Self-assembling modified β-cyclodextrin nanoparticles as neuronal sirna delivery vectors: focus on huntington’s disease.
Abstract
Huntington's disease (HD) is a rare autosomal dominant neurodegenerative disease caused by the expression of a toxic Huntingtin (HTT) protein. The use of short interfering RNAs (siRNAs) to silence the mutant protein is one of the most promising therapeutic strategies under investigation. The biggest caveat to siRNA-based approaches is the lack of efficient and nontoxic delivery vectors for siRNA delivery to the central nervous system. In this study, we investigated the potential of modified amphiphilic β-cyclodextrins (CDs), oligosaccharide-based molecules, as novel siRNA neuronal carriers. We show that CDs formed nanosize particles which were stable in artificial cerebrospinal fluid. Moreover, these complexes were able to reduce the expression of the HTT gene in rat striatal cells (ST14A-HTT120Q) and in human HD primary fibroblasts. Only limited toxicity was observed with CD·siRNA nanoparticles in any of the in vitro models used. Sustained knockdown effects were observed in the striatum of the R6/2 mouse model of HD after single direct injections of CD·siRNA nanoparticles. Repeated brain injections of CD·siRNA complexes resulted in selective alleviation of motor deficits in this mouse model. Together these data support the utility of modified β-CDs as efficient and safe siRNA delivery vectors for RNAi-based therapies for neuropsychiatric and neurodegenerative disorders.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
siRNA encapsulated cyclodextrin nanoparticles reduce expression of huntingtin mRNA both in vivo and in vitro.
“short-interfering RNA (siRNA) encapsulated cyclodextrin nanoparticles were found capable to reduce expression of HTT mRNA both in vivo as well as in vitro”
From Nanotechnology: A Promising Approach for Delivery of Neuroprotective Drugs (2020) · cites this paper as Godinho et al., 2013
Cites
The effect of particle design on cellular internalization pathways
Gratton, S. E. A., Ropp, P. A., Pohlhaus, P. D., Luft, J. C., Madden, V. J., Napier, M. E., & DeSimone, J. M. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0801763105 · Cited by 2,918 · see citing works
Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles
Davis, M. E., Zuckerman, J. E., Choi, C. H. J., Seligson, D. B., Tolcher, A. W., Alabi, C. A., Yen, Y., Heidel, J. D., & Ribas, A. (2010) · Nature · doi:10.1038/nature08956 · Cited by 2,508 · see citing works
Toxicity of cationic lipids and cationic polymers in gene delivery
Lv, H., Zhang, S., Wang, B., Cui, S., & Yan, J. (2006) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.04.014 · Cited by 2,286 · see citing works
Nanocarriers’ entry into the cell: relevance to drug delivery
Hillaireau, H., & Couvreur, P. (2009) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-009-0053-z · Cited by 1,549 · see citing works
Davis, M. E. (2009) · Molecular Pharmaceutics · doi:10.1021/mp900015y · Cited by 979 · see citing works
Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease
Zuccato, C., Valenza, M., & Cattaneo, E. (2010) · Physiological Reviews · doi:10.1152/physrev.00041.2009 · Cited by 848 · see citing works
Harper, S. Q., Staber, P. D., He, X., Eliason, S. L., Martins, I. H., Mao, Q., Yang, L., Kotin, R. M., Paulson, H. L., & Davidson, B. L. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0501507102 · Cited by 682 · see citing works
Chemically modified siRNA: tools and applications
Watts, J. K., Deleavey, G. F., & Damha, M. J. (2008) · Drug Discovery Today · doi:10.1016/j.drudis.2008.05.007 · Cited by 431 · see citing works
DiFiglia, M., Sena‐Esteves, M., Chase, K. O., Sapp, E., Pfister, E., Sass, M., Yoder, J., Reeves, P., Pandey, R. K., Rajeev, K. G., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0708285104 · Cited by 417 · see citing works
Heidel, J. D., Yu, Z., Liu, J. Y.-C., Rele, S. M., Liang, Y., Zeidan, R. K., Kornbrust, D. J., & Davis, M. E. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0701458104 · Cited by 382 · see citing works
Engineering RNA for Targeted siRNA Delivery and Medical Application
Guo, P., Coban, O., Snead, N. M., Trebley, J., Hoeprich, S., Guo, S., & Shu, Y. (2010) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2010.03.008 · Cited by 301 · see citing works
Stability of Self-Assembled Polymeric Micelles in Serum
Lu, J., Owen, S. C., & Shoichet, M. S. (2011) · Macromolecules · doi:10.1021/ma200675w · Cited by 299 · see citing works
Rodríguez-Lebrón, E., Denovan‐Wright, E. M., Nash, K. R., Lewin, A. S., & Mandel, R. J. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.05.006 · Cited by 277 · see citing works
Nonviral methods for siRNA delivery
