.3389/fbioe.2022.993298. eCollection 2022.
Front Bioeng Biotechnol. 2022.
PMID: 36277393
Free PMC article.
mRNA-based therapy proves superior to the standard of care for treating hereditary tyrosinemia 1 in a mouse model.
Cacicedo ML, Weinl-Tenbruck C, Frank D, Wirsching S, Straub BK, Hauke J, Okun JG, Horscroft N, Hennermann JB, Zepp F, Chevessier-Tünnesen F, Gehring S.
Cacicedo ML, et al. Among authors: chevessier tunnesen f.
Mol Ther Methods Clin Dev. 2022 Jul 15;26:294-308. doi: 10.1016/j.omtm.2022.07.006. eCollection 2022 Sep 8.
Mol Ther Methods Clin Dev. 2022.
PMID: 35949297
Free PMC article.
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mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5.
Hauser S, Poenisch M, Schelling Y, Höflinger P, Schuster S, Teegler A, Betten R, Gustafsson JÅ, Hübener-Schmid J, Schlake T, Chevessier-Tünnesen F, Horscroft N, Björkhem I, Schöls L.
Hauser S, et al. Among authors: chevessier tunnesen f.
Mol Ther Methods Clin Dev. 2019 Oct 31;15:359-370. doi: 10.1016/j.omtm.2019.10.011. eCollection 2019 Dec 13.
Mol Ther Methods Clin Dev. 2019.
PMID: 31828178
Free PMC article.
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