Abstract
Over the last few years, huge progress has been made in the understanding of molecular mechanisms underlying the pathogenesis of retinal degenerative diseases. Such knowledge has led to the development of gene therapy approaches to treat these devastating disorders. Non-viral gene delivery has been recognized as a prospective treatment for retinal degenerative diseases. In this review, we will summarize the constituent characteristics and recent applications of three representative nanoparticles (NPs) in ocular therapy.
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Adijanto J, Naash MI (2015) Nanoparticle-based technologies for retinal gene therapy. Eur J Pharm Biopharm 95(Pt B):353–367
Cai X, McGinnis JF (2016a) Diabetic retinopathy: animal models, therapies, and perspectives. J Diabetes Res 2016:3789217
Cai X, McGinnis JF (2016b) Nanoceria: a potential therapeutic for dry AMD. Adv Exp Med Biol 854:111–118
Cai X, Conley SM, Nash Z et al (2010) Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa. FASEB J 24:1178–1191
Cai X, Seal S, McGinnis JF (2014a) Sustained inhibition of neovascularization in vldlr−/− mice following intravitreal injection of cerium oxide nanoparticles and the role of the ASK1-P38/JNK-NF-kappaB pathway. Biomaterials 35:249–258
Cai X, Yodoi J, Seal S et al (2014b) Nanoceria and thioredoxin regulate a common antioxidative gene network in tubby mice. Adv Exp Med Biol 801:829–836
Chen J, Patil S, Seal S et al (2006) Rare earth nanoparticles prevent retinal degeneration induced by intracellular peroxides. Nat Nanotechnol 1:142–150
Conley SM, Naash MI (2010) Nanoparticles for retinal gene therapy. Prog Retin Eye Res 29:376–397
del Pozo-Rodriguez A, Delgado D, Gascon AR et al (2013) Lipid nanoparticles as drug/gene delivery systems to the retina. J Ocul Pharmacol Ther 29:173–188
Han Z, Conley SM, Naash MI (2011) AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects. Invest Ophthalmol Vis Sci 52:3051–3059
Han Z, Conley SM, Makkia R et al (2012a) Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery. PLoS One 7:e52189
Han Z, Conley SM, Makkia RS et al (2012b) DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice. J Clin Invest 122:3221–3226
Han Z, Banworth MJ, Makkia R et al (2015) Genomic DNA nanoparticles rescue rhodopsin-associated retinitis pigmentosa phenotype. FASEB J 29:2535–2544
Honda M, Asai T, Oku N et al (2013) Liposomes and nanotechnology in drug development: focus on ocular targets. Int J Nanomedicine 8:495–503
Koirala A, Conley SM, Makkia R et al (2013a) Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases. J Control Release 172:745–752
Koirala A, Makkia RS, Conley SM et al (2013b) S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA. Hum Mol Genet 22:1632–1642
Koirala A, Conley SM, Naash MI (2014) Episomal maintenance of S/MAR-containing non-viral vectors for RPE-based diseases. Adv Exp Med Biol 801:703–709
Kong L, Cai X, Zhou X et al (2011) Nanoceria extend photoreceptor cell lifespan in tubby mice by modulation of apoptosis/survival signaling pathways. Neurobiol Dis 42:514–523
Li S, Huang L (1997) In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther 4:891–900
Li S, Rizzo MA, Bhattacharya S et al (1998) Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Ther 5:930–937
Ma K, Wang DD, Lin Y et al (2013) Synergetic targeted delivery of sleeping-beauty transposon system to mesenchymal stem cells using LPD nanoparticles modified with a phage-displayed targeting peptide. Adv Funct Mater 23:1172–1181
Mashaghi S, Jadidi T, Koenderink G et al (2013) Lipid nanotechnology. Int J Mol Sci 14:4242–4282
Mitra RN, Merwin MJ, Han Z et al (2014) Yttrium oxide nanoparticles prevent photoreceptor death in a light-damage model of retinal degeneration. Free Radic Biol Med 75:140–148
Mitra RN, Nichols CA, Guo J et al (2016) Nanoparticle-mediated miR200-b delivery for the treatment of diabetic retinopathy. J Control Release 236:31–37
Rajala A, Wang Y, Zhu Y et al (2014) Nanoparticle-assisted targeted delivery of eye-specific genes to eyes significantly improves the vision of blind mice in vivo. Nano Lett 14:5257–5263
Silva AC, Amaral MH, Lobo JM et al (2015) Lipid nanoparticles for the delivery of biopharmaceuticals. Curr Pharm Biotechnol 16:291–302
Takahashi Y, Chen Q, Rajala RV et al (2015) Micro RNA-184 modulates cannocial Wnt signaling through regulation of frizzled-7 expression in the retina with ischemia-induced neovascularization. FEBS Lett 589:1143–1149
Walkey C, Das S, Seal S et al (2015) Catalytic properties and biomedical applications of cerium oxide nanoparticles. Environ Sci Nano 2:33–53
Wang Y, Rajala A, Rajala RV (2015) Lipid nanoparticles for ocular gene delivery. J Funct Biomater 6:379–394
Wang Y, Rajala A, Cao B et al (2016) Cell-specific promoters enable lipid-based nanoparticles to deliver genes to specific cells of the retina in vivo. Theranostics 6:1514–1527
Wong LL, McGinnis JF (2014) Nanoceria as bona fide catalytic antioxidants in medicine: what we know and what we want to know…. Adv Exp Med Biol 801:821–828
Zhou X, Wong LL, Karakoti AS et al (2011) Nanoceria inhibit the development and promote the regression of pathologic retinal neovascularization in the Vldlr knockout mouse. PLoS One 6:e16733
Zhu N, Liggitt D, Liu Y et al (1993) Systemic gene expression after intravenous DNA delivery into adult mice. Science 261:209–211
Znoiko SL, Rohrer B, Lu K et al (2005) Downregulation of cone-specific gene expression and degeneration of cone photoreceptors in the Rpe65−/− mouse at early ages. Invest Ophthalmol Vis Sci 46:1473–1479
Acknowledgments
Dr. Yuhong Wang is a Foundation Fighting Blindness Travel Awardee. This study was supported by grants from the National Institutes of Health (EY00871 and NEI core grant EY021725) and an unrestricted grant from Research to Prevent Blindness, Inc., to the Department of Ophthalmology. The authors thank Mr. Christopher Kooker for reading the manuscript.
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Wang, Y., Rajala, A., Rajala, R.V.S. (2018). Nanoparticles as Delivery Vehicles for the Treatment of Retinal Degenerative Diseases. In: Ash, J., Anderson, R., LaVail, M., Bowes Rickman, C., Hollyfield, J., Grimm, C. (eds) Retinal Degenerative Diseases. Advances in Experimental Medicine and Biology, vol 1074. Springer, Cham. https://doi.org/10.1007/978-3-319-75402-4_15
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DOI: https://doi.org/10.1007/978-3-319-75402-4_15
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