Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia
Raal FJ, Kallend D, Ray KK, Turner T, Koenig W, Wright RS, Wijngaard PLJ, Curcio D, Jaros MJ, Leiter LA, Kastelein JJP; ORION-9 Investigators. N Engl J Med. 2020 Mar 18. PubMed
Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol
Ray KK, Wright RS, Kallend D, Koenig W, Leiter LA, Raal FJ, Bisch JA, Richardson T, Jaros M, Wijngaard PLJ, Kastelein JJP; ORION-10 and ORION-11 Investigators. N Engl J Med. 2020 Mar 18. PubMed
Endocytosis Controls siRNA Efficiency: Implications for siRNA Delivery Vehicle Design and Cell-Specific Targeting
Vocelle D, Chan C, Walton SP. Nucleic Acid Ther. 2020 Feb;30(1):22-32. PubMed
Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold
Xie J, Tai PWL, Brown A, Gong S, Zhu S, Wang Y, Li C, Colpan C, Su Q, He R, Ma H, Li J, Ye H, Ko J, Zamore PD, Gao G. Mol Ther. 2020 Feb 5;28(2):422-430. PubMed
Impact of Phosphorothioate Chirality on Double-Stranded siRNAs: A Systematic Evaluation of Stereopure siRNA Designs
Sakamuri S, Eltepu L, Liu D, Lam S, Meade BR, Liu B, Dello Iacono G, Kabakibi A, Luukkonen L, Leedom T, Foster M, Bradshaw CW. Chembiochem. 2019 Dec 21. PubMed
Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid (siRNA), Givosiran, in Patients with Acute Hepatic Porphyria.
Agarwal S, Simon AR, Goel V, Habtemariam BA, Clausen VA, Kim JB, Robbie GJ. Clin Pharmacol Ther. 2020 Jan 29. PubMed
Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis.
Sutherland JE, Hettinger JL, Chan A, Gilbert J, Warner GL, Davis WP. Nucleic Acid Ther. 2019 Dec 10. PubMed
Knockdown of Virus Antigen Expression Increases Therapeutic Vaccine Efficacy in High-titer HBV Carrier Mice.
Michler T, Kosinska AD, Festag J, Bunse T, Su J, Ringelhan M, Imhof H, Grimm D, Steiger K, Mogler C, Heikenwalder M, Michel ML, Guzman CA, Milstein S, Sepp-Lorenzino L, Knolle P, Protzer U. Gastroenterology. 2020 Jan 28. pii: S0016-5085(20)30125-6. PubMed
The effector mechanism of siRNA spherical nucleic acids.
Yamankurt G, Stawicki RJ, Posadas DM, Nguyen JQ, Carthew RW, Mirkin CA. Proc Natl Acad Sci U S A. 2020 Jan 21;117(3):1312-1320. PubMed
HER3-targeted protein chimera forms endosomolytic capsomeres and self-assembles into stealth nucleocapsids for systemic tumor homing of RNA interference in vivo.
Alonso-Valenteen F, Pacheco S, Srinivas D, Rentsendorj A, Chu D, Lubow J, Sims J, Miao T, Mikhael S, Hwang JY, Abrol R, Medina Kauwe LK. Nucleic Acids Res. 2019 Dec 2;47(21):11020-11043. PubMed