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Adenine base editing in an adult mouse model of tyrosinaemia.
Song CQ, Jiang T, Richter M, Rhym LH, Koblan LW, Zafra MP, Schatoff EM, Doman JL, Cao Y, Dow LE, Zhu LJ, Anderson DG, Liu DR, Yin H, Xue W. Song CQ, et al. Among authors: rhym lh. Nat Biomed Eng. 2020 Jan;4(1):125-130. doi: 10.1038/s41551-019-0357-8. Epub 2019 Feb 25. Nat Biomed Eng. 2020. PMID: 31740768 Free PMC article.
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing.
Yin H, Song CQ, Suresh S, Wu Q, Walsh S, Rhym LH, Mintzer E, Bolukbasi MF, Zhu LJ, Kauffman K, Mou H, Oberholzer A, Ding J, Kwan SY, Bogorad RL, Zatsepin T, Koteliansky V, Wolfe SA, Xue W, Langer R, Anderson DG. Yin H, et al. Among authors: rhym lh. Nat Biotechnol. 2017 Dec;35(12):1179-1187. doi: 10.1038/nbt.4005. Epub 2017 Nov 13. Nat Biotechnol. 2017. PMID: 29131148 Free PMC article.
Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs.
Fenton OS, Kauffman KJ, McClellan RL, Kaczmarek JC, Zeng MD, Andresen JL, Rhym LH, Heartlein MW, DeRosa F, Anderson DG. Fenton OS, et al. Among authors: rhym lh. Angew Chem Int Ed Engl. 2018 Oct 8;57(41):13582-13586. doi: 10.1002/anie.201809056. Epub 2018 Sep 14. Angew Chem Int Ed Engl. 2018. PMID: 30112821 Free PMC article.