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A status report on RNAi therapeutics.
Vaishnaw AK, Gollob J, Gamba-Vitalo C, Hutabarat R, Sah D, Meyers R, de Fougerolles T, Maraganore J. Vaishnaw AK, et al. Silence. 2010 Jul 8;1(1):14. doi: 10.1186/1758-907X-1-14. Silence. 2010. PMID: 20615220 Free PMC article.
Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis.
Butler JS, Chan A, Costelha S, Fishman S, Willoughby JL, Borland TD, Milstein S, Foster DJ, Gonçalves P, Chen Q, Qin J, Bettencourt BR, Sah DW, Alvarez R, Rajeev KG, Manoharan M, Fitzgerald K, Meyers RE, Nochur SV, Saraiva MJ, Zimmermann TS. Butler JS, et al. Among authors: sah dw. Amyloid. 2016 Jun;23(2):109-18. doi: 10.3109/13506129.2016.1160882. Epub 2016 Mar 31. Amyloid. 2016. PMID: 27033334 Free PMC article.
Harnessing a physiologic mechanism for siRNA delivery with mimetic lipoprotein particles.
Nakayama T, Butler JS, Sehgal A, Severgnini M, Racie T, Sharman J, Ding F, Morskaya SS, Brodsky J, Tchangov L, Kosovrasti V, Meys M, Nechev L, Wang G, Peng CG, Fang Y, Maier M, Rajeev KG, Li R, Hettinger J, Barros S, Clausen V, Zhang X, Wang Q, Hutabarat R, Dokholyan NV, Wolfrum C, Manoharan M, Kotelianski V, Stoffel M, Sah DW. Nakayama T, et al. Among authors: sah dw. Mol Ther. 2012 Aug;20(8):1582-9. doi: 10.1038/mt.2012.33. Epub 2012 Jun 5. Mol Ther. 2012. PMID: 22850721 Free PMC article.
Lipid-like materials for low-dose, in vivo gene silencing.
Love KT, Mahon KP, Levins CG, Whitehead KA, Querbes W, Dorkin JR, Qin J, Cantley W, Qin LL, Racie T, Frank-Kamenetsky M, Yip KN, Alvarez R, Sah DW, de Fougerolles A, Fitzgerald K, Koteliansky V, Akinc A, Langer R, Anderson DG. Love KT, et al. Among authors: sah dw. Proc Natl Acad Sci U S A. 2010 Feb 2;107(5):1864-9. doi: 10.1073/pnas.0910603106. Epub 2010 Jan 11. Proc Natl Acad Sci U S A. 2010. PMID: 20080679 Free PMC article.
Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.
Akinc A, Querbes W, De S, Qin J, Frank-Kamenetsky M, Jayaprakash KN, Jayaraman M, Rajeev KG, Cantley WL, Dorkin JR, Butler JS, Qin L, Racie T, Sprague A, Fava E, Zeigerer A, Hope MJ, Zerial M, Sah DW, Fitzgerald K, Tracy MA, Manoharan M, Koteliansky V, Fougerolles Ad, Maier MA. Akinc A, et al. Among authors: sah dw. Mol Ther. 2010 Jul;18(7):1357-64. doi: 10.1038/mt.2010.85. Epub 2010 May 11. Mol Ther. 2010. PMID: 20461061 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: sah dw. Nat Biotechnol. 2008 May;26(5):561-9. doi: 10.1038/nbt1402. Epub 2008 Apr 27. Nat Biotechnol. 2008. PMID: 18438401 Free PMC article.
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