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1,254 results

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Page 1
From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.
Schlegel MK, Janas MM, Jiang Y, Barry JD, Davis W, Agarwal S, Berman D, Brown CR, Castoreno A, LeBlanc S, Liebow A, Mayo T, Milstein S, Nguyen T, Shulga-Morskaya S, Hyde S, Schofield S, Szeto J, Woods LB, Yilmaz VO, Manoharan M, Egli M, Charissé K, Sepp-Lorenzino L, Haslett P, Fitzgerald K, Jadhav V, Maier MA. Schlegel MK, et al. Among authors: fitzgerald k. Nucleic Acids Res. 2022 Jul 8;50(12):6656-6670. doi: 10.1093/nar/gkac539. Nucleic Acids Res. 2022. PMID: 35736224 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: 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. Proc Natl Acad Sci U S A. 2008. PMID: 18695239 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: fitzgerald k. 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.
Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics.
Maier MA, Jayaraman M, Matsuda S, Liu J, Barros S, Querbes W, Tam YK, Ansell SM, Kumar V, Qin J, Zhang X, Wang Q, Panesar S, Hutabarat R, Carioto M, Hettinger J, Kandasamy P, Butler D, Rajeev KG, Pang B, Charisse K, Fitzgerald K, Mui BL, Du X, Cullis P, Madden TD, Hope MJ, Manoharan M, Akinc A. Maier MA, et al. Among authors: fitzgerald k. Mol Ther. 2013 Aug;21(8):1570-8. doi: 10.1038/mt.2013.124. Epub 2013 Jun 25. Mol Ther. 2013. PMID: 23799535 Free PMC article.
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. Fitzgerald K, et al. Lancet. 2014 Jan 4;383(9911):60-68. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3. Lancet. 2014. PMID: 24094767 Free PMC article. Clinical Trial.
RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice.
Yasuda M, Gan L, Chen B, Kadirvel S, Yu C, Phillips JD, New MI, Liebow A, Fitzgerald K, Querbes W, Desnick RJ. Yasuda M, et al. Among authors: fitzgerald k. Proc Natl Acad Sci U S A. 2014 May 27;111(21):7777-82. doi: 10.1073/pnas.1406228111. Epub 2014 May 12. Proc Natl Acad Sci U S A. 2014. PMID: 24821812 Free PMC article.
Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing.
Nair JK, Willoughby JL, Chan A, Charisse K, Alam MR, Wang Q, Hoekstra M, Kandasamy P, Kel'in AV, Milstein S, Taneja N, O'Shea J, Shaikh S, Zhang L, van der Sluis RJ, Jung ME, Akinc A, Hutabarat R, Kuchimanchi S, Fitzgerald K, Zimmermann T, van Berkel TJ, Maier MA, Rajeev KG, Manoharan M. Nair JK, et al. Among authors: fitzgerald k. J Am Chem Soc. 2014 Dec 10;136(49):16958-61. doi: 10.1021/ja505986a. Epub 2014 Dec 1. J Am Chem Soc. 2014. PMID: 25434769
Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo.
Rajeev KG, Nair JK, Jayaraman M, Charisse K, Taneja N, O'Shea J, Willoughby JL, Yucius K, Nguyen T, Shulga-Morskaya S, Milstein S, Liebow A, Querbes W, Borodovsky A, Fitzgerald K, Maier MA, Manoharan M. Rajeev KG, et al. Among authors: fitzgerald k. Chembiochem. 2015 Apr 13;16(6):903-8. doi: 10.1002/cbic.201500023. Epub 2015 Mar 18. Chembiochem. 2015. PMID: 25786782
Identification of siRNA delivery enhancers by a chemical library screen.
Gilleron J, Paramasivam P, Zeigerer A, Querbes W, Marsico G, Andree C, Seifert S, Amaya P, Stöter M, Koteliansky V, Waldmann H, Fitzgerald K, Kalaidzidis Y, Akinc A, Maier MA, Manoharan M, Bickle M, Zerial M. Gilleron J, et al. Among authors: fitzgerald k. Nucleic Acids Res. 2015 Sep 18;43(16):7984-8001. doi: 10.1093/nar/gkv762. Epub 2015 Jul 28. Nucleic Acids Res. 2015. PMID: 26220182 Free PMC article.
Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification.
Chan A, Liebow A, Yasuda M, Gan L, Racie T, Maier M, Kuchimanchi S, Foster D, Milstein S, Charisse K, Sehgal A, Manoharan M, Meyers R, Fitzgerald K, Simon A, Desnick RJ, Querbes W. Chan A, et al. Among authors: fitzgerald k. Mol Ther Nucleic Acids. 2015 Nov 3;4(11):e263. doi: 10.1038/mtna.2015.36. Mol Ther Nucleic Acids. 2015. PMID: 26528940 Free PMC article.
1,254 results