RNAi-based nanomedicines for targeted personalized therapy

被引:137
作者
Daka, Ala [1 ]
Peer, Dan [1 ]
机构
[1] Tel Aviv Univ, Ctr Nanosci & Nanotechnol, IL-69978 Tel Aviv, Israel
基金
以色列科学基金会;
关键词
Short interfering RNA (siRNA); In vivo; Systemic delivery; Nanoparticles; Clinical trials; Personalized medicine; SMALL INTERFERING RNA; SIRNA DELIVERY-SYSTEM; LIPID-LIKE MATERIALS; SHORT HAIRPIN RNA; IN-VIVO DELIVERY; GENE-EXPRESSION; CARBON NANOTUBES; CHEMICAL-MODIFICATION; MACROMOLECULAR THERAPEUTICS; ANTISENSE OLIGONUCLEOTIDES;
D O I
10.1016/j.addr.2012.08.014
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
RNA interference (RNAi) has just made it through the pipeline to clinical trials. However, in order for RNAi to serve as an ideal personalized therapeutics and be clinically approved safe, specific, and potent strategies must be devised for efficient delivery of RNAi payloads to specific cell types, which despite the immense potential, remains a challenge. Through evaluating the recent reported studies in this field, we introduce the progress in designing targeted nano-scaled strategies that are anticipated to overcome the delivery drawbacks and along with the exciting "omics" discipline to personalize RNAi-based therapeutics. (C) 2012 Elsevier B.V. All rights reserved.
引用
收藏
页码:1508 / 1521
页数:14
相关论文
共 156 条
[1]   RNAi therapeutics: Principles, prospects and challenges [J].
Aagaard, Lars ;
Rossi, John J. .
ADVANCED DRUG DELIVERY REVIEWS, 2007, 59 (2-3) :75-86
[2]   Antisense and siRNA as agonists of Toll-like receptors [J].
Agrawal, S ;
Kandimalla, ER .
NATURE BIOTECHNOLOGY, 2004, 22 (12) :1533-1537
[3]   A combinatorial library of lipid-like materials for delivery of RNAi therapeutics [J].
Akinc, Akin ;
Zumbuehl, Andreas ;
Goldberg, Michael ;
Leshchiner, Elizaveta S. ;
Busini, Valentina ;
Hossain, Naushad ;
Bacallado, Sergio A. ;
Nguyen, David N. ;
Fuller, Jason ;
Alvarez, Rene ;
Borodovsky, Anna ;
Borland, Todd ;
Constien, Rainer ;
de Fougerolles, Antonin ;
Dorkin, J. Robert ;
Jayaprakash, K. Narayanannair ;
Jayaraman, Muthusamy ;
John, Matthias ;
Koteliansky, Victor ;
Manoharan, Muthiah ;
Nechev, Lubomir ;
Qin, June ;
Racie, Timothy ;
Raitcheva, Denitza ;
Rajeev, Kallanthottathil G. ;
Sah, Dinah W. Y. ;
Soutschek, Juergen ;
Toudjarska, Ivanka ;
Vornlocher, Hans-Peter ;
Zimmermann, Tracy S. ;
Langer, Robert ;
Anderson, Daniel G. .
NATURE BIOTECHNOLOGY, 2008, 26 (05) :561-569
[4]   Factors affecting the clearance and biodistribution of polymeric nanoparticles [J].
Alexis, Frank ;
Pridgen, Eric ;
Molnar, Linda K. ;
Farokhzad, Omid C. .
MOLECULAR PHARMACEUTICS, 2008, 5 (04) :505-515
[5]   Supramolecular assemblies in functional siRNA delivery: Where do we stand? [J].
Aliabadi, Hamidreza M. ;
Landry, Breanne ;
Sun, Chongbo ;
Tang, Tian ;
Uludag, Hasan .
BIOMATERIALS, 2012, 33 (08) :2546-2569
[6]   Fully 2′-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA [J].
Allerson, CR ;
Sioufi, N ;
Jarres, R ;
Prakash, TP ;
Naik, N ;
Berdeja, A ;
Wanders, L ;
Griffey, RH ;
Swayze, EE ;
Bhat, B .
JOURNAL OF MEDICINAL CHEMISTRY, 2005, 48 (04) :901-904
[7]   Comparison of antisense oligonucleotides and siRNAs in cell culture and in vivo [J].
Bertrand, JR ;
Pottier, M ;
Vekris, A ;
Opolon, P ;
Maksimenko, A ;
Malvy, C .
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2002, 296 (04) :1000-1004
[8]   3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets [J].
Birmingham, A ;
Anderson, EM ;
Reynolds, A ;
Ilsley-Tyree, D ;
Leake, D ;
Fedorov, Y ;
Baskerville, S ;
Maksimova, E ;
Robinson, K ;
Karpilow, J ;
Marshall, WS ;
Khvorova, A .
NATURE METHODS, 2006, 3 (03) :199-204
[9]   Inhibition of respiratory viruses by nasally administered siRNA [J].
Bitko, V ;
Musiyenko, A ;
Shulyayeva, O ;
Barik, S .
NATURE MEDICINE, 2005, 11 (01) :50-55
[10]   Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo [J].
Boudreau, Ryan L. ;
Martins, Ines ;
Davidson, Beverly L. .
MOLECULAR THERAPY, 2009, 17 (01) :169-175