Non-viral vectors for gene-based therapy H Yin, RL Kanasty, AA Eltoukhy, AJ Vegas, JR Dorkin, DG Anderson Nature reviews genetics 15 (8), 541-555, 2014 | 3324 | 2014 |
Delivery materials for siRNA therapeutics R Kanasty, JR Dorkin, A Vegas, D Anderson Nature materials 12 (11), 967-977, 2013 | 2036 | 2013 |
Rational design of cationic lipids for siRNA delivery SC Semple, A Akinc, J Chen, AP Sandhu, BL Mui, CK Cho, DWY Sah, ... Nature biotechnology 28 (2), 172-176, 2010 | 1916 | 2010 |
A combinatorial library of lipid-like materials for delivery of RNAi therapeutics A Akinc, A Zumbuehl, M Goldberg, ES Leshchiner, V Busini, N Hossain, ... Nature biotechnology 26 (5), 561-569, 2008 | 1439 | 2008 |
Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms A Akinc, W Querbes, S De, J Qin, M Frank-Kamenetsky, KN Jayaprakash, ... Molecular Therapy 18 (7), 1357-1364, 2010 | 1112 | 2010 |
Lipid-like materials for low-dose, in vivo gene silencing KT Love, KP Mahon, CG Levins, KA Whitehead, W Querbes, JR Dorkin, ... Proceedings of the National Academy of Sciences 107 (5), 1864-1869, 2010 | 1098 | 2010 |
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo H Yin, CQ Song, JR Dorkin, LJ Zhu, Y Li, Q Wu, A Park, J Yang, S Suresh, ... Nature biotechnology 34 (3), 328-333, 2016 | 959 | 2016 |
Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates M Frank-Kamenetsky, A Grefhorst, NN Anderson, TS Racie, B Bramlage, ... Proceedings of the National Academy of Sciences 105 (33), 11915-11920, 2008 | 810 | 2008 |
Optimization of lipid nanoparticle formulations for mRNA delivery in vivo with fractional factorial and definitive screening designs KJ Kauffman, JR Dorkin, JH Yang, MW Heartlein, F DeRosa, FF Mir, ... Nano letters 15 (11), 7300-7306, 2015 | 647 | 2015 |
Lipid nanoparticle assisted mRNA delivery for potent cancer immunotherapy MA Oberli, AM Reichmuth, JR Dorkin, MJ Mitchell, OS Fenton, A Jaklenec, ... Nano letters 17 (3), 1326-1335, 2017 | 628 | 2017 |
Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity KA Whitehead, JR Dorkin, AJ Vegas, PH Chang, O Veiseh, J Matthews, ... Nature communications 5 (1), 4277, 2014 | 537 | 2014 |
Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates Y Dong, KT Love, JR Dorkin, S Sirirungruang, Y Zhang, D Chen, ... Proceedings of the National Academy of Sciences 111 (11), 3955-3960, 2014 | 510 | 2014 |
Development of lipidoid–siRNA formulations for systemic delivery to the liver A Akinc, M Goldberg, J Qin, JR Dorkin, C Gamba-Vitalo, M Maier, ... Molecular Therapy 17 (5), 872-879, 2009 | 459 | 2009 |
Lipid formulation J Chen, S Ansell, A Akinc, JR Dorkin, X Qin, W Cantley, M Manoharan, ... US Patent 8,158,601, 2012 | 413 | 2012 |
Bioinspired alkenyl amino alcohol ionizable lipid materials for highly potent in vivo mRNA delivery OS Fenton, KJ Kauffman, RL McClellan, EA Appel, JR Dorkin, MW Tibbitt, ... Advanced materials (Deerfield Beach, Fla.) 28 (15), 2939, 2016 | 220 | 2016 |
Synthesis and biological evaluation of ionizable lipid materials for the in vivo delivery of messenger RNA to B lymphocytes OS Fenton, KJ Kauffman, JC Kaczmarek, RL McClellan, S Jhunjhunwala, ... Advanced Materials 29 (33), 1606944, 2017 | 210 | 2017 |
Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system F DeRosa, B Guild, S Karve, L Smith, K Love, JR Dorkin, KJ Kauffman, ... Gene therapy 23 (10), 699-707, 2016 | 166 | 2016 |
Lipid formulation J Chen, S Ansell, A Akinc, JR Dorkin, X Qin, W Cantley, M Manoharan, ... US Patent 8,802,644, 2014 | 127 | 2014 |
In Vitro–In Vivo Translation of Lipid Nanoparticles for Hepatocellular siRNA Delivery KA Whitehead, J Matthews, PH Chang, F Niroui, JR Dorkin, M Severgnini, ... ACS nano 6 (8), 6922-6929, 2012 | 126 | 2012 |
lipid formulation A Aking, W Querbes, FMP Wong, JR Dorkin, X Qin, W Cantley, ... US Patent App. 13/147,189, 2012 | 117 | 2012 |