Aptamer-functionalized lipid nanoparticles targeting osteoblasts as a novel RNA interference-based bone anabolic strategy

Nature Medicine

Volumn 21, Issue 3, 2015, Pages 288-294

  Liang, Chao ^a,b,c,d   Guo, Baosheng ^a,b,d   Wu, Heng ^a,d   Shao, Ningsheng ^e   Li, Defang ^a,d   Liu, Jin ^a,d   Dang, Lei ^a,d   Wang, Cheng ^a,f   Li, Hui ^e   Li, Shaohua ^e   Lau, Wing Ki ^a   Cao, Yu ^c   Yang, Zhijun ^a,d   Lu, Cheng ^a,b,d   He, Xiaojuan ^a,b,d   Au, D W T ^g   Pan, Xiaohua ^a   Zhang, Bao Ting ^h   Lu, Changwei ^a   Zhang, Hongqi ^a   Yue, Kinman ^a   Qian, Airong ^a,i   Shang, Peng ^a,i   Xu, Jiake ^j   Xiao, Lianbo ^a,k   Bian, Zhaoxiang ^a,d   Tan, Weihong ^a,l,m,n,o   Liang, Zicai ^d   He, Fuchu ^c   Zhang, Lingqiang ^c   Lu, Aiping ^a,b,d,k   Zhang, Ge ^a,b,d,i,k   more..

^a HONG KONG BAPTIST UNIVERSITY   (Hong Kong)

^b INSTITUTE OF CHINESE MATERIA MEDICA   (China)

^c BEIJING INSTITUTE OF RADIATION MEDICINE   (China)

^d Kunshan Industrial Technology Research Institute   (China)

^e INSTITUTE OF BASIC MEDICAL SCIENCES   (China)

^f OCEAN UNIVERSITY OF CHINA   (China)

^g CITY UNIVERSITY OF HONG KONG   (Hong Kong)

^h and Hong Kong Branch of CAS Center for Excellence in Animal Evolution and Genetics   (Hong Kong)

ⁱ NORTHWESTERN POLYTECHNICAL UNIVERSITY   (China)

^j UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^k Institute of Arthritis Research Shanghai Academy of Chinese Medical Sciences   (China)

^l HUNAN UNIVERSITY   (China)

^m Key Laboratory for Space Bioscience and Biotechnology Institute of Special Environmental Biophysics Northwestern Polytechnical University ^*  (United States)

ⁿ UNIVERSITY OF FLORIDA   (United States)

^o UNIVERSITY OF FLORIDA   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

APTAMER; CASEIN KINASE II; LIPID; NANOPARTICLE; PLECKSTRIN HOMOLOGY DOMAIN CONTAINING FAMILY O MEMBER 1; SMALL INTERFERING RNA; UNCLASSIFIED DRUG; PLEKHO1 PROTEIN, HUMAN; SIGNAL PEPTIDE;

ANIMAL CELL; ARTICLE; BONE MASS; COMBINATORIAL CHEMISTRY; CONTROLLED STUDY; GENE SILENCING; HUMAN; HUMAN CELL; NONHUMAN; OSSIFICATION; OSTEOBLAST; PINOCYTOSIS; PRIORITY JOURNAL; RAT; RNA INTERFERENCE; ANIMAL; BONE DEVELOPMENT; DRUG DELIVERY SYSTEM; GENETICS; METABOLISM;

ANIMALS; APTAMERS, NUCLEOTIDE; DRUG DELIVERY SYSTEMS; GENE SILENCING; HUMANS; INTRACELLULAR SIGNALING PEPTIDES AND PROTEINS; LIPIDS; NANOPARTICLES; OSTEOBLASTS; OSTEOGENESIS; RATS; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 84925516251     PISSN: 10788956     EISSN: 1546170X     Source Type: Journal    
DOI: 10.1038/nm.3791     Document Type: Article

References (35)

