Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery
Open accessAbstract
Neurological diseases and disorders (NDDs) present a significant societal burden and currently available drug- and biological-based therapeutic strategies have proven inadequate to alleviate it. Gene therapy is a suitable alternative to treat NDDs compared to conventional systems since it can be tailored to specifically alter select gene expression, reverse disease phenotype and restore normal function. The scope of gene therapy has broadened over the years with the advent of RNA interference and genome editing technologies. Consequently, encouraging results from central nervous system (CNS)-targeted gene delivery studies have led to their transition from preclinical to clinical trials. As we shift to an exciting gene therapy era, a retrospective of available literature on CNS-associated gene delivery is in order. This review is timely in this regard, since it analyzes key challenges and major findings from the last two decades and evaluates future prospects of brain gene delivery. We emphasize major areas consisting of physiological and pharmacological challenges in gene therapy, function-based selection of a ideal cellular target(s), available therapy modalities, and diversity of viral vectors and nanoparticles as vehicle systems. Further, we present plausible answers to key questions such as strategies to circumvent low blood-brain barrier permeability and most suitable CNS cell types for targeting. We compare and contrast pros and cons of the tested viral vectors in the context of delivery systems used in past and current clinical trials. Gene vector design challenges are also evaluated in the context of cell-specific promoters. Key challenges and findings reported for recent gene therapy clinical trials, assessing viral vectors and nanoparticles are discussed from the perspective of bench to bedside gene therapy translation. We conclude this review by tying together gene delivery challenges, available vehicle systems and comprehensive analyses of neuropathogenesis to outline future prospects of CNS-targeted gene therapies.
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AAV vector administration to the CNS has an excellent safety and efficacy profile.
“AAV vector administration to the CNS has an excellent safety and efficacy profile.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [53]
Section 5.1 describes AAV vectors as suitable for brain gene delivery because of their low immunogenicity and reports effective transduction. Section 7 reports that an AAV2 trial was safe and well tolerated, but says its efficacy was still being evaluated; the paper also reports AAV9 immune activation and possible neurotoxicity in Section 5.1. These qualifications do not support an unqualified claim of an excellent safety and efficacy profile for CNS AAV administration. (LLM, conf=0.88)
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Vos, T., Abajobir, A. A., Abate, K. H., Abbafati, C., Abbas, K. M., Abd-Allah, F., Abdulkader, R. S., Abdulle, A. M., Abebo, T. A., Abera, S. F., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)32154-2 · Cited by 13,220 · see citing works
Polazzi, E., Mengoni, I., Caprini, M., Peña‐Altamira, E., Kurtys, E., & Monti, B. (2012) · Neurosignals · doi:10.1159/000337115 · Cited by 9,023 · see citing works
Vos, T., Barber, R. M., Bell, B., Bertozzi-Villa, A., Biryukov, S., Bolliger, I., Charlson, F., Davis, A., Degenhardt, L., Dicker, D., et al. (2015) · The Lancet · doi:10.1016/s0140-6736(15)60692-4 · Cited by 6,414 · see citing works
Astrocyte-endothelial interactions at the blood-brain barrier
Abbott, N. J., Rönnbäck, L., & Hansson, E. (2006) · Nature Reviews Neuroscience · doi:10.1038/nrn1824 · Cited by 5,595 · see citing works
In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M., & Trono, D. (1996) · Science · doi:10.1126/science.272.5259.263 · Cited by 4,903 · see citing works
Microglia-mediated neurotoxicity: uncovering the molecular mechanisms
Block, M. L., Zecca, L., & Hong, J.-S. (2007) · Nature Reviews Neuroscience · doi:10.1038/nrn2038 · Cited by 4,110 · see citing works
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
Gaj, T., Gersbach, C. A., & Barbas, C. F. (2013) · Trends in Biotechnology · doi:10.1016/j.tibtech.2013.04.004 · Cited by 3,820 · see citing works
Aggregation of Huntingtin in Neuronal Intranuclear Inclusions and Dystrophic Neurites in Brain
DiFiglia, M., Sapp, E., Chase, K. O., Davies, S. W., Bates, G. P., Vonsattel, J., & D, N. A. M. (1997) · Science · doi:10.1126/science.277.5334.1990 · Cited by 2,849 · see citing works
Bellini, M., Charalampidis, S., Herbert, P., Bonatsos, V., Crane, J., Muthusamy, A., Dor, F., & Papalois, V. E. (2022) · BioMed Research International · doi:10.1155/2738 · Cited by 2,431 · see citing works
Neuroinflammation in Parkinson’s disease: A target for neuroprotection?