Gao, K., & Huang, L. (2009) · Molecular Pharmaceutics · doi:10.1021/mp800134q · Cited by 244 · see citing works
Tan, P.-H., Yang, L.-C., Hc, S., Lan, K.-C., & Cheng, J.-T. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302376 · Cited by 222 · see citing works
A Novel Mechanism Is Involved in Cationic Lipid-Mediated Functional siRNA Delivery
Lu, J. J.-Q., Langer, R., & Chen, J. (2009) · Molecular Pharmaceutics · doi:10.1021/mp900023v · Cited by 219 · see citing works
Davidson, T. J., Harel, S., Arboleda, V. A., Prunell, G., Shelanski, M. L., Greene, L. A., & Troy, C. M. (2004) · Journal of Neuroscience · doi:10.1523/jneurosci.3643-04.2004 · Cited by 215 · see citing works
Li, J. Y., Popović, N., & Brundin, P. (2005) · NeuroRx · doi:10.1602/neurorx.2.3.447 · Cited by 211 · see citing works
Thakker, D. R., Natt, F., Hüsken, D., Maier, R., Müller, M., van der Putten, H., Hoyer, D., & Cryan, J. F. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0406214101 · Cited by 211 · see citing works
Self-citationClinico-pathological rescue of a model mouse of Huntington's disease by siRNA
Wang, Y., Liu, W., Wada, E., Murata, M., Wada, K., & Kanazawa, I. (2005) · Neuroscience Research · doi:10.1016/j.neures.2005.06.021 · Cited by 192 · see citing works
RNAi functions in cultured mammalian neurons
Krichevsky, A. M., & Kosik, K. S. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182272699 · Cited by 175 · see citing works
siRNA-mediated knockdown of the serotonin transporter in the adult mouse brain
Thakker, D. R., Natt, F., Hüsken, D., van der Putten, H., Maier, R., Hoyer, D., & Cryan, J. F. (2005) · Molecular Psychiatry · doi:10.1038/sj.mp.4001687 · Cited by 159 · see citing works
Self-citationSix-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum
Grondin, R., Kaytor, M. D., Ai, Y., Nelson, P. T., Thakker, D. R., Heisel, J., Weatherspoon, M. R., Blum, J. L., Burright, E. N., Zhang, Z., et al. (2012) · Brain · doi:10.1093/brain/awr333 · Cited by 153 · see citing works
Efficient delivery of siRNA to cortical neurons using layered double hydroxide nanoparticles
Wong, Y., Markham, K., Xu, Z. P., Chen, M., Lu, G. Q., Bartlett, P. F., & Cooper, H. M. (2010) · Biomaterials · doi:10.1016/j.biomaterials.2010.07.077 · Cited by 152 · see citing works
Functional Delivery of siRNA in Mice Using Dendriworms
Agrawal, A., Min, D., Singh, N., Zhu, H., Birjiniuk, A., von Maltzahn, G., Harris, T. J., Xing, D., Woolfenden, S. D., Sharp, P. A., et al. (2009) · ACS Nano · doi:10.1021/nn900201e · Cited by 147 · see citing works
Kim, I., Lim, C., Kim, J., Nam, H. Y., Nam, K., Kim, S.-W., Park, J., & Lee, J.-K. (2009) · Journal of Controlled Release · doi:10.1016/j.jconrel.2009.11.011 · Cited by 131 · see citing works
Anisamide-targeted cyclodextrin nanoparticles for siRNA delivery to prostate tumours in mice
Guo, J., Ogier, J. R., Desgranges, S., Darcy, R., & O’Driscoll, C. M. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.07.012 · Cited by 127 · see citing works
Self-citationLombardi, M. S., Jaspers, L., Spronkmans, C., Gellera, C., Taroni, F., Maria, E. D., Donato, S. D., & Kaemmerer, W. F. (2009) · Experimental Neurology · doi:10.1016/j.expneurol.2009.03.004 · Cited by 124 · see citing works
Cyclodextrin-based siRNA delivery nanocarriers: a state-of-the-art review
Chaturvedi, K., Ganguly, K., Kulkarni, A. R., Kulkarni, V. H., Nadagouda, M. N., Rudzinski, W. E., & Aminabhavi, T. M. (2011) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2011.610790 · Cited by 110 · see citing works
Tönges, L., Lingor, P., Egle, R., Dietz, G. P. H., Fahr, A., & Bähr, M. (2006) · RNA · doi:10.1261/rna.2252206 · Cited by 103 · see citing works
Therapeutic targeting in the silent era: advances in non-viral siRNA delivery
Guo, J., Fisher, K. A., Darcy, R., Cryan, J. F., & O’Driscoll, C. M. (2010) · Molecular BioSystems · doi:10.1039/c001050m · Cited by 97 · see citing works
Self-citationTherapeutic potential of RNA interference for neurological disorders
Sah, D. W. Y. (2006) · Life Sciences · doi:10.1016/j.lfs.2006.06.011 · Cited by 97 · see citing works
Widespread suppression of huntingtin with convection-enhanced delivery of siRNA
Stiles, D. K., Zhang, Z., Ge, P., Nelson, B. D., Grondin, R., Ai, Y., Hardy, P., Nelson, P. T., Guzaev, A. P., Butt, M. T., et al. (2011) · Experimental Neurology · doi:10.1016/j.expneurol.2011.11.020 · Cited by 91 · see citing works
Tf-lipoplexes for neuronal siRNA delivery: A promising system to mediate gene silencing in the CNS
Cardoso, A. L., Simões, S., de Almeida, L. P., Plesnila, N., de Lima, M. C. P., Wagner, E., & Culmsee, C. (2008) · Journal of Controlled Release · doi:10.1016/j.jconrel.2008.08.014 · Cited by 86 · see citing works
