BMB Reports

Korean Society for Biochemistry and Molecular Biology (생화학분자생물학회)
권호 표지
DOI QR코드

DOI QR Code

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)
  • Received : 2014.04.29
  • Accepted : 2014.07.31
  • Published : 2015.03.31
Download PDF
⟨ Previous Next ⟩

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

References

  1. 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 https://doi.org/10.1038/35888
  2. 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 https://doi.org/10.1038/35078107
  3. 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 https://doi.org/10.1016/j.addr.2010.03.008
  4. Lima WF, Prakash TP, Murray HM et al (2012) Single-stranded siRNAs activate RNAi in animals. Cell 150, 883-894 https://doi.org/10.1016/j.cell.2012.08.014
  5. de Fougerolles AR (2008) Delivery vehicles for small interfering RNA in vivo. Hum Gene Ther 19, 125-132 https://doi.org/10.1089/hum.2008.928
  6. Chen M, Gao S, Dong M et al (2012) Chitosan/siRNA nanoparticles encapsulated in PLGA nanofibers for siRNA delivery. ACS Nano 6, 4835-4844 https://doi.org/10.1021/nn300106t
  7. 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 https://doi.org/10.1016/j.jconrel.2014.01.008
  8. Zimmermann TS, Lee AC, Akinc A et al (2006) RNAimediated gene silencing in non-human primates. Nature 441, 111-114 https://doi.org/10.1038/nature04688
  9. Ahmed Z, Kalinski H, Berry M et al (2011) Ocular neuroprotection by siRNA targeting caspase-2. Cell Death Dis 2, e173 https://doi.org/10.1038/cddis.2011.54
  10. 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 https://doi.org/10.1038/mt.2013.217
  11. 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 https://doi.org/10.1038/mt.2013.216
  12. 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 https://doi.org/10.1016/j.cell.2012.08.002
  13. 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 https://doi.org/10.1073/pnas.0708285104
  14. 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 https://doi.org/10.1038/nbt.1560
  15. 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 https://doi.org/10.1016/j.biomaterials.2013.12.054
  16. 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 https://doi.org/10.1016/j.jconrel.2013.10.003
  17. 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 https://doi.org/10.1126/scitranslmed.3007048
  18. 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 https://doi.org/10.1016/j.jconrel.2014.01.012
  19. 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 https://doi.org/10.1073/pnas.0806044105

Cited by

  1. Release of siRNA from Liposomes Induced by Curcumin vol.2016, 2016, https://doi.org/10.1155/2016/7051523
  2. Dendrosome mediated topical gene silencing by PLK-1 specific siRNA: implication in treatment of skin cancer in mouse model vol.6, pp.8, 2016, https://doi.org/10.1039/C5RA15270D
  3. Inducible Lentivirus-Mediated siRNA against TLR4 Reduces Nociception in a Rat Model of Bone Cancer Pain vol.2015, 2015, https://doi.org/10.1155/2015/523896
  4. A Convenient In Vivo Model Using Small Interfering RNA Silencing to Rapidly Assess Skeletal Gene Function vol.11, pp.11, 2016, https://doi.org/10.1371/journal.pone.0167222
  5. ClC-7 Deficiency Impairs Tooth Development and Eruption vol.6, pp.1, 2016, https://doi.org/10.1038/srep19971
  6. Identification of microRNAs involved in gefitinib resistance of non-small-cell lung cancer through the insulin-like growth factor receptor 1 signaling pathway vol.14, pp.4, 2017, https://doi.org/10.3892/etm.2017.4847
  7. Strategies for improving the specificity of siRNAs for enhanced therapeutic potential vol.13, pp.8, 2018, https://doi.org/10.1080/17460441.2018.1480607