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Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway.
John M, Constien R, Akinc A, Goldberg M, Moon YA, Spranger M, Hadwiger P, Soutschek J, Vornlocher HP, Manoharan M, Stoffel M, Langer R, Anderson DG, Horton JD, Koteliansky V, Bumcrot D. John M, et al. Among authors: bumcrot d. Nature. 2007 Oct 11;449(7163):745-7. doi: 10.1038/nature06179. Epub 2007 Sep 26. Nature. 2007. PMID: 17898712 Free PMC article.
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs.
Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Röhl I, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP. Soutschek J, et al. Among authors: bumcrot d. Nature. 2004 Nov 11;432(7014):173-8. doi: 10.1038/nature03121. Nature. 2004. PMID: 15538359
RNAi-mediated gene silencing in non-human primates.
Zimmermann TS, Lee AC, Akinc A, Bramlage B, Bumcrot D, Fedoruk MN, Harborth J, Heyes JA, Jeffs LB, John M, Judge AD, Lam K, McClintock K, Nechev LV, Palmer LR, Racie T, Röhl I, Seiffert S, Shanmugam S, Sood V, Soutschek J, Toudjarska I, Wheat AJ, Yaworski E, Zedalis W, Koteliansky V, Manoharan M, Vornlocher HP, MacLachlan I. Zimmermann TS, et al. Among authors: bumcrot d. Nature. 2006 May 4;441(7089):111-4. doi: 10.1038/nature04688. Epub 2006 Mar 26. Nature. 2006. PMID: 16565705
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.
Maeder ML, Stefanidakis M, Wilson CJ, Baral R, Barrera LA, Bounoutas GS, Bumcrot D, Chao H, Ciulla DM, DaSilva JA, Dass A, Dhanapal V, Fennell TJ, Friedland AE, Giannoukos G, Gloskowski SW, Glucksmann A, Gotta GM, Jayaram H, Haskett SJ, Hopkins B, Horng JE, Joshi S, Marco E, Mepani R, Reyon D, Ta T, Tabbaa DG, Samuelsson SJ, Shen S, Skor MN, Stetkiewicz P, Wang T, Yudkoff C, Myer VE, Albright CF, Jiang H. Maeder ML, et al. Among authors: bumcrot d. Nat Med. 2019 Feb;25(2):229-233. doi: 10.1038/s41591-018-0327-9. Epub 2019 Jan 21. Nat Med. 2019. PMID: 30664785
Tissue-specific gene silencing monitored in circulating RNA.
Sehgal A, Chen Q, Gibbings D, Sah DW, Bumcrot D. Sehgal A, et al. Among authors: bumcrot d. RNA. 2014 Feb;20(2):143-9. doi: 10.1261/rna.042507.113. Epub 2013 Dec 19. RNA. 2014. PMID: 24355758 Free PMC article.
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