Salahpour, A., Medvedev, I. O., Beaulieu, J., Gainetdinov, R. R., & Caron, M. G. (2006) · Biological Psychiatry · doi:10.1016/j.biopsych.2006.03.020 · Cited by 80 · see citing works
CAG repeat lengths ≥ 335 attenuate the phenotype in the R6/2 Huntington's disease transgenic mouse
Dragatsis, I., Goldowitz, D., Mar, N. D., Deng, Y. P., Meade, C. A., Liu, L., Sun, Z., Dietrich, P., Yue, J., & Reiner, A. (2008) · Neurobiology of Disease · doi:10.1016/j.nbd.2008.10.009 · Cited by 78 · see citing works
Click-Modified Cyclodextrins as Nonviral Vectors for Neuronal siRNA Delivery
O’Mahony, A., Godinho, B. M. D. C., Ogier, J. R., Devocelle, M. C., Darcy, R., Cryan, J. F., & O’Driscoll, C. M. (2012) · ACS Chemical Neuroscience · doi:10.1021/cn3000372 · Cited by 75 · see citing works
Self-citationNeill, M. J. O., Guo, J., Byrne, C. J., Darcy, R., & O’Driscoll, C. M. (2011) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2011.04.021 · Cited by 71 · see citing works
Self-citationO’Mahony, A., Ogier, J. R., Desgranges, S., Cryan, J. F., Darcy, R., & O’Driscoll, C. M. (2012) · Organic & Biomolecular Chemistry · doi:10.1039/c2ob25490e · Cited by 70 · see citing works
Self-citationMenalled, L., Patry, M., Ragland, N., Lowden, P. A. S., Goodman, J., Minnich, J., Zahasky, B., Park, L., Leeds, J. M., Howland, D., et al. (2010) · PLoS ONE · doi:10.1371/journal.pone.0009793 · Cited by 70 · see citing works
Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats
Fiszer, A., Mykowska, A., & Krzyżosiak, W. J. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr156 · Cited by 69 · see citing works
Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery
Chen, Q., Butler, D., Querbes, W., Pandey, R. K., Ge, P., Maier, M. A., Zhang, L., Rajeev, K. G., Nechev, L. V., Koteliansky, V., et al. (2010) · Journal of Controlled Release · doi:10.1016/j.jconrel.2010.02.011 · Cited by 67 · see citing works
Wang, H., Ghosh, A., Baigude, H., Yang, C., Qiu, L., Xia, X., Zhou, H., Rana, T. M., & Xu, Z. (2008) · Journal of Biological Chemistry · doi:10.1074/jbc.m800834200 · Cited by 66 · see citing works
Nakajima, H., Kubo, T., Semi, Y., Itakura, M., Kuwamura, M., Izawa, T., Azuma, Y., & Takeuchi, T. (2011) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2011.10.003 · Cited by 63 · see citing works
Toxicity in mice expressing short hairpin RNAs gives new insight into RNAi
Snøve, O., & Rossi, J. J. (2006) · Genome biology · doi:10.1186/gb-2006-7-8-231 · Cited by 54 · see citing works
Gold Nanorod–siRNA Induces Efficient In Vivo Gene Silencing in The Rat Hippocampus
Bonoiu, A. C., Bergey, E. J., Ding, H. Y., Hu, R., Kumar, R., Yong, K., Prasad, P. N., Mahajan, S. D., Picchione, K. E., Bhattacharjee, A., et al. (2011) · Nanomedicine · doi:10.2217/nnm.11.20 · Cited by 54 · see citing works
RNAi therapeutics for CNS disorders
Boudreau, R. L., & Davidson, B. L. (2010) · Brain Research · doi:10.1016/j.brainres.2010.03.038 · Cited by 52 · see citing works
Targeted gene delivery to hepatocytes with galactosylated amphiphilic cyclodextrins
Mcmahon, A., O’Neill, M. J., Gómez‐Orte, E., Donohue, R., Forde, D., Darcy, R., & O’Driscoll, C. M. (2012) · Journal of Pharmacy and Pharmacology · doi:10.1111/j.2042-7158.2012.01497.x · Cited by 44 · see citing works
Self-citationsiRNA Delivery to CNS Cells using a Membrane Translocation Peptide
Ifediba, M. A., Medarova, Z. O., Ng, S., Yang, J., & Moore, A. V. (2010) · Bioconjugate Chemistry · doi:10.1021/bc900488e · Cited by 36 · see citing works
Gene Therapy: A Primer for Neurosurgeons
Chiocca, E. (2003) · Neurosurgery · doi:10.1227/01.neu.0000073532.05714.2b · Cited by 21 · see citing works
A Note on Regulatory Concerns and Toxicity Assessment in Lipid-Based Delivery Systems (LDS)
Souto, E. B., Martins‐Lopes, P., Lopes, C. M., Gaivão, I., Silva, A. M., & Guedes‐Pinto, H. (2009) · Journal of Biomedical Nanotechnology · doi:10.1166/jbn.2009.1041 · Cited by 19 · see citing works
Erratum: Progress and prospects: immune responses to viral vectors
Nayak, S., & HERZOG, R. W. (2010) · Gene Therapy · doi:10.1038/gt.2009.168 · Cited by 2 · see citing works
Flagged first, then by citations
Cited by
Sorted by citations
Nanotechnology: A Promising Approach for Delivery of Neuroprotective Drugs
Naqvi, S., Panghal, A., & Flora, S. J. S. (2020) · Frontiers in Neuroscience · doi:10.3389/fnins.2020.00494 · Cited by 305 · see citing works
Cyclodextrin complexes: Perspective from drug delivery and formulation
Jacob, S., & Nair, A. B. (2018) · Drug Development Research · doi:10.1002/ddr.21452 · Cited by 263 · see citing works
Emerging Applications of Nanotechnology in Healthcare Systems: Grand Challenges and Perspectives
Anjum, S., Ishaque, S., Fatima, H., Farooq, W., Hano, C., Abbasi, B. H., & Anjum, I. (2021) · Pharmaceuticals · doi:10.3390/ph14080707 · Cited by 258 · see citing works
