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Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
Pfister EL, DiNardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid SJ, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N. Pfister EL, et al. Among authors: liu p. Hum Gene Ther. 2018 Jun;29(6):663-673. doi: 10.1089/hum.2017.199. Epub 2018 Feb 23. Hum Gene Ther. 2018. PMID: 29207890 Free PMC article.
Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector.
Zhang H, Bamidele N, Liu P, Ojelabi O, Gao XD, Rodriguez T, Cheng H, Kelly K, Watts JK, Xie J, Gao G, Wolfe SA, Xue W, Sontheimer EJ. Zhang H, et al. Among authors: liu p. GEN Biotechnol. 2022 Jun 1;1(3):285-299. doi: 10.1089/genbio.2022.0015. Epub 2022 Jun 14. GEN Biotechnol. 2022. PMID: 35811581 Free PMC article.
5'-Modifications improve potency and efficacy of DNA donors for precision genome editing.
Ghanta KS, Chen Z, Mir A, Dokshin GA, Krishnamurthy PM, Yoon Y, Gallant J, Xu P, Zhang XO, Ozturk AR, Shin M, Idrizi F, Liu P, Gneid H, Edraki A, Lawson ND, Rivera-Pérez JA, Sontheimer EJ, Watts JK, Mello CC. Ghanta KS, et al. Among authors: liu p. Elife. 2021 Oct 19;10:e72216. doi: 10.7554/eLife.72216. Elife. 2021. PMID: 34665130 Free PMC article.
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