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Results: 10

1.

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial.

Fitzgerald K, Frank-Kamenetsky M, Shulga-Morskaya S, Liebow A, Bettencourt BR, Sutherland JE, Hutabarat RM, Clausen VA, Karsten V, Cehelsky J, Nochur SV, Kotelianski V, Horton J, Mant T, Chiesa J, Ritter J, Munisamy M, Vaishnaw AK, Gollob JA, Simon A.

Lancet. 2014 Jan 4;383(9911):60-8. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3.

2.

Silencing of lipid metabolism genes through IRE1α-mediated mRNA decay lowers plasma lipids in mice.

So JS, Hur KY, Tarrio M, Ruda V, Frank-Kamenetsky M, Fitzgerald K, Koteliansky V, Lichtman AH, Iwawaki T, Glimcher LH, Lee AH.

Cell Metab. 2012 Oct 3;16(4):487-99. doi: 10.1016/j.cmet.2012.09.004.

3.

Activation of ER stress and mTORC1 suppresses hepatic sortilin-1 levels in obese mice.

Ai D, Baez JM, Jiang H, Conlon DM, Hernandez-Ono A, Frank-Kamenetsky M, Milstein S, Fitzgerald K, Murphy AJ, Woo CW, Strong A, Ginsberg HN, Tabas I, Rader DJ, Tall AR.

J Clin Invest. 2012 May;122(5):1677-87. doi: 10.1172/JCI61248. Epub 2012 Apr 2.

4.

The Scap/SREBP pathway is essential for developing diabetic fatty liver and carbohydrate-induced hypertriglyceridemia in animals.

Moon YA, Liang G, Xie X, Frank-Kamenetsky M, Fitzgerald K, Koteliansky V, Brown MS, Goldstein JL, Horton JD.

Cell Metab. 2012 Feb 8;15(2):240-6. doi: 10.1016/j.cmet.2011.12.017.

5.

IRE1α activation protects mice against acetaminophen-induced hepatotoxicity.

Hur KY, So JS, Ruda V, Frank-Kamenetsky M, Fitzgerald K, Koteliansky V, Iwawaki T, Glimcher LH, Lee AH.

J Exp Med. 2012 Feb 13;209(2):307-18. doi: 10.1084/jem.20111298. Epub 2012 Jan 30.

6.

From noncoding variant to phenotype via SORT1 at the 1p13 cholesterol locus.

Musunuru K, Strong A, Frank-Kamenetsky M, Lee NE, Ahfeldt T, Sachs KV, Li X, Li H, Kuperwasser N, Ruda VM, Pirruccello JP, Muchmore B, Prokunina-Olsson L, Hall JL, Schadt EE, Morales CR, Lund-Katz S, Phillips MC, Wong J, Cantley W, Racie T, Ejebe KG, Orho-Melander M, Melander O, Koteliansky V, Fitzgerald K, Krauss RM, Cowan CA, Kathiresan S, Rader DJ.

Nature. 2010 Aug 5;466(7307):714-9. doi: 10.1038/nature09266.

7.

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.

Mol Ther. 2010 Jul;18(7):1357-64. doi: 10.1038/mt.2010.85. Epub 2010 May 11.

8.

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.

Proc Natl Acad Sci U S A. 2010 Feb 2;107(5):1864-9. doi: 10.1073/pnas.0910603106. Epub 2010 Jan 11. Erratum in: Proc Natl Acad Sci U S A. 2010 May 25;107(21):9915.

9.

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.

Proc Natl Acad Sci U S A. 2008 Aug 19;105(33):11915-20. doi: 10.1073/pnas.0805434105. Epub 2008 Aug 11.

10.

Small-molecule modulators of Hedgehog signaling: identification and characterization of Smoothened agonists and antagonists.

Frank-Kamenetsky M, Zhang XM, Bottega S, Guicherit O, Wichterle H, Dudek H, Bumcrot D, Wang FY, Jones S, Shulok J, Rubin LL, Porter JA.

J Biol. 2002 Nov 6;1(2):10.

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