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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.
Rab5 is necessary for the biogenesis of the endolysosomal system in vivo.
Zeigerer A, Gilleron J, Bogorad RL, Marsico G, Nonaka H, Seifert S, Epstein-Barash H, Kuchimanchi S, Peng CG, Ruda VM, Del Conte-Zerial P, Hengstler JG, Kalaidzidis Y, Koteliansky V, Zerial M. Zeigerer A, et al. Among authors: koteliansky v. Nature. 2012 May 23;485(7399):465-70. doi: 10.1038/nature11133. Nature. 2012. PMID: 22622570
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.
Loss of α-catenin elicits a cholestatic response and impairs liver regeneration.
Herr KJ, Tsang YH, Ong JW, Li Q, Yap LL, Yu W, Yin H, Bogorad RL, Dahlman JE, Chan YG, Bay BH, Singaraja R, Anderson DG, Koteliansky V, Viasnoff V, Thiery JP. Herr KJ, et al. Among authors: koteliansky v. Sci Rep. 2014 Oct 30;4:6835. doi: 10.1038/srep06835. Sci Rep. 2014. PMID: 25355493 Free PMC article.
205 results