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Page 1
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: akinc a. 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
Development of lipidoid-siRNA formulations for systemic delivery to the liver.
Akinc A, Goldberg M, Qin J, Dorkin JR, Gamba-Vitalo C, Maier M, Jayaprakash KN, Jayaraman M, Rajeev KG, Manoharan M, Koteliansky V, Röhl I, Leshchiner ES, Langer R, Anderson DG. Akinc A, et al. Mol Ther. 2009 May;17(5):872-9. doi: 10.1038/mt.2009.36. Epub 2009 Mar 3. Mol Ther. 2009. PMID: 19259063 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. 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: akinc a. 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.
5'-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates.
Parmar R, Willoughby JL, Liu J, Foster DJ, Brigham B, Theile CS, Charisse K, Akinc A, Guidry E, Pei Y, Strapps W, Cancilla M, Stanton MG, Rajeev KG, Sepp-Lorenzino L, Manoharan M, Meyers R, Maier MA, Jadhav V. Parmar R, et al. Among authors: akinc a. Chembiochem. 2016 Jun 2;17(11):985-9. doi: 10.1002/cbic.201600130. Epub 2016 Apr 28. Chembiochem. 2016. PMID: 27121751
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates.
Nair JK, Attarwala H, Sehgal A, Wang Q, Aluri K, Zhang X, Gao M, Liu J, Indrakanti R, Schofield S, Kretschmer P, Brown CR, Gupta S, Willoughby JLS, Boshar JA, Jadhav V, Charisse K, Zimmermann T, Fitzgerald K, Manoharan M, Rajeev KG, Akinc A, Hutabarat R, Maier MA. Nair JK, et al. Among authors: akinc a. Nucleic Acids Res. 2017 Nov 2;45(19):10969-10977. doi: 10.1093/nar/gkx818. Nucleic Acids Res. 2017. PMID: 28981809 Free PMC article.
Reversal of siRNA-mediated gene silencing in vivo.
Zlatev I, Castoreno A, Brown CR, Qin J, Waldron S, Schlegel MK, Degaonkar R, Shulga-Morskaya S, Xu H, Gupta S, Matsuda S, Akinc A, Rajeev KG, Manoharan M, Maier MA, Jadhav V. Zlatev I, et al. Among authors: akinc a. Nat Biotechnol. 2018 Jul;36(6):509-511. doi: 10.1038/nbt.4136. Epub 2018 May 14. Nat Biotechnol. 2018. PMID: 29786096
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev KG, Zimmermann T, Sepp-Lorenzino L, Xu Y, Akinc A, Fitzgerald K, Vaishnaw AK, Smith PF, Manoharan M, Jadhav V, Wu JT, Maier MA. Janas MM, et al. Among authors: akinc a. Nucleic Acids Res. 2019 Apr 23;47(7):3306-3320. doi: 10.1093/nar/gkz140. Nucleic Acids Res. 2019. PMID: 30820542 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: akinc a. 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.
Unique gene-silencing and structural properties of 2'-fluoro-modified siRNAs.
Manoharan M, Akinc A, Pandey RK, Qin J, Hadwiger P, John M, Mills K, Charisse K, Maier MA, Nechev L, Greene EM, Pallan PS, Rozners E, Rajeev KG, Egli M. Manoharan M, et al. Among authors: akinc a. Angew Chem Int Ed Engl. 2011 Mar 1;50(10):2284-8. doi: 10.1002/anie.201006519. Epub 2011 Jan 31. Angew Chem Int Ed Engl. 2011. PMID: 21351337 Free PMC article. No abstract available.
51 results