Developing siRNA therapies to address osteoporosis

Therapeutic Delivery

Volumn 4, Issue 10, 2013, Pages 1239-1246

  Wang, Yuwei ^a   Grainger, David W ^a  

^a UNIVERSITY OF UTAH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84885580339     PISSN: 20415990     EISSN: 20416008     Source Type: Journal    
DOI: 10.4155/tde.13.85     Document Type: Article

References (42)

1. Hollick RJ, Reid DM. Role of bisphosphonates in the management of postmenopausal osteoporosis: an update on recent safety anxieties. Menopause Int. 17(2), 66-72 (2011).
2. Schilcher J, Michaelsson K, Aspenberg P. Bisphosphonate use and atypical fractures of the femoral shaft. N. Engl. J. Med. 364(18), 1728-1737 (2011).
3. Jilka RL. Molecular and cellular mechanisms of the anabolic effect of intermittent PTH. Bone 40(6), 1434-1446 (2007).
4. Wang Y, Nishida S, Boudignon BM et al. IGF-I receptor is required for the anabolic actions of parathyroid hormone on bone. J. Bone Miner. Res. 22(9), 1329-1337 (2007).
5. Bikle DD, Sakata T, Leary C et al. Insulin-like growth factor I is required for the anabolic actions of parathyroid hormone on mouse bone. J. Bone Miner. Res. 17(9), 1570-1578 (2002).
6. Aagaard L, Rossi JJ. RNAi therapeutics: principles, prospects and challenges. Adv. Drug Deliv. Rev. 59(2-3), 75-86 (2007).
7. Haussecker D. The business of RNAi therapeutics in 2012. Mol. Ther. Nucleic Acids 1, e8 (2012).
8. Gogakos AI, Bassett JHD, Williams GR. Bone signaling pathways and treatment of osteoporosis. Expert Rev. Endocrinol. Metab. 4(6), 639-650 (2009).
9. Wang Y, Grainger DW. RNA therapeutics targeting osteoclast-mediated excessive bone resorption. Adv. Drug Deliv. Rev. 64(12), 1341-1357 (2012).
10. Li X, Ominsky MS, Warmington KS et al. Sclerostin antibody treatment increases bone formation, bone mass, and bone strength in a rat model of postmenopausal osteoporosis. J. Bone Miner. Res. 24(4), 578-588 (2009).
11. Morvan F, Boulukos K, Clement-Lacroix P et al. Deletion of a single allele of the Dkk1 gene leads to an increase in bone formation and bone mass. J. Bone Miner. Res. 21(6), 934-945 (2006).
12. Mcnamara JO 2nd, Andrechek ER, Wang Y et al. Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat. Biotechnol. 24(8), 1005-1015 (2006).
13. Lee NJ, Nguyen AD, Enriquez RF et al. Osteoblast specific Y1 receptor deletion enhances bone mass. Bone 48(3), 461-467 (2011).
14. Buckbinder L, Crawford DT, Qi H et al. Proline-rich tyrosine kinase 2 regulates osteoprogenitor cells and bone formation, and offers an anabolic treatment approach for osteoporosis. Proc. Natl Acad. Sci. USA 104(25), 10619-10624 (2007).
15. Shi Q, Zhang XL, Dai KR, Benderdour M, Fernandes JC. siRNA therapy for cancer and non-lethal diseases such as arthritis and osteoporosis. Expert Opin. Biol. Ther. 11(1), 5-16 (2011).
16. You L, Pan L, Chen L et al. Suppression of zinc finger protein 467 alleviates osteoporosis
17. Rey JP, Ellies DL. Wnt modulators in the biotech pipeline. Dev. Dyn. 239(1), 102-114 (2010).
18. Yeo H, Mcdonald JM, Zayzafoon M. NFATc1: a novel anabolic therapeutic target for osteoporosis. Ann. NY Acad. Sci. 1068, 564-567 (2006).
19. Lacal JC. Regulation of proliferation and apoptosis by Ras and Rho GTPases through specific phospholipid-dependent signaling. FEBS Lett. 410(1), 73-77 (1997).
20. Tenenbaum HC, Shelemay A, Girard B, Zohar R, Fritz PC. Bisphosphonates and periodontics: potential applications for regulation of bone mass in the periodontium and other therapeutic/diagnostic uses. J. Periodontol. 73(7), 813-822 (2002).
21. Reinholz GG, Getz B, Sanders ES et al. Distinct mechanisms of bisphosphonate action between osteoblasts and breast cancer cells: identity of a potent new bisphosphonate analogue. Breast Cancer Res. Treat. 71(3), 257-268 (2002).