Treatment of neurodegenerative disorders through the blood–brain barrier using nanocarriers
Poovaiah, N., Davoudi, Z., Peng, H., Schlichtmann, B. W., Mallapragada, S. K., Narasimhan, B., & Wang, Q. (2018) · Nanoscale · doi:10.1039/c8nr04073g · Cited by 227 · see citing works
Sun, D., Zou, Y., Song, L., Han, S., Yang, H., Chu, D., Dai, Y., Ma, J., O’Driscoll, C. M., Yu, Z., et al. (2021) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2021.06.005 · Cited by 197 · see citing works
Self-citationCano, A., Sánchez-López, E., Ettcheto, M., López-Machado, A., Espina, M., Souto, E. B., Galindo, R., Camins, A., García, M. L., & Turowski, P. (2020) · Nanomedicine · doi:10.2217/nnm-2019-0443 · Cited by 144 · see citing works
Carthy, D. J. M., Malhotra, M., O’Mahony, A., Cryan, J. F., & O’Driscoll, C. M. (2014) · Pharmaceutical Research · doi:10.1007/s11095-014-1545-6 · Cited by 132 · see citing works
Self-citationA Vision for Cyclodextrin Nanoparticles in Drug Delivery Systems and Pharmaceutical Applications
Lakkakula, J., & Krause, R. W. M. (2014) · Nanomedicine · doi:10.2217/nnm.14.41 · Cited by 130 · see citing works
Neurotheranostics as Personalized Medicines
Kevadiya, B. D., Ottemann, B. M., Thomas, M. B., Mukadam, I., Nigam, S., McMillan, J., Gorantla, S., Bronich, T. K., Edagwa, B., & Gendelman, H. E. (2019) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2018.10.011 · Cited by 124 · see citing works
Coisne, C., Tilloy, S., Monflier, É., Wils, D., Fénart, L., & Gosselet, F. (2016) · Molecules · doi:10.3390/molecules21121748 · Cited by 120 · see citing works
Cyclodextrin-based nanostructures
Narayanan, G., Shen, J., Matai, I., Sachdev, A., Boy, R., & Tonelli, A. E. (2021) · Progress in Materials Science · doi:10.1016/j.pmatsci.2021.100869 · Cited by 119 · see citing works
Fitzgerald, K. A., Guo, J., Tierney, E. G., Curtin, C. M., Malhotra, M., Darcy, R., O’Brien, F. J., & O’Driscoll, C. M. (2015) · Biomaterials · doi:10.1016/j.biomaterials.2015.07.019 · Cited by 112 · see citing works
Self-citationCyclodextrins in drug delivery: applications in gene and combination therapy
Haley, R. M., Gottardi, R., Langer, R., & Mitchell, M. J. (2020) · Drug Delivery and Translational Research · doi:10.1007/s13346-020-00724-5 · Cited by 109 · see citing works
Cyclodextrins, Blood–Brain Barrier, and Treatment of Neurological Diseases
Vecsernyés, M., Fenyvesi, F., Bácskay, I., Deli, M. A., Szente, L., & Fenyvesi, É. (2014) · Archives of Medical Research · doi:10.1016/j.arcmed.2014.11.020 · Cited by 102 · see citing works
Cyclodextrin-based nanoparticles for pharmaceutical applications: a review
Pandey, A. (2021) · Environmental Chemistry Letters · doi:10.1007/s10311-021-01275-y · Cited by 94 · see citing works
Kapilov-Buchman, Y., Lellouche, E., Zigdon, S., Bechor, M., Michaeli, S., & Lellouche, J. (2013) · Bioconjugate Chemistry · doi:10.1021/bc4004316 · Cited by 92 · see citing works
Zerkoune, L., Angelova, A., & Lesieur, S. (2014) · Nanomaterials · doi:10.3390/nano4030741 · Cited by 91 · see citing works
Fitzgerald, K. A., Malhotra, M., Gooding, M., Sallas, F., Evans, J. C., Darcy, R., & O’Driscoll, C. M. (2015) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2015.12.055 · Cited by 81 · see citing works
Self-citationMcCarthy, J., O'Neill, M. J., Bourré, L., Walsh, D. P., Quinlan, A., Hurley, G., Ogier, J. R., Shanahan, F., Melgar, S., Darcy, R., et al. (2013) · Journal of Controlled Release · doi:10.1016/j.jconrel.2013.03.004 · Cited by 74 · see citing works
Self-citationMendonça, M. C. P., Kont, A., Aburto, M. R., Cryan, J. F., & O’Driscoll, C. M. (2021) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.0c01238 · Cited by 73 · see citing works
Self-citationWang, J., Dou, B., & Bao, Y. (2013) · Materials Science and Engineering C · doi:10.1016/j.msec.2013.08.035 · Cited by 72 · see citing works
Guo, J., Russell, E. G., Darcy, R., Cotter, T. G., McKenna, S. L., Cahill, M. R., & O’Driscoll, C. M. (2017) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.6b01150 · Cited by 70 · see citing works
Self-citationNanogels as Novel Nanocarrier Systems for Efficient Delivery of CNS Therapeutics
Zhang, Y., Zou, Z., Liu, S., Miao, S., & Liu, H. (2022) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2022.954470 · Cited by 69 · see citing works
Gooding, M., Malhotra, M., McCarthy, D. J., Godinho, B. M. D. C., Cryan, J. F., Darcy, R., & O’Driscoll, C. M. (2015) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2015.02.007 · Cited by 68 · see citing works
Self-citationRiccardi, C., Napolitano, F., Montesarchio, D., Sampaolo, S., & Melone, M. A. B. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13111897 · Cited by 66 · see citing works
