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Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates.
Brown KM, Nair JK, Janas MM, Anglero-Rodriguez YI, Dang LTH, Peng H, Theile CS, Castellanos-Rizaldos E, Brown C, Foster D, Kurz J, Allen J, Maganti R, Li J, Matsuda S, Stricos M, Chickering T, Jung M, Wassarman K, Rollins J, Woods L, Kelin A, Guenther DC, Mobley MW, Petrulis J, McDougall R, Racie T, Bombardier J, Cha D, Agarwal S, Johnson L, Jiang Y, Lentini S, Gilbert J, Nguyen T, Chigas S, LeBlanc S, Poreci U, Kasper A, Rogers AB, Chong S, Davis W, Sutherland JE, Castoreno A, Milstein S, Schlegel MK, Zlatev I, Charisse K, Keating M, Manoharan M, Fitzgerald K, Wu JT, Maier MA, Jadhav V. Brown KM, et al. Among authors: maier ma. Nat Biotechnol. 2022 Oct;40(10):1500-1508. doi: 10.1038/s41587-022-01334-x. Epub 2022 Jun 2. Nat Biotechnol. 2022. PMID: 35654979
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. 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.
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.
RNA interference-mediated silencing of the respiratory syncytial virus nucleocapsid defines a potent antiviral strategy.
Alvarez R, Elbashir S, Borland T, Toudjarska I, Hadwiger P, John M, Roehl I, Morskaya SS, Martinello R, Kahn J, Van Ranst M, Tripp RA, DeVincenzo JP, Pandey R, Maier M, Nechev L, Manoharan M, Kotelianski V, Meyers R. Alvarez R, et al. Antimicrob Agents Chemother. 2009 Sep;53(9):3952-62. doi: 10.1128/AAC.00014-09. Epub 2009 Jun 8. Antimicrob Agents Chemother. 2009. PMID: 19506055 Free PMC article.
Rational design of cationic lipids for siRNA delivery.
Semple SC, Akinc A, Chen J, Sandhu AP, Mui BL, Cho CK, Sah DW, Stebbing D, Crosley EJ, Yaworski E, Hafez IM, Dorkin JR, Qin J, Lam K, Rajeev KG, Wong KF, Jeffs LB, Nechev L, Eisenhardt ML, Jayaraman M, Kazem M, Maier MA, Srinivasulu M, Weinstein MJ, Chen Q, Alvarez R, Barros SA, De S, Klimuk SK, Borland T, Kosovrasti V, Cantley WL, Tam YK, Manoharan M, Ciufolini MA, Tracy MA, de Fougerolles A, MacLachlan I, Cullis PR, Madden TD, Hope MJ. Semple SC, et al. Among authors: maier ma. Nat Biotechnol. 2010 Feb;28(2):172-6. doi: 10.1038/nbt.1602. Epub 2010 Jan 17. Nat Biotechnol. 2010. PMID: 20081866
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: maier ma. 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.
Modulation of thermal stability can enhance the potency of siRNA.
Addepalli H, Meena, Peng CG, Wang G, Fan Y, Charisse K, Jayaprakash KN, Rajeev KG, Pandey RK, Lavine G, Zhang L, Jahn-Hofmann K, Hadwiger P, Manoharan M, Maier MA. Addepalli H, et al. Among authors: maier ma. Nucleic Acids Res. 2010 Nov;38(20):7320-31. doi: 10.1093/nar/gkq568. Epub 2010 Jul 7. Nucleic Acids Res. 2010. PMID: 20610434 Free PMC article.
192 results