1. López-Fraga, M., Martinez, T. & Jimenez, A. RNA interference technologies and therapeutics: from basic research to products. BioDrugs 23, 305-332 (2009).
2. Novina, C.D. & Sharp, P.A. The RNAi revolution. Nature 430, 161-164 (2004).
3. Wang, Y. & Grainger, D.W. RNA therapeutics targeting osteoclast-mediated excessive bone resorption. Adv. Drug Deliv. Rev. 64, 1341-1357 (2012).
4. Lu, K. et al. Targeting WW domains linker of HECT-type ubiquitin ligase Smurf1 for activation by CKIP-1. Nat. Cell Biol. 10, 994-1002 (2008).
5. Zhang, G. et al. A delivery system targeting bone formation surfaces to facilitate RNAi-based anabolic therapy. Nat. Med. 18, 307-314 (2012).
6. Wang, D., Miller, S.C., Kopeckova, P. & Kopecek, J. Bone-targeting macromolecular therapeutics. Adv. Drug Deliv. Rev. 57, 1049-1076 (2005).
7. Wang, X. et al. miR-214 targets ATF4 to inhibit bone formation. Nat. Med. 19, 93-100 (2013).
8. Li, S.D. & Huang, L. Pharmacokinetics and biodistribution of nanoparticles. Mol. Pharm. 5, 496-504 (2008).
9. Zamboni, W.C. Liposomal, nanoparticle, and conjugated formulations of anticancer agents. Clin. Cancer Res. 11, 8230-8234 (2005).
10. Wang, G., Kucharski, C., Lin, X. & Uludag, H. Bisphosphonate-coated BSA nanoparticles lack bone targeting after systemic administration. J. Drug Target. 18, 611-626 (2010).
11. Akhtar, S. & Benter, I.F. Nonviral delivery of synthetic siRNAs in vivo. J. Clin. Invest. 117, 3623-3632 (2007).
12. Ye, M. et al. Generating aptamers by cell-SELEX for applications in molecular medicine. Int. J. Mol. Sci. 13, 3341-3353 (2012).
13. Sefah, K., Shangguan, D., Xiong, X., O'Donoghue, M.B. & Tan, W. Development of DNA aptamers using cell-SELEX. Nat. Protoc. 5, 1169-1185 (2010).
14. Tam, Y.Y., Chen, S. & Cullis, P.R. Advances in lipid nanoparticles for siRNA delivery. Pharmaceutics 5, 498-507 (2013).
15. Auguste, D.T. et al. Triggered release of siRNA from poly(ethylene glycol)-protected, pH-dependent liposomes. J. Control. Release 130, 266-274 (2008).
16. Mui, B.L. et al. Influence of polyethylene glycol lipid desorption rates on pharmacokinetics and pharmacodynamics of siRNA lipid nanoparticles. Mol. Ther. Nucleic Acids 2, e139 (2013).
17. Rockey, W.M. et al. Rational truncation of an RNA aptamer to prostate-specific membrane antigen using computational structural modeling. Nucleic Acid Ther. 21, 299-314 (2011).
18. Keefe, A.D., Pai, S. & Ellington, A. Aptamers as therapeutics. Nat. Rev. Drug Discov. 9, 537-550 (2010).
19. Gilbert, J.C. et al. First-in-human evaluation of anti von Willebrand factor therapeutic aptamer ARC1779 in healthy volunteers. Circulation 116, 2678-2686 (2007).
20. Cao, H., Chen, J., Awoniyi, M., Henley, J.R. & McNiven, M.A. Dynamin 2 mediates fluid-phase micropinocytosis in epithelial cells. J. Cell Sci. 120, 4167-4177 (2007).
21. Vercauteren, D. et al. The use of inhibitors to study endocytic pathways of gene carriers: optimization and pitfalls. Mol. Ther. 18, 561-569 (2010).
22. Lim, J.P. & Gleeson, P.A. Macropinocytosis: an endocytic pathway for internalising large gulps. Immunol. Cell Biol. 89, 836-843 (2011).
23. Zernik, J., Twarog, K. & Upholt, W.B. Regulation of alkaline phosphatase and alpha 2(I) procollagen synthesis during early intramembranous bone formation in the rat mandible. Differentiation 44, 207-215 (1990).
24. Fukushima, N., Hiraoka, K., Shirachi, I., Kojima, M. & Nagata, K. Isolation and characterization of a novel peptide, osteoblast activating peptide (OBAP), associated with osteoblast differentiation and bone formation. Biochem. Biophys. Res. Commun. 400, 157-163 (2010).
25. Ishikawa, S. et al. Involvement of FcRgamma in signal transduction of osteoclast- associated receptor (OSCAR). Int. Immunol. 16, 1019-1025 (2004).
26. Herman, S. et al. Induction of osteoclast-associated receptor, a key osteoclast costimulation molecule, in rheumatoid arthritis. Arthritis Rheum. 58, 3041-3050 (2008).
27. Lotinun, S. et al. Osteoclast-specific cathepsin K deletion stimulates S1P-dependent bone formation. J. Clin. Invest. 123, 666-681 (2013).
28. Guo, B. et al. Therapeutic RNA interference targeting CKIP-1 with a cross-species sequence to stimulate bone formation. Bone 59, 76-88 (2014).
29. Sanguineti, R., Storace, D., Monacelli, F., Federici, A. & Odetti, P. Pentosidine effects on human osteoblasts in vitro. Ann. NY Acad. Sci. 1126, 166-172 (2008).
30. Kishimoto, Y. et al. Gene expression relevant to osteoclastogenesis in the synovium and bone marrow of mature rats with collagen-induced arthritis. Rheumatology (Oxford) 43, 1496-1503 (2004).
31. Susa, M., Luong-Nguyen, N.H., Cappellen, D., Zamurovic, N. & Gamse, R. Human primary osteoclasts: in vitro generation and applications as pharmacological and clinical assay. J. Transl. Med. 2, 6 (2004).
32. Yang, M. et al. Developing aptamer probes for acute myelogenous leukemia detection and surface protein biomarker discovery. J.Hematol. Oncol. 7, 5 (2014).
33. Wu, H. et al. Preparation of aptamer-functionalized lipid nanoparticles (LNPs) encapsulating siRNAs. Protocol Exchange doi:10.1038/protex.2014.053 (2014).
34. Hao, Y.J. et al. Changes of microstructure and mineralized tissue in the middle and late phase of osteoporotic fracture healing in rats. Bone 41, 631-638 (2007).
35. Semple, S.C. et al. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol. 28, 172-176 (2010).