Multifunctional Nanotheranostics for Overcoming the Blood–Brain Barrier
Beygi, M. H. M., Oroojalian, F., Azizi‐Arani, S., Hosseini, S. S., Mokhtarzadeh, A., Kesharwani, P., & Sahebkar, A. (2024) · Advanced Functional Materials · doi:10.1002/adfm.202310881 · Cited by 65 · see citing works
Nanomaterial applications for neurological diseases and central nervous system injury
Huang, L., Hu, J., Huang, S., Wang, B. Z., Siaw-Debrah, F., Nyanzu, M. K., Zhang, Y., & Zhuge, Q. (2017) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2017.07.003 · Cited by 65 · see citing works
Molecular nanoparticle-based gene delivery systems
Blanco, J. L. J., Benito, J. M., Mellet, C. O., & Fernández, J. M. G. (2017) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2017.03.012 · Cited by 63 · see citing works
Gallon, E., Matini, T., Sasso, L., Mantovani, G., Armiñán, A., Sanchis, J., Caliceti, P., Alexander, C., Vicent, M. J., & Salmaso, S. (2015) · Biomacromolecules · doi:10.1021/acs.biomac.5b00286 · Cited by 62 · see citing works
Self-Assembled Cationic β-Cyclodextrin Nanostructures for siRNA Delivery
Singh, R. P., Hidalgo, T., Cazade, P., Darcy, R., Cronin, M. F., Dorin, I., O’Driscoll, C. M., & Thompson, D. (2019) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.8b01307 · Cited by 61 · see citing works
Self-citationZou, Y., Xiao, F., Song, L., Sun, B., Sun, D., Chu, D., Wang, L., Han, S., Yu, Z., O’Driscoll, C. M., et al. (2021) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2021.120888 · Cited by 59 · see citing works
Self-citationsiRNA as a tool to improve the treatment of brain diseases: Mechanism, targets and delivery
Gomes, M. J., Martins, S., & Sarmento, B. F. C. C. (2015) · Ageing Research Reviews · doi:10.1016/j.arr.2015.03.001 · Cited by 59 · see citing works
James, E. N., Delany, A. M., & Nair, L. S. (2014) · Acta Biomaterialia · doi:10.1016/j.actbio.2014.04.026 · Cited by 59 · see citing works
Surfactants influence polymer nanoparticle fate within the brain
Joseph, A., Simo, G. M., Gao, T. A., Alhindi, N., Xu, N., Graham, D. J., Gamble, L. J., & Nance, E. A. (2021) · Biomaterials · doi:10.1016/j.biomaterials.2021.121086 · Cited by 58 · see citing works
Recent advances in cyclodextrin delivery techniques
Arima, H., Hayashi, Y., Higashi, T., & Motoyama, K. (2015) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2015.1026893 · Cited by 57 · see citing works
Evans, J. C., Malhotra, M., Guo, J., O’Shea, J. P., Hanrahan, K., O’Neill, A., Landry, W. D., Griffin, B. T., Darcy, R., Watson, R. W. G., et al. (2016) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2016.06.014 · Cited by 56 · see citing works
Self-citationGene delivery based on macrocyclic amphiphiles
Geng, W., Huang, Q., Xu, Z., Wang, R., & Guo, D. (2019) · Theranostics · doi:10.7150/thno.31914 · Cited by 56 · see citing works
O’Mahony, A., Godinho, B. M. D. C., Cryan, J. F., & O’Driscoll, C. M. (2013) · Journal of Pharmaceutical Sciences · doi:10.1002/jps.23672 · Cited by 55 · see citing works
Self-citationGao, L., Wang, J., & Bi, Y. (2025) · International Journal of Nanomedicine · doi:10.2147/ijn.s549893 · Cited by 52 · see citing works
Godinho, B. M. D. C., Ogier, J. R., Quinlan, A., Darcy, R., Griffin, B. T., Cryan, J. F., & O’Driscoll, C. M. (2014) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2014.06.054 · Cited by 52 · see citing works
Self-citationMazibuko, Z., Choonara, Y. E., Kumar, P., du Toit, L. C., Modi, G., Naidoo, D., & Pillay, V. (2015) · Journal of Pharmaceutical Sciences · doi:10.1002/jps.24322 · Cited by 51 · see citing works
Role of Nanomedicine-Based Therapeutics in the Treatment of CNS Disorders
Guo, Z.-H., Khattak, S., Rauf, M. A., Ansari, M. A., Alomary, M. N., Razak, S., Yang, C., Wu, D., & Ji, X. (2023) · Molecules · doi:10.3390/molecules28031283 · Cited by 49 · see citing works
Polyglutamine (PolyQ) Diseases: Navigating the Landscape of Neurodegeneration
Tenchov, R., Sasso, J. M., & Zhou, Q. A. (2024) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.4c00184 · Cited by 49 · see citing works
Delivering a disease-modifying treatment for Huntington's disease
Godinho, B. M. D. C., Malhotra, M., O’Driscoll, C. M., & Cryan, J. F. (2014) · Drug Discovery Today · doi:10.1016/j.drudis.2014.09.011 · Cited by 47 · see citing works
Self-citationNanotechnology in Drug Discovery and Development
Gutiérrez, L., Stepien, G., Gutiérrez, L., Pérez-Hernández, M., Pardo, J., Pardo, J., Grazú, V., & de la Fuente, J. M. (2017) · Elsevier eBooks · doi:10.1016/b978-0-12-409547-2.12292-9 · Cited by 46 · see citing works
Mendonça, M. C. P., Cronin, M. F., Cryan, J. F., & O’Driscoll, C. M. (2021) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2021.11.003 · Cited by 46 · see citing works
