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In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels.
Rothgangl T, Dennis MK, Lin PJC, Oka R, Witzigmann D, Villiger L, Qi W, Hruzova M, Kissling L, Lenggenhager D, Borrelli C, Egli S, Frey N, Bakker N, Walker JA 2nd, Kadina AP, Victorov DV, Pacesa M, Kreutzer S, Kontarakis Z, Moor A, Jinek M, Weissman D, Stoffel M, van Boxtel R, Holden K, Pardi N, Thöny B, Häberle J, Tam YK, Semple SC, Schwank G. Rothgangl T, et al. Among authors: semple sc. Nat Biotechnol. 2021 Aug;39(8):949-957. doi: 10.1038/s41587-021-00933-4. Epub 2021 May 19. Nat Biotechnol. 2021. PMID: 34012094 Free PMC article.
Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo.
Gorsuch CL, Nemec P, Yu M, Xu S, Han D, Smith J, Lape J, van Buuren N, Ramirez R, Muench RC, Holdorf MM, Feierbach B, Falls G, Holt J, Shoop W, Sevigny E, Karriker F, Brown RV, Joshi A, Goodwin T, Tam YK, Lin PJC, Semple SC, Leatherbury N, Delaney Iv WE, Jantz D, Rhoden Smith A. Gorsuch CL, et al. Among authors: semple sc. Mol Ther. 2022 Sep 7;30(9):2909-2922. doi: 10.1016/j.ymthe.2022.05.013. Epub 2022 May 16. Mol Ther. 2022. PMID: 35581938 Free PMC article.
The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs.
Akinc A, Maier MA, Manoharan M, Fitzgerald K, Jayaraman M, Barros S, Ansell S, Du X, Hope MJ, Madden TD, Mui BL, Semple SC, Tam YK, Ciufolini M, Witzigmann D, Kulkarni JA, van der Meel R, Cullis PR. Akinc A, et al. Among authors: semple sc. Nat Nanotechnol. 2019 Dec;14(12):1084-1087. doi: 10.1038/s41565-019-0591-y. Nat Nanotechnol. 2019. PMID: 31802031 No abstract available.
Rational design of cationic lipids for siRNA delivery.
Semple SC, Akinc A, Chen J, Sandhu AP, Mui BL, Cho CK, Sah DW, Stebbing D, Crosley EJ, Yaworski E, Hafez IM, Dorkin JR, Qin J, Lam K, Rajeev KG, Wong KF, Jeffs LB, Nechev L, Eisenhardt ML, Jayaraman M, Kazem M, Maier MA, Srinivasulu M, Weinstein MJ, Chen Q, Alvarez R, Barros SA, De S, Klimuk SK, Borland T, Kosovrasti V, Cantley WL, Tam YK, Manoharan M, Ciufolini MA, Tracy MA, de Fougerolles A, MacLachlan I, Cullis PR, Madden TD, Hope MJ. Semple SC, et al. Nat Biotechnol. 2010 Feb;28(2):172-6. doi: 10.1038/nbt.1602. Epub 2010 Jan 17. Nat Biotechnol. 2010. PMID: 20081866
35 results