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Page 1
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: kandasamy p. 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.
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: kandasamy p. 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
siRNA conjugates carrying sequentially assembled trivalent N-acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes.
Matsuda S, Keiser K, Nair JK, Charisse K, Manoharan RM, Kretschmer P, Peng CG, V Kel'in A, Kandasamy P, Willoughby JL, Liebow A, Querbes W, Yucius K, Nguyen T, Milstein S, Maier MA, Rajeev KG, Manoharan M. Matsuda S, et al. Among authors: kandasamy p. ACS Chem Biol. 2015 May 15;10(5):1181-7. doi: 10.1021/cb501028c. Epub 2015 Mar 2. ACS Chem Biol. 2015. PMID: 25730476
An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.
Sehgal A, Barros S, Ivanciu L, Cooley B, Qin J, Racie T, Hettinger J, Carioto M, Jiang Y, Brodsky J, Prabhala H, Zhang X, Attarwala H, Hutabarat R, Foster D, Milstein S, Charisse K, Kuchimanchi S, Maier MA, Nechev L, Kandasamy P, Kel'in AV, Nair JK, Rajeev KG, Manoharan M, Meyers R, Sorensen B, Simon AR, Dargaud Y, Negrier C, Camire RM, Akinc A. Sehgal A, et al. Among authors: kandasamy p. Nat Med. 2015 May;21(5):492-7. doi: 10.1038/nm.3847. Epub 2015 Apr 13. Nat Med. 2015. PMID: 25849132
Metabolically Stable Anomeric Linkages Containing GalNAc-siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways.
Kandasamy P, Mori S, Matsuda S, Erande N, Datta D, Willoughby JLS, Taneja N, O'Shea J, Bisbe A, Manoharan RM, Yucius K, Nguyen T, Indrakanti R, Gupta S, Gilbert JA, Racie T, Chan A, Liu J, Hutabarat R, Nair JK, Charisse K, Maier MA, Rajeev KG, Egli M, Manoharan M. Kandasamy P, et al. J Med Chem. 2023 Feb 23;66(4):2506-2523. doi: 10.1021/acs.jmedchem.2c01337. Epub 2023 Feb 9. J Med Chem. 2023. PMID: 36757090
Quantitative Measurement of Cytosolic and Nuclear Penetration of Oligonucleotide Therapeutics.
Deprey K, Batistatou N, Debets MF, Godfrey J, VanderWall KB, Miles RR, Shehaj L, Guo J, Andreucci A, Kandasamy P, Lu G, Shimizu M, Vargeese C, Kritzer JA. Deprey K, et al. Among authors: kandasamy p. ACS Chem Biol. 2022 Feb 18;17(2):348-360. doi: 10.1021/acschembio.1c00830. Epub 2022 Jan 15. ACS Chem Biol. 2022. PMID: 35034446 Free PMC article.
Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species.
Byrne M, Vathipadiekal V, Apponi L, Iwamoto N, Kandasamy P, Longo K, Liu F, Looby R, Norwood L, Shah A, Shelke JD, Shivalila C, Yang H, Yin Y, Guo L, Bowman K, Vargeese C. Byrne M, et al. Among authors: kandasamy p. Transl Vis Sci Technol. 2021 Jan 12;10(1):23. doi: 10.1167/tvst.10.1.23. eCollection 2021 Jan. Transl Vis Sci Technol. 2021. PMID: 33510962 Free PMC article.
Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS.
Kandasamy P, Liu Y, Aduda V, Akare S, Alam R, Andreucci A, Boulay D, Bowman K, Byrne M, Cannon M, Chivatakarn O, Shelke JD, Iwamoto N, Kawamoto T, Kumarasamy J, Lamore S, Lemaitre M, Lin X, Longo K, Looby R, Marappan S, Metterville J, Mohapatra S, Newman B, Paik IH, Patil S, Purcell-Estabrook E, Shimizu M, Shum P, Standley S, Taborn K, Tripathi S, Yang H, Yin Y, Zhao X, Dale E, Vargeese C. Kandasamy P, et al. Nucleic Acids Res. 2022 Jun 10;50(10):5401-5423. doi: 10.1093/nar/gkac037. Nucleic Acids Res. 2022. PMID: 35106589 Free PMC article.
Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides.
Monian P, Shivalila C, Lu G, Shimizu M, Boulay D, Bussow K, Byrne M, Bezigian A, Chatterjee A, Chew D, Desai J, Favaloro F, Godfrey J, Hoss A, Iwamoto N, Kawamoto T, Kumarasamy J, Lamattina A, Lindsey A, Liu F, Looby R, Marappan S, Metterville J, Murphy R, Rossi J, Pu T, Bhattarai B, Standley S, Tripathi S, Yang H, Yin Y, Yu H, Zhou C, Apponi LH, Kandasamy P, Vargeese C. Monian P, et al. Among authors: kandasamy p. Nat Biotechnol. 2022 Jul;40(7):1093-1102. doi: 10.1038/s41587-022-01225-1. Epub 2022 Mar 7. Nat Biotechnol. 2022. PMID: 35256816
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models.
Liu Y, Dodart JC, Tran H, Berkovitch S, Braun M, Byrne M, Durbin AF, Hu XS, Iwamoto N, Jang HG, Kandasamy P, Liu F, Longo K, Ruschel J, Shelke J, Yang H, Yin Y, Donner A, Zhong Z, Vargeese C, Brown RH Jr. Liu Y, et al. Among authors: kandasamy p. Nat Commun. 2021 Feb 8;12(1):847. doi: 10.1038/s41467-021-21112-8. Nat Commun. 2021. PMID: 33558503 Free PMC article.
90 results