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Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications.
Friedland AE, Baral R, Singhal P, Loveluck K, Shen S, Sanchez M, Marco E, Gotta GM, Maeder ML, Kennedy EM, Kornepati AV, Sousa A, Collins MA, Jayaram H, Cullen BR, Bumcrot D. Friedland AE, et al. Among authors: bumcrot d. Genome Biol. 2015 Nov 24;16:257. doi: 10.1186/s13059-015-0817-8. Genome Biol. 2015. PMID: 26596280 Free PMC article.
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
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
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 Free article.
38 results