Therapeutic siRNA: state of the art.

作者: Bo Hu , Liping Zhong , Yuhua Weng , Ling Peng , Yuanyu Huang

DOI: 10.1038/S41392-020-0207-X

关键词:

摘要: … For example, the combination of 2′-OMe and phosphorothioate (PS) modifications facilitates the systemic administration of cholesterol-conjugated siRNA and achieves efficient gene …

参考文章(296)
Elisabeth C.R. Solano, Douglas J. Kornbrust, Amber Beaudry, Jeffrey W.-D. Foy, David J. Schneider, James D. Thompson, Toxicological and Pharmacokinetic Properties of QPI-1007, a Chemically Modified Synthetic siRNA Targeting Caspase 2 mRNA, Following Intravitreal Injection Nucleic Acid Therapeutics. ,vol. 24, pp. 258- 266 ,(2014) , 10.1089/NAT.2014.0489
Magdolna G. Sebestyén, So C. Wong, Vladimir Trubetskoy, David L. Lewis, Christine I. Wooddell, Targeted In Vivo Delivery of siRNA and an Endosome-Releasing Agent to Hepatocytes RNA Interference. ,vol. 1218, pp. 163- 186 ,(2015) , 10.1007/978-1-4939-1538-5_10
Lakshmipathi Panderarinathan, Karen Bulock, Lyn Libertine, Gabriel Luna, Steven K Fisher, James Cardia, Geoffrey P Lewis, Pamela A Pavco, Michael Byrne, RXI-109 Treatment for Proliferative Vitreoretinopathy (PVR) and other Ocular Disorders Investigative Ophthalmology & Visual Science. ,vol. 55, pp. 1120- 1120 ,(2014)
Jens Gottlieb, Martin R. Zamora, Tony Hodges, A.W. Musk, Urte Sommerwerk, Daniel Dilling, Selim Arcasoy, John DeVincenzo, Verena Karsten, Shaily Shah, Brian R. Bettencourt, Jeffrey Cehelsky, Sara Nochur, Jared Gollob, Akshay Vaishnaw, Amy R. Simon, Allan R. Glanville, ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients Journal of Heart and Lung Transplantation. ,vol. 35, pp. 213- 221 ,(2016) , 10.1016/J.HEALUN.2015.08.012
B.P. Monia, E.A. Lesnik, C. Gonzalez, W.F. Lima, D. McGee, C.J. Guinosso, A.M. Kawasaki, P.D. Cook, S.M. Freier, Evaluation of 2'-modified oligonucleotides containing 2'-deoxy gaps as antisense inhibitors of gene expression Journal of Biological Chemistry. ,vol. 268, pp. 14514- 14522 ,(1993) , 10.1016/S0021-9258(19)85268-7
DANIEL A. GESELOWITZ, LEONARD M. NECKERS, Bovine serum albumin is a major oligonucleotide-binding protein found on the surface of cultured cells. Antisense research and development. ,vol. 5, pp. 213- 217 ,(1995) , 10.1089/ARD.1995.5.213
Sayda M. Elbashir, Jens Harborth, Winfried Lendeckel, Abdullah Yalcin, Klaus Weber, Thomas Tuschl, Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells Nature. ,vol. 411, pp. 494- 498 ,(2001) , 10.1038/35078107
Qili Chu, Christina L. Frey, Vladimir S. Trubetskoy, Julia Hegge, Darren Wakefield, David B. Rozema, David L. Lewis, So C. Wong, Jason J. Klein, Holly L. Hamilton, Co-Injection of a Targeted, Reversibly Masked Endosomolytic Polymer Dramatically Improves the Efficacy of Cholesterol-Conjugated Small Interfering RNAs In Vivo Nucleic Acid Therapeutics. ,vol. 22, pp. 380- 390 ,(2012) , 10.1089/NAT.2012.0389
James D. Thompson, Douglas J. Kornbrust, Jeffrey W-D. Foy, Elisabeth C.R. Solano, David J. Schneider, Elena Feinstein, Bruce A. Molitoris, Shai Erlich, Toxicological and pharmacokinetic properties of chemically modified siRNAs targeting p53 RNA following intravenous administration. Nucleic Acid Therapeutics. ,vol. 22, pp. 255- 264 ,(2012) , 10.1089/NAT.2012.0371