Self-citationSun, Y., Cronin, M. F., Mendonça, M. C. P., Guo, J., & O’Driscoll, C. M. (2023) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2023.106427 · Cited by 46 · see citing works
Self-citationProspects for neuroprotective therapies in prodromal Huntington's disease
Chandra, A., Johri, A., & Beal, M. F. (2014) · Movement Disorders · doi:10.1002/mds.25835 · Cited by 41 · see citing works
Short interfering RNA therapeutics: nanocarriers, prospects and limitations
Mehrotra, N., & Tripathi, R. M. (2015) · IET Nanobiotechnology · doi:10.1049/iet-nbt.2015.0018 · Cited by 41 · see citing works
Godinho, B. M. D. C., McCarthy, D. J., Torres‐Fuentes, C., Beltrán, C. J., McCarthy, J., Quinlan, A., Ogier, J. R., Darcy, R., O’Driscoll, C. M., & Cryan, J. F. (2013) · Biomaterials · doi:10.1016/j.biomaterials.2013.09.068 · Cited by 39 · see citing works
Self-citationMedical and Nonmedical Applications of Synthetic Transmembrane Anion Transporters
Rathnaweera, U. M. C., Chowdhury, S. M., Salam, R., & Busschaert, N. (2025) · Chemical Reviews · doi:10.1021/acs.chemrev.5c00129 · Cited by 38 · see citing works
Oligonucleotide-based therapy for neurodegenerative diseases
Magen, I., & Hornstein, E. (2014) · Brain Research · doi:10.1016/j.brainres.2014.04.005 · Cited by 38 · see citing works
Delivering RNAi therapeutics with non-viral technology: a promising strategy for prostate cancer?
Guo, J., Evans, J. C., & O’Driscoll, C. M. (2013) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2013.02.002 · Cited by 38 · see citing works
Self-citationCyclodextrin-siRNA conjugates as versatile gene silencing agents
Malhotra, M., Gooding, M., Evans, J. C., O’Driscoll, D., Darcy, R., & O’Driscoll, C. M. (2017) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2017.11.024 · Cited by 38 · see citing works
Self-citationsiRNA delivery for treatment of degenerative diseases, new hopes and challenges
Zahir‐Jouzdani, F., Mottaghitalab, F., Dinarvand, M., & Atyabi, F. (2018) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2018.04.001 · Cited by 37 · see citing works
pH-Responsive Polymersome Microparticles as Smart Cyclodextrin-Releasing Agents
Puglisi, A., Bayır, E., Tımur, S., & Yağcı, Y. (2019) · Biomacromolecules · doi:10.1021/acs.biomac.9b01083 · Cited by 36 · see citing works
Cyclodextrin-Based Functional Glyconanomaterials
Rivero-Barbarroja, G., Benito, J. M., Mellet, C. O., & Fernández, J. M. G. (2020) · Nanomaterials · doi:10.3390/nano10122517 · Cited by 35 · see citing works
Evans, J. C., Malhotra, M., Sweeney, K. G., Darcy, R., Nelson, C. C., Hollier, B. G., & O’Driscoll, C. M. (2017) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2017.09.013 · Cited by 35 · see citing works
Self-citationO’Mahony, A., Ogier, J. R., Darcy, R., Cryan, J. F., & O’Driscoll, C. M. (2013) · PLoS ONE · doi:10.1371/journal.pone.0066413 · Cited by 34 · see citing works
Self-citationLi, Y., Liu, L., Ji, W., Peng, H., Zhao, R., & Zhang, X. (2020) · Nano Today · doi:10.1016/j.nantod.2020.101006 · Cited by 33 · see citing works
Non-Viral Nucleic Acid Delivery Strategies to the Central Nervous System
Tan, J. Y., Sellers, D. L., Pham, B., Pun, S. H., & Horner, P. J. (2016) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2016.00108 · Cited by 33 · see citing works
Sun, Y., Cronin, M. F., Mendonça, M. C. P., Guo, J., & O’Driscoll, C. M. (2023) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.3c00769 · Cited by 32 · see citing works
Self-citationExploiting mesoporous silica, silver and gold nanoparticles for neurodegenerative diseases treatment
Ribeiro, T. D. C., Sábio, R. M., Carvalho, G. C., Fonseca‐Santos, B., & Chorilli, M. (2022) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2022.121978 · Cited by 32 · see citing works
Evans, J. C., McCarthy, J., Torres‐Fuentes, C., Cryan, J. F., Ogier, J. R., Darcy, R., Watson, R. W. G., & O’Driscoll, C. M. (2015) · Gene Therapy · doi:10.1038/gt.2015.50 · Cited by 28 · see citing works
Self-citationSevim, S., & Şanlıer, N. (2024) · Journal of Food Science · doi:10.1111/1750-3841.17527 · Cited by 27 · see citing works
“Click” chemistry mediated construction of cationic curdlan nanocarriers for efficient gene delivery
Han, J., Wang, X., Liu, L., Li, D., Suyaola, S., Wang, T., & Baigude, H. (2017) · Carbohydrate Polymers · doi:10.1016/j.carbpol.2017.01.055 · Cited by 26 · see citing works
Arima, H., Motoyama, K., & Higashi, T. (2017) · Therapeutic Delivery · doi:10.4155/tde-2016-0064 · Cited by 23 · see citing works
Revisiting gene delivery to the brain: silencing and editing
Conniot, J., Talebian, S., Simões, S. M. N., Ferreira, L., & Conde, J. (2020) · Biomaterials Science · doi:10.1039/d0bm01278e · Cited by 23 · see citing works
Incontinence products and devices for the elderly.
