Biology Reference
In-Depth Information
Acknowledgments and Author Disclosure Statements
This work is supported by grants from
the National Institutes of Health AI29329, AI42552, and HL07470 awarded to J.J.R.
J.Z. drafted the article. J.J.R. revised it and gave final approval of the version to be published.
All authors read and approved the final article.
The authors declare no competing financial interests.
References
1. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T (2001) Duplexes of
21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature
411:494-498
2. Zamore PD, Tuschl T, Sharp PA, Bartel DP (2000) RNAi: double-stranded RNA directs the
ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell 101:25-33
3. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC (1998) Potent and specific
genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391:806-811
4. Davidson BL, McCray PB Jr (2011) Current prospects for RNA interference-based therapies.
Nat Rev Genet 12:329-340
5. Castanotto D, Rossi JJ (2009) The promises and pitfalls of RNA-interference-based therapeu-
tics. Nature 457:426-433
6. Lares MR, Rossi JJ, Ouellet DL (2010) RNAi and small interfering RNAs in human disease
therapeutic applications. Trends Biotechnol 28:570-579
7. Singerman L (2009) Combination therapy using the small interfering RNA bevasiranib. Retina
29:S49-S50
8. DeVincenzo J et al (2008) Evaluation of the safety, tolerability and pharmacokinetics of ALN-
RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV).
Antiviral Res 77:225-231
9. DeVincenzo J et al (2010) A randomized, double-blind, placebo-controlled study of an RNAi-
based therapy directed against respiratory syncytial virus. Proc Natl Acad Sci USA
107:8800-8805
10. Alvarez-Erviti L, Seow Y, Yin H, Betts C, Lakhal S, Wood MJ (2011) Delivery of siRNA to
the mouse brain by systemic injection of targeted exosomes. Nat Biotechnol 29:341-345
11. Zamora MR et al (2011) RNA interference therapy in lung transplant patients infected with
respiratory syncytial virus. Am J Respir Crit Care Med 183:531-538
12. Davis ME et al (2010) Evidence of RNAi in humans from systemically administered siRNA
via targeted nanoparticles. Nature 464:1067-1070
13. Whitehead KA, Langer R, Anderson DG (2009) Knocking down barriers: advances in siRNA
delivery. Nat Rev Drug Discov 8:129-138
14. Juliano R, Alam MR, Dixit V, Kang H (2008) Mechanisms and strategies for effective delivery
of antisense and siRNA oligonucleotides. Nucleic Acids Res 36:4158-4171
15. Perez-Martinez FC, Guerra J, Posadas I, Cena V (2011) Barriers to non-viral vector-mediated
gene delivery in the nervous system. Pharm Res 28:1843-1858
16. Wang J, Lu Z, Wientjes MG, Au JL (2010) Delivery of siRNA therapeutics: barriers and carri-
ers. AAPS J 12:492-503
17. Kaiser PK et al (2010) RNAi-based treatment for neovascular age-related macular degenera-
tion by Sirna-027. Am J Ophthalmol 150(33-39):e32
18. Kleinman ME et al (2008) Sequence- and target-independent angiogenesis suppression by
siRNA via TLR3. Nature 452:591-597
19. Shim MS, Kwon YJ (2010) Efficient and targeted delivery of siRNA in vivo. FEBS J
277:4814-4827
20. Zhou J, Rossi JJ (2009) The therapeutic potential of cell-internalizing aptamers. Curr Top Med
Chem 9:1144-1157
Search WWH ::
Custom Search