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Year | Number of Results |
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2023 | 3 |
2024 | 1 |
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A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant.
HGG Adv. 2024 Jan 11;5(1):100253. doi: 10.1016/j.xhgg.2023.100253. Epub 2023 Nov 2.
HGG Adv. 2024.
PMID: 37922902
Free PMC article.
Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need.
Brooks DL, Whittaker MN, Qu P, Musunuru K, Ahrens-Nicklas RC, Wang X.
Brooks DL, et al.
Am J Hum Genet. 2023 Dec 7;110(12):2003-2014. doi: 10.1016/j.ajhg.2023.10.005. Epub 2023 Nov 3.
Am J Hum Genet. 2023.
PMID: 37924808
Free PMC article.
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Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing.
Brooks DL, Carrasco MJ, Qu P, Peranteau WH, Ahrens-Nicklas RC, Musunuru K, Alameh MG, Wang X.
Brooks DL, et al.
Nat Commun. 2023 Jun 10;14(1):3451. doi: 10.1038/s41467-023-39246-2.
Nat Commun. 2023.
PMID: 37301931
Free PMC article.
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