22. Wang Y, Panasiuk A, Grainger DW. Small interfering RNA knocks down the molecular target of alendronate, farnesyl pyrophosphate synthase, in osteoclast and osteoblast cultures. Mol. Pharm. 8(4), 1016-1024 (2011).
23. Fahid FS, Jiang J, Zhu Q et al. Application of small interfering RNA for inhibition of lipopolysaccharide-induced osteoclast formation and cytokine stimulation. J. Endod. 34(5), 563-569 (2008).
24. Sundaram K, Nishimura R, Senn J, Youssef RF, London SD, Reddy SV. RANK ligand signaling modulates the matrix metalloproteinase-9 gene expression during osteoclast differentiation. Exp. Cell Res. 313(1), 168-178 (2007).
25. Yeo H, Beck LH, Mcdonald JM, Zayzafoon M. Cyclosporin A elicits dose-dependent biphasic effects on osteoblast differentiation and bone formation. Bone 40(6), 1502-1516 (2007).
26. Tiemann K, Rossi JJ. RNAi-based therapeutics-current status, challenges and prospects. EMBO Mol. Med. 1(3), 142-151 (2009).
27. Gary DJ, Puri N, Won YY. Polymer-based siRNA delivery: perspectives on the fundamental and phenomenological distinctions from polymer-based DNA delivery. J. Control. Release 121(1-2), 64-73 (2007).
28. Mosna F, Sensebe L, Krampera M. Human bone marrow and adipose tissue mesenchymal stem cells: a user's guide. Stem Cells Dev. 19(10), 1449-1470 (2010).
29. Tzeng SY, Hung BP, Grayson WL, Green JJ. Cystamine-Terminated poly(beta-Amino ester)s for siRNA delivery to human mesenchymal stem cells and enhancement of osteogenic differentiation. Biomaterials 33(32), 8142-8151 (2012).
30. Benoit DS, Boutin ME. Controlling mesenchymal stem cell gene expression using polymer-mediated delivery of siRNA. Biomacromolecules 13(11), 3841-3849 (2012).
31. Ducy P, Zhang R, Geoffroy V, Ridall AL, Karsenty G. Osf2/Cbfa1: a transcriptional activator of osteoblast differentiation. Cell 89(5), 747-754 (1997).
32. Rios CN, Skoracki RJ, Mathur AB. GNAS1 and PHD2 short-interfering RNA support bone regeneration in vitro and in an in vivo sheep model. Clin. Orthop. Relat. Res. 470(9), 2541-2553 (2012).
33. Ramasubramanian A, Shiigi S, Lee GK, Yang F. Non-viral delivery of inductive and suppressive genes to adipose-derived stem cells for osteogenic differentiation. Pharm. Res. 28(6), 1328-1337 (2011).
34. Andersen MO, Nygaard JV, Burns JS et al. siRNA nanoparticle functionalization of nanostructured scaffolds enables controlled multilineage differentiation of stem cells. Mol. Ther. 18(11), 2018-2027 (2010).
35. Leachman SA, Hickerson RP, Schwartz ME et al. First-in-human mutation-Targeted siRNA Phase Ib trial of an inherited skin disorder. Mol. Ther. 18(2), 442-446 (2010).
36. Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference- based therapeutics. Nature 457(7228), 426-433 (2009).
37. Zhang G, Guo B, Wu H et al. A delivery system targeting bone formation surfaces to facilitate RNAi-based anabolic therapy. Nat. Med. 18(2), 307-314 (2012).
38. Wang D, Miller SC, Shlyakhtenko LS et al. Osteotropic peptide that differentiates functional domains of the skeleton. Bioconjug. Chem. 18(5), 1375-1378 (2007).
39. Yarbrough DK, Hagerman E, Eckert R et al. Specific binding and mineralization of calcified surfaces by small peptides. Calcif. Tissue Int. 86(1), 58-66 (2010).
40. Manaka T, Suzuki A, Takayama K, Imai Y, Nakamura H, Takaoka K. Local delivery of siRNA using a biodegradable polymer application to enhance BMP-induced bone formation. Biomaterials 32(36), 9642-9648 (2011).
41. Kwong FN, Richardson SM, Evans CH. Chordin knockdown enhances the osteogenic differentiation of human mesenchymal stem cells. Arthritis Res. Ther. 10(3), R65 (2008).
42. Wang Y, Tran KK, Shen H, Grainger DW. Selective local delivery of RANK siRNA to bone phagocytes using bone augmentation biomaterials. Biomaterials 33(33), 8540-8547 (2012).