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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: weinl tenbruck c. 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.
Phenylalanine hydroxylase mRNA rescues the phenylketonuria phenotype in mice.
Cacicedo ML, Weinl-Tenbruck C, Frank D, Limeres MJ, Wirsching S, Hilbert K, Pasha Famian MA, Horscroft N, Hennermann JB, Zepp F, Chevessier-Tünnesen F, Gehring S. Cacicedo ML, et al. Among authors: weinl tenbruck c. 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.
An mRNA-encoded dominant-negative inhibitor of transcription factor RUNX1 suppresses vitreoretinal disease in experimental models.
O'Hare M, Miller WP, Arevalo-Alquichire S, Amarnani D, Apryani E, Perez-Corredor P, Marino C, Shu DY, Vanderleest TE, Muriel-Torres A, Gordon HB, Gunawan AL, Kaplan BA, Barake KW, Bejjani RP, Doan TH, Lin R, Delgado-Tirado S, Gonzalez-Buendia L, Rossin EJ, Zhao G, Eliott D, Weinl-Tenbruck C, Chevessier-Tünnesen F, Rejman J, Montrasio F, Kim LA, Arboleda-Velasquez JF. O'Hare M, et al. Among authors: weinl tenbruck c. Sci Transl Med. 2024 Nov 27;16(775):eadh0994. doi: 10.1126/scitranslmed.adh0994. Epub 2024 Nov 27. Sci Transl Med. 2024. PMID: 39602510