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

来源：

ADVANCED DRUG DELIVERY REVIEWS | 2012年 / 64卷 / 13期

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

页码：1508 / 1521

页数：14

共 156 条

[41] Postexposure protection of non-human primates against a lethal Ebola virus challenge with RNA interference: a proof-of-concept study [J].

Geisbert, Thomas W. ;

Lee, Amy C. H. ;

Robbins, Marjorie ;

Geisbert, Joan B. ;

Honko, Anna N. ;

Sood, Vandana ;

Johnson, Joshua C. ;

de Jong, Susan ;

Tavakoli, Iran ;

Judge, Adam ;

Hensley, Lisa E. ;

MacLachlan, Ian .

LANCET, 2010, 375 (9729) :1896-1905

[42]

Goldsmith M, 2011, NANOMEDICINE-UK, V6, P1771, DOI [10.2217/nnm.11.162, 10.2217/NNM.11.162]

[43] Inhibition of gene expression in mice muscle by in vivo electrically mediated siRNA delivery [J].

Golzio, M ;

Mazzolini, L ;

Moller, P ;

Rols, MP ;

Teissié, J .

GENE THERAPY, 2005, 12 (03) :246-251

[44] Silencing primary dystonia: Lentiviral-mediated RNA interference therapy for DYT1 dystonia [J].

Gonzalez-Alegre, P ;

Bode, N ;

Davidson, BL ;

Paulson, HL .

JOURNAL OF NEUROSCIENCE, 2005, 25 (45) :10502-10509

[45] Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways [J].

Grimm, Dirk ;

Streetz, Konrad L. ;

Jopling, Catherine L. ;

Storm, Theresa A. ;

Pandey, Kusum ;

Davis, Corrine R. ;

Marion, Patricia ;

Salazar, Felix ;

Kay, Mark A. .

NATURE, 2006, 441 (7092) :537-541

[46] Genes and mechanisms related to RNA interference regulate expression of the small temporal RNAs that control C-elegans developmental timing [J].

Grishok, A ;

Pasquinelli, AE ;

Conte, D ;

Li, N ;

Parrish, S ;

Ha, I ;

Baillie, DL ;

Fire, A ;

Ruvkun, G ;

Mello, CC .

CELL, 2001, 106 (01) :23-34

[47] RNA interference-mediated gene silencing of pleiotrophin through polyethylenimine-complexed small interfering RNAs in vivo exerts antitumoral effects in glioblastoma xenografts [J].

Grzelinski, Marius ;

Urban-Klein, Beata ;

Martens, Tobias ;

Lamszus, Katrin ;

Bakowsky, Udo ;

Hoebel, Sabrina ;

Czubayko, Frank ;

Aigner, Achim .

HUMAN GENE THERAPY, 2006, 17 (07) :751-766

[48] Engineering RNA for Targeted siRNA Delivery and Medical Application [J].

Guo, Peixuan ;

Coban, Oana ;

Snead, Nicholas M. ;

Trebley, Joe ;

Hoeprich, Steve ;

Guo, Songchuan ;

Shu, Yi .

ADVANCED DRUG DELIVERY REVIEWS, 2010, 62 (06) :650-666

[49] Steric stabilization of fusogenic liposomes by a low-pH sensitive PEG-diortho ester-lipid conjugate [J].

Guo, X ;

Szoka, FC .

BIOCONJUGATE CHEMISTRY, 2001, 12 (02) :291-300

[50] Lipid-mediated siRNA delivery down-regulates exogenous gene expression in the mouse brain at picomolar levels [J].

Hassani, Z ;

Lemkine, GF ;

Erbacher, P ;

Palmier, K ;

Alfama, G ;

Giovannangeli, C ;

Behr, JP ;

Demeneix, BA .

JOURNAL OF GENE MEDICINE, 2005, 7 (02) :198-207

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