Browse > Article
http://dx.doi.org/10.5483/BMBRep.2015.48.3.089

New paradigms on siRNA local application  

Pan, Meng (State Key Laboratory of Military Stomatology, Department of Oral Biology, School of Stomatology, the Fourth Military Medical University)
Ni, Jinwen (State Key Laboratory of Military Stomatology, Department of Oral Biology, School of Stomatology, the Fourth Military Medical University)
He, Huiming (State Key Laboratory of Military Stomatology, Department of Prosthodontics, School of Stomatology, the Fourth Military Medical University)
Gao, Shan (School of Stomatology, Central South University)
Duan, Xiaohong (State Key Laboratory of Military Stomatology, Department of Oral Biology, School of Stomatology, the Fourth Military Medical University)
Publication Information
BMB Reports / v.48, no.3, 2015 , pp. 147-152 More about this Journal
Abstract
Small interfering RNA (siRNA) functions through pairing with specific mRNA sequences and results in the mRNA's degradation. It is a potential therapeutic approach for many diseases caused by altered gene expression. The delivery of siRNA is still a major problem due to its rapid degradation in the circulation. Various strategies have been proposed to help with the cellular uptake of siRNA and short or small hairpin RNA (shRNA). Here, we reviewed recently published data regarding local applications of siRNA. Compared with systemic delivery methods, local delivery of siRNA/shRNA has many advantages, such as targeting the specific tissues or organs, mimicking a gene knockout effect, or developing certain diseases models. The eye, brain, and tumor tissues are 'hot' target tissues/organs for local siRNA delivery. The siRNA can be delivered locally, in naked form, with chemical modifications, or in formulations with viral or non-viral vectors, such as liposomes and nanoparticles. This review provides a comprehensive overview of RNAi local administration and potential future applications in clinical treatment.
Keywords
In vivo; Local injection; shRNA; siRNA;
Citations & Related Records
연도 인용수 순위
  • Reference
1 Chen M, Gao S, Dong M et al (2012) Chitosan/siRNA nanoparticles encapsulated in PLGA nanofibers for siRNA delivery. ACS Nano 6, 4835-4844   DOI   ScienceOn
2 DiFiglia M, Sena-Esteves M, Chase K et al (2007) Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits. Proc Natl Acad Sci U S A 104, 17204-17209   DOI   ScienceOn
3 Lima WF, Prakash TP, Murray HM et al (2012) Single-stranded siRNAs activate RNAi in animals. Cell 150, 883-894   DOI   ScienceOn
4 de Fougerolles AR (2008) Delivery vehicles for small interfering RNA in vivo. Hum Gene Ther 19, 125-132   DOI   ScienceOn
5 Pittella F, Cabral H, Maeda Y et al (2014) Systemic siRNA delivery to a spontaneous pancreatic tumor model in transgenic mice by PEGylated calcium phosphate hybrid micelles. J Control Release 178C, 18-24   DOI   ScienceOn
6 Zimmermann TS, Lee AC, Akinc A et al (2006) RNAimediated gene silencing in non-human primates. Nature 441, 111-114   DOI   ScienceOn
7 Ahmed Z, Kalinski H, Berry M et al (2011) Ocular neuroprotection by siRNA targeting caspase-2. Cell Death Dis 2, e173   DOI
8 Moreno-Montañés J, Sádaba B, Ruz V et al (2014) Phase I clinical trial of SYL040012, a small interfering RNA targeting β-adrenergic receptor 2, for lowering intraocular pressure. Mol Ther 22, 226-232   DOI
9 Martínez T, González MV, Roehl I, Wright N, Pañeda C and Jiménez AI (2014) In vitro and in vivo efficacy of SYL040012, a novel siRNA compound for treatment of glaucoma. Mol Ther 22, 81-91   DOI   ScienceOn
10 Guo P, Coban O, Snead NM et al (2010) Engineering RNA for targeted siRNA delivery and medical application. Adv Drug Deliv Rev 62, 650-666   DOI   ScienceOn
11 Yu D, Pendergraff H, Liu J et al (2012) Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell 150, 895-908   DOI   ScienceOn
12 Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE and Mello CC (1998) Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391, 806-811   DOI   ScienceOn
13 Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K and Tuschl T (2001) Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411, 494-498   DOI   ScienceOn
14 Lai Kwan Lam Q, King Hung Ko O, Zheng BJ and Lu L (2008) Local BAFF gene silencing suppresses Th17-cell generation and ameliorates autoimmune arthritis. Proc Natl Acad Sci U S A 105, 14993-14998   DOI   ScienceOn
15 Brock A, Krause S, Li H et al (2014) Silencing HoxA1 by intraductal injection of siRNA lipidoid nanoparticles prevents mammary tumor progression in mice. Sci Transl Med 6, 217ra2   DOI   ScienceOn
16 Dassie JP, Liu XY, Thomas GS et al (2009) Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors. Nat Biotechnol 27, 839-849   DOI   ScienceOn
17 Lai WY, Wang WY, Chang YC, Chang CJ, Yang PC and Peck K (2014) Synergistic inhibition of lung cancer cell invasion, tumor growth and angiogenesis using aptamersiRNA chimeras. Biomaterials 35, 2905-2914   DOI   ScienceOn
18 Sakurai Y, Hatakeyama H, Sato Y et al (2014) RNAi-mediated gene knockdown and anti-angiogenic therapy of RCCs using a cyclic RGD-modified liposomal-siRNA system. J Control Release 173, 110-118   DOI   ScienceOn
19 Wang J, Lu Z, Yeung BZ, Wientjes MG, Cole DJ and Au JL (2014) Tumor priming enhances siRNA delivery and transfection in intraperitoneal tumors. J Control Release 178, 79-85   DOI   ScienceOn