Nonviral delivery of synthetic siRNAs in vivo

被引：389

作者：

Akhtar, Saghir

Benter, Ibrahim F.

机构：

[1] Kuwait Univ, Hlth Sci Ctr, Fac Med, Dept Pharmacol & Toxicol, Safat 13110, Kuwait

[2] SA Pharma, Sutton Coldfield, W Midlands, England

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2007年 / 117卷 / 12期

关键词：

D O I：

10.1172/JCI33494

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Sequence-specific gene silencing using small interfering RNA (siRNA) is a Nobel prize-winning technology that is now being evaluated in clinical trials as a potentially novel therapeutic strategy. This article provides an overview of the major pharmaceutical challenges facing siRNA therapeutics, focusing on the delivery strategies for synthetic siRNA duplexes in vivo, as this remains one of the most important issues to be resolved. This article also highlights the importance of understanding the genocompatibility/toxicogenomics of siRNA delivery reagents in terms of their impact on gene-silencing activity and specificity. Collectively, this information is essential for the selection of optimally acting siRNA delivery system combinations for the many proposed applications of RNA interference.

引用

收藏

页码：3623 / 3632

页数：10

相关论文

共 123 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] Delivery systems for the direct application of siRNAs to induce RNA interference (RNAi) in vivo [J].

Aigner, Achim .

JOURNAL OF BIOMEDICINE AND BIOTECHNOLOGY, 2006,

[3] RNAi-induced gene silencing by local electroporation in targeting brain region [J].

Akaneya, Y ;

Jiang, B ;

Tsumoto, T .

JOURNAL OF NEUROPHYSIOLOGY, 2005, 93 (01) :594-602

[4]

Akhtar S, 2006, GENE THER, V13, P739, DOI 10.1038/sj.gt.3302692

[5] In vivo studies with antisense oligonucleotides [J].

Akhtar, S ;

Agrawal, S .

TRENDS IN PHARMACOLOGICAL SCIENCES, 1997, 18 (01) :12-18

[6] The delivery of antisense therapeutics [J].

Akhtar, S ;

Hughes, MD ;

Khan, A ;

Bibby, M ;

Hussain, M ;

Nawaz, Q ;

Double, J ;

Sayyed, P .

ADVANCED DRUG DELIVERY REVIEWS, 2000, 44 (01) :3-21

[7] Toxicogenomics of non-viral drug delivery systems for RNAi: Potential impact on siRNA-mediated gene silencing activity and specificity [J].

Akhtar, Saghir ;

Benter, Ibrahim .

ADVANCED DRUG DELIVERY REVIEWS, 2007, 59 (2-3) :164-182

[8] Specific β1-adrenergic receptor silencing with small interfering RNA lowers high blood pressure and improves cardiac function in myocardial ischemia [J].

Arnold, Anne-Sophie ;

Tang, Yao Liang ;

Qian, Keping ;

Shen, Leping ;

Valencia, Valery ;

Phillips, Michael Ian ;

Zhang, Yuan Clare .

JOURNAL OF HYPERTENSION, 2007, 25 (01) :197-205

[9] Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging [J].

Bartlett, DW ;

Davis, ME .

NUCLEIC ACIDS RESEARCH, 2006, 34 (01) :322-333

[10] Gene silencing nucleic acids designed by scanning arrays: Anti-EGFR activity of siRNA, ribozyme and DNA enzymes targeting a single hybridization-accessible region using the same delivery system [J].

Beale, G ;

Hollins, AJ ;

Benboubetra, M ;

Sohail, M ;

Fox, SP ;

Benter, I ;

Akhtar, S .

JOURNAL OF DRUG TARGETING, 2003, 11 (07) :449-456

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