Newman, D. K. (2004) · PubMed · Cited by 23 · see citing works
Nanobiotechnology in Neurodegenerative Diseases
Jampílek, J., Kráľová, K., Novák, P., & Novák, M. (2019) · Springer eBooks · doi:10.1007/978-3-030-30930-5_4 · Cited by 22 · see citing works
Jiang, Q., Yue, S., Yu, K., Tian, T., Zhang, J., Chu, H., Cui, Z., & Bi, S. (2021) · Journal of Nanobiotechnology · doi:10.1186/s12951-021-01040-x · Cited by 22 · see citing works
Theranostical application of nanomedicine for treating central nervous system disorders
Wang, R., & Xue, X. (2018) · Science China Life Sciences · doi:10.1007/s11427-017-9292-7 · Cited by 22 · see citing works
Cyclodextrins as Active Therapeutic Agents: Beyond Their Role as Excipients
Pîrvu, A. S., Văruţ, R.-M., Trașcă, D.-M., Stoica, G. A., Radivojević, K., Carmen, S., Arsenie, C. C., & Popescu, C. (2025) · Pharmaceuticals · doi:10.3390/ph18101592 · Cited by 21 · see citing works
Engineered Self-Assembly of Amphiphilic Cyclodextrin Conjugates for Drug Encapsulation
Brettner, F. E. B., Schreiner, J., Vogel‐Kindgen, S., & Windbergs, M. (2022) · ACS Biomaterials Science & Engineering · doi:10.1021/acsbiomaterials.2c01023 · Cited by 21 · see citing works
Ooi, Y. J., Huang, C., Lau, K., Chew, S. Y., Park, J. G., & Chan‐Park, M. B. E. (2024) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.3c10620 · Cited by 20 · see citing works
Application of Nanomaterials in Neurodegenerative Diseases
Cui, W., Fu, W., Lin, Y., & Zhang, T. (2020) · Current Stem Cell Research & Therapy · doi:10.2174/1574888x15666200326093410 · Cited by 20 · see citing works
Kont, A., Mendonça, M. C. P., Cronin, M. F., Cahill, M. R., & O’Driscoll, C. M. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23179791 · Cited by 19 · see citing works
Self-citationCyclodextrin-Containing Drug Delivery Systems and Their Applications in Neurodegenerative Disorders
Xing, Y., Meng, B., & Chen, Q. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms251910834 · Cited by 19 · see citing works
A biodegradable adamantane polymer with ketal linkages in its backbone for gene therapy
Maity, S., Choudhary, P., Manjunath, M., Kulkarni, A., & Murthy, N. (2015) · Chemical Communications · doi:10.1039/c5cc05242d · Cited by 18 · see citing works
Cyclodextrin-Based Nanoparticles for Delivery of Antisense Oligonucleotides Targeting Huntingtin
Mendonça, M. C. P., Sun, Y., Cronin, M. F., Lindsay, A. J., Cryan, J. F., & O’Driscoll, C. M. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15020520 · Cited by 18 · see citing works
Self-citationSingh, S., Hema, Sharma, N., Sachdeva, M., Behl, T., Zahoor, I., Fuloria, N. K., Sekar, M., Fuloria, S., Subramaniyan, V., et al. (2022) · Environmental Science and Pollution Research · doi:10.1007/s11356-022-22830-2 · Cited by 17 · see citing works
Escalona-Rayo, O., Fuentes-Vázquez, P., Leyva‐Gómez, G., Cisneros, B., Villalobos, R., Magaña, J. J., & Quintanar‐Guerrero, D. (2017) · Drug Development and Industrial Pharmacy · doi:10.1080/03639045.2017.1281949 · Cited by 17 · see citing works
Singh, D., Kapahi, H., Rashid, M., Prakash, A. K., Majeed, A. B. A., & Mishra, N. (2015) · Artificial Cells Nanomedicine and Biotechnology · doi:10.3109/21691401.2015.1029622 · Cited by 17 · see citing works
Ahmed, T. (2024) · European Journal of Neuroscience · doi:10.1111/ejn.16336 · Cited by 16 · see citing works
Gastrointestinal gene delivery by cyclodextrins – In vitro quantification of extracellular barriers
O’Neill, M. J., O’Mahony, A., Byrne, C. J., Darcy, R., & O’Driscoll, C. M. (2013) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2013.08.073 · Cited by 16 · see citing works
Self-citationGomes, M. J., Dreier, J., Brewer, J. R., Martins, S., Brandl, M., & Sarmento, B. F. C. C. (2016) · Journal of Membrane Science · doi:10.1016/j.memsci.2016.01.002 · Cited by 15 · see citing works
Khan, S., Bano, N., Ahamad, S., Dar, N. J., Nazir, A., & Bhat, S. A. (2024) · Coordination Chemistry Reviews · doi:10.1016/j.ccr.2024.216206 · Cited by 14 · see citing works
Recent Advancements in Nanomaterials: A Promising Way to Manage Neurodegenerative Disorders
