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Engineered tRNAs suppress nonsense mutations in cells and in vivo.
Nature. 2023 Jun;618(7966):842-848. doi: 10.1038/s41586-023-06133-1. Epub 2023 May 31.
Nature. 2023.
PMID: 37258671
Free PMC article.
Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model.
Diaz-Trelles R, Lee S, Kuakini K, Park J, Dukanovic A, Gonzalez JA, Dam T, Kim JH, Vega JB, Sablad M, Karmali PP, Tachikawa K, Chivukula P.
Diaz-Trelles R, et al. Among authors: kuakini k.
Mol Genet Metab Rep. 2022 May 14;32:100882. doi: 10.1016/j.ymgmr.2022.100882. eCollection 2022 Sep.
Mol Genet Metab Rep. 2022.
PMID: 35600090
Free PMC article.
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