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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo.
Yin H, Song CQ, Dorkin JR, Zhu LJ, Li Y, Wu Q, Park A, Yang J, Suresh S, Bizhanova A, Gupta A, Bolukbasi MF, Walsh S, Bogorad RL, Gao G, Weng Z, Dong Y, Koteliansky V, Wolfe SA, Langer R, Xue W, Anderson DG. Yin H, et al. Among authors: koteliansky v. Nat Biotechnol. 2016 Mar;34(3):328-33. doi: 10.1038/nbt.3471. Epub 2016 Feb 1. Nat Biotechnol. 2016. PMID: 26829318 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: koteliansky v. 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.
Partial DNA-guided Cas9 enables genome editing with reduced off-target activity.
Yin H, Song CQ, Suresh S, Kwan SY, Wu Q, Walsh S, Ding J, Bogorad RL, Zhu LJ, Wolfe SA, Koteliansky V, Xue W, Langer R, Anderson DG. Yin H, et al. Among authors: koteliansky v. Nat Chem Biol. 2018 Mar;14(3):311-316. doi: 10.1038/nchembio.2559. Epub 2018 Jan 29. Nat Chem Biol. 2018. PMID: 29377001 Free PMC article.
A combinatorial library of lipid-like materials for delivery of RNAi therapeutics.
Akinc A, Zumbuehl A, Goldberg M, Leshchiner ES, Busini V, Hossain N, Bacallado SA, Nguyen DN, Fuller J, Alvarez R, Borodovsky A, Borland T, Constien R, de Fougerolles A, Dorkin JR, Narayanannair Jayaprakash K, Jayaraman M, John M, Koteliansky V, Manoharan M, Nechev L, Qin J, Racie T, Raitcheva D, Rajeev KG, Sah DW, Soutschek J, Toudjarska I, Vornlocher HP, Zimmermann TS, Langer R, Anderson DG. Akinc A, et al. Among authors: koteliansky v. Nat Biotechnol. 2008 May;26(5):561-9. doi: 10.1038/nbt1402. Epub 2008 Apr 27. Nat Biotechnol. 2008. PMID: 18438401 Free PMC article.
Therapeutic siRNA silencing in inflammatory monocytes in mice.
Leuschner F, Dutta P, Gorbatov R, Novobrantseva TI, Donahoe JS, Courties G, Lee KM, Kim JI, Markmann JF, Marinelli B, Panizzi P, Lee WW, Iwamoto Y, Milstein S, Epstein-Barash H, Cantley W, Wong J, Cortez-Retamozo V, Newton A, Love K, Libby P, Pittet MJ, Swirski FK, Koteliansky V, Langer R, Weissleder R, Anderson DG, Nahrendorf M. Leuschner F, et al. Among authors: koteliansky v. Nat Biotechnol. 2011 Oct 9;29(11):1005-10. doi: 10.1038/nbt.1989. Nat Biotechnol. 2011. PMID: 21983520 Free PMC article.
Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.
Dong Y, Love KT, Dorkin JR, Sirirungruang S, Zhang Y, Chen D, Bogorad RL, Yin H, Chen Y, Vegas AJ, Alabi CA, Sahay G, Olejnik KT, Wang W, Schroeder A, Lytton-Jean AK, Siegwart DJ, Akinc A, Barnes C, Barros SA, Carioto M, Fitzgerald K, Hettinger J, Kumar V, Novobrantseva TI, Qin J, Querbes W, Koteliansky V, Langer R, Anderson DG. Dong Y, et al. Among authors: koteliansky v. Proc Natl Acad Sci U S A. 2014 Mar 18;111(11):3955-60. doi: 10.1073/pnas.1322937111. Epub 2014 Feb 10. Proc Natl Acad Sci U S A. 2014. PMID: 24516150 Free PMC article.
205 results