Nguyen, T. T., Nguyen‐Thi, P., Nguyen, T. H. A., Ho, T.-T., An, T. N. M., Toi, V. V., & Giau, V. V. (2023) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-023-00654-1 · Cited by 14 · see citing works
O’Mahony, A., Cronin, M. F., Mcmahon, A., Evans, J. C., Daly, K., Darcy, R., & O’Driscoll, C. M. (2014) · Journal of Pharmaceutical Sciences · doi:10.1002/jps.23922 · Cited by 14 · see citing works
Self-citationBorbolla‐Jiménez, F. V., Prado-Audelo, M. L. D., Cisneros, B., Caballero-Florán, I. H., Leyva‐Gómez, G., & Magaña, J. J. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13071018 · Cited by 13 · see citing works
β-Cyclodextrin-based polyelectrolyte complexes for drug delivery
Zhao, R., Tang, B., Xu, Z., & Fang, G. (2025) · Coordination Chemistry Reviews · doi:10.1016/j.ccr.2025.216581 · Cited by 12 · see citing works
Preclinical Nanomedicines for Polyglutamine-Based Neurodegenerative Diseases
Lee, L. K. C., Leong, L. I., Liu, Y., Luo, M., Chan, H. Y. E., & Choi, C. H. J. (2020) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.0c00506 · Cited by 12 · see citing works
Innovation in nanomedicine: nanocarriers for central nervous system disorders management
Waqar, M. A., Mubarak, N., Khan, A. M., Ahmad, S., Zaib, M., Kanwal, U., Khan, I. N., Ayesha, M., Mughal, M., & Fahad, M. (2024) · International Journal of Polymeric Materials · doi:10.1080/00914037.2024.2399271 · Cited by 11 · see citing works
Tetraethylenepentamine-Coated β Cyclodextrin Nanoparticles for Dual DNA and siRNA Delivery
Liu, C., Shih, P.-Y., Lin, C.-H., Chen, Y.-J., Wu, W., & Wang, C. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14050921 · Cited by 11 · see citing works
Chakraborty, P., Bhattacharyya, C., Sahu, R., Dua, T. K., Kandimalla, R. J. L., & Dewanjee, S. (2023) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2023.105267 · Cited by 11 · see citing works
RNAi therapeutics for brain cancer: current advancements in RNAi delivery strategies
Malhotra, M., Toulouse, A., Godinho, B. M. D. C., Carthy, D. J. M., Cryan, J. F., & O’Driscoll, C. M. (2015) · Molecular BioSystems · doi:10.1039/c5mb00278h · Cited by 8 · see citing works
Self-citationHsia, T., & Chen, Y. (2024) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2024.114234 · Cited by 7 · see citing works
Small RNA drugs for prion disease: a new frontier
Boese, A. S., Majer, A., Saba, R., & Booth, S. A. (2013) · Expert Opinion on Drug Discovery · doi:10.1517/17460441.2013.818976 · Cited by 7 · see citing works
Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives
Khawaja, S., Ali, R. H., Ahmed, I., & Umair, M. (2025) · Current Genomics · doi:10.2174/0113892029361490250310041259 · Cited by 6 · see citing works
Holghoomi, R., Rahdar, A., Kadkhoda, J., Mohammadzadeh, V., Shirzad, M., Salehi, B., Ebrahimi, M.-S., & Pandey, S. (2026) · Nano LIFE · doi:10.1142/s179398442630013x · Cited by 6 · see citing works
- Authors
- Godinho, B. M. D. C., Ogier, J. R., Darcy, R., O’Driscoll, C. M., & Cryan, J. F.
- Affiliations
- University College Cork (IE)
- University College Dublin (IE)
- Pharmacodelivery Group, School of Pharmacy, University College Cork, Cork, Ireland
- Corresponding author
- Cryan, John F.
- Venue
- Molecular Pharmaceutics
- Publisher
- American Chemical Society (ACS)
- Published
- 2013-01-09
- Volume
- 10
- Issue
- 2
- Pages
- 640-649
- ISSN
- 1543-8384 (ISSN-L)
- 1543-8392
- OpenAlex
- W2064441112
- Cited by
- 126
- References
- 52
- Citations by year
- 2026: 4 · 2025: 11 · 2024: 12 · 2023: 8 · 2022: 7 · 2021: 14 · 2020: 10 · 2019: 5 · 2018: 6 · 2017: 10 · 2016: 5 · 2015: 12 · 2014: 11 · 2013: 10
- FWCI
- 4.24
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Molecular Pharmaceutics (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Physical Sciences › Materials Science › Polymers and Plastics › Dendrimers and Hyperbranched Polymers
- Keywords
- Huntington's disease, siRNA delivery, modified β-cyclodextrin, RNAi therapy, brain drug delivery, striatal cells, R6/2 mouse model, motor impairment, nanoparticle toxicity
- MeSH terms
- Animals, Cells, Cultured, Genetic Vectors, Humans, Huntington Disease, Genetic Therapy, Neurodegenerative Diseases, RNA, Small Interfering, beta-Cyclodextrins, Mice, Rats, Nanoparticles
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review