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Page 1
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: vornlocher hp. 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 RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates.
Frank-Kamenetsky M, Grefhorst A, Anderson NN, Racie TS, Bramlage B, Akinc A, Butler D, Charisse K, Dorkin R, Fan Y, Gamba-Vitalo C, Hadwiger P, Jayaraman M, John M, Jayaprakash KN, Maier M, Nechev L, Rajeev KG, Read T, Röhl I, Soutschek J, Tan P, Wong J, Wang G, Zimmermann T, de Fougerolles A, Vornlocher HP, Langer R, Anderson DG, Manoharan M, Koteliansky V, Horton JD, Fitzgerald K. Frank-Kamenetsky M, et al. Among authors: vornlocher hp. Proc Natl Acad Sci U S A. 2008 Aug 19;105(33):11915-20. doi: 10.1073/pnas.0805434105. Epub 2008 Aug 11. Proc Natl Acad Sci U S A. 2008. PMID: 18695239 Free PMC article.
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: vornlocher hp. 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: vornlocher hp. Nature. 2004 Nov 11;432(7014):173-8. doi: 10.1038/nature03121. Nature. 2004. PMID: 15538359 Free article.
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: vornlocher hp. Nature. 2007 Oct 11;449(7163):745-7. doi: 10.1038/nature06179. Epub 2007 Sep 26. Nature. 2007. PMID: 17898712 Free PMC article.
Hepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infection.
Wooddell CI, Rozema DB, Hossbach M, John M, Hamilton HL, Chu Q, Hegge JO, Klein JJ, Wakefield DH, Oropeza CE, Deckert J, Roehl I, Jahn-Hofmann K, Hadwiger P, Vornlocher HP, McLachlan A, Lewis DL. Wooddell CI, et al. Among authors: vornlocher hp. Mol Ther. 2013 May;21(5):973-85. doi: 10.1038/mt.2013.31. Epub 2013 Feb 26. Mol Ther. 2013. PMID: 23439496 Free PMC article.
Delivery of siRNA to the mouse lung via a functionalized lipopolyamine.
Polach KJ, Matar M, Rice J, Slobodkin G, Sparks J, Congo R, Rea-Ramsey A, McClure D, Brunhoeber E, Krampert M, Schuster A, Jahn-Hofmann K, John M, Vornlocher HP, Fewell JG, Anwer K, Geick A. Polach KJ, et al. Among authors: vornlocher hp. Mol Ther. 2012 Jan;20(1):91-100. doi: 10.1038/mt.2011.210. Epub 2011 Oct 11. Mol Ther. 2012. PMID: 21988874 Free PMC article.
54 results