Hirsch, E. C., & Hunot, S. (2009) · The Lancet Neurology · doi:10.1016/s1474-4422(09)70062-6 · Cited by 1,984 · see citing works
Astrocyte scar formation aids central nervous system axon regeneration
Anderson, M. A., Burda, J. E., Ren, Y., Ao, Y., O’Shea, T. M., Kawaguchi, R., Coppola, G., Khakh, B. S., Deming, T. J., & Sofroniew, M. V. (2016) · Nature · doi:10.1038/nature17623 · Cited by 1,851 · see citing works
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456 · cited 8× · see citing works
“…antibodies to AAV and EPO (Yang et al. 2013) Crosses blood-brain barrier, neonatal mice: neuronal tropism, adult mice: astrocyte tropism (Foust et al. 2009) GFP Spinal muscular atrophy (SMA) Motor neurons, Astrocytes Survival motor neurons Δ7 and B6 mice Intramuscular injection…”
InfluentialALS: A Disease of Motor Neurons and Their Nonneuronal Neighbors
Boillée, S., Velde, C. V., & Cleveland, D. W. (2006) · Neuron · doi:10.1016/j.neuron.2006.09.018 · Cited by 1,426 · see citing works
The Mystery and Magic of Glia: A Perspective on Their Roles in Health and Disease
Barres, B. A. (2008) · Neuron · doi:10.1016/j.neuron.2008.10.013 · Cited by 1,352 · see citing works
Frontal–subcortical neuronal circuits and clinical neuropsychiatry
Tekin, S., & Cummings, J. L. (2002) · Journal of Psychosomatic Research · doi:10.1016/s0022-3999(02)00428-2 · Cited by 1,240 · see citing works
Excitotoxicity and stroke: Identifying novel targets for neuroprotection
Lai, T. W., Zhang, S., & Wang, Y. T. (2014) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2013.11.006 · Cited by 1,106 · see citing works
Chloride Accumulation and Swelling in Endosomes Enhances DNA Transfer by Polyamine-DNA Polyplexes
Sonawane, N. D., Szoka, F. C., & Verkman, A. S. (2003) · Journal of Biological Chemistry · doi:10.1074/jbc.m308643200 · Cited by 1,083 · see citing works
Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., Bland, R. J., Young, D., Strybing, K., Eidelberg, D., et al. (2007) · The Lancet · doi:10.1016/s0140-6736(07)60982-9 · Cited by 1,067 · see citing works
4 Histology and Histopathology
(2015) · Thieme eBooks · doi:10.1055/b-0035-108581 · Cited by 1,052 · see citing works
Apoptosis and autophagy in nigral neurons of patients with Parkinson's disease.
Anglade, P., Vyas, S., Javoy-Agid, F., Herrero, M. T., Michel, P. P., Marquez, J., Prigent, A., Ruberg, M., Hirsch, É. C., & Agid, Y. (1997) · PubMed · Cited by 1,045 · see citing works
Neurodegenerative diseases and oxidative stress
Emerit, J., Edeas, M. A., & Bricaire, F. (2003) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2003.11.004 · Cited by 1,031 · see citing works
Inflammation in Alzheimer Disease--A Brief Review of the Basic Science and Clinical Literature
Wyss-Coray, T., & Rogers, J. (2012) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a006346 · Cited by 975 · see citing works
Boutin, S., Monteilhet, V., Veron, P., Leborgne, C., Benveniste, O., Montus, M. F., & Masurier, C. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.182 · Cited by 970 · see citing works
Oligodendrocytes: biology and pathology
Bradl, M., & Lassmann, H. (2010) · Acta Neuropathologica · doi:10.1007/s00401-009-0601-5 · Cited by 933 · see citing works
Glia — more than just brain glue
Allen, N. J., & Barres, B. A. (2009) · Nature · doi:10.1038/457675a · Cited by 856 · see citing works
The tripartite synapse: roles for gliotransmission in health and disease
Halassa, M. M., Fellin, T., & Haydon, P. G. (2007) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2006.12.005 · Cited by 852 · see citing works
Microglia: Scapegoat, saboteur, or something else?
Aguzzi, A., Barres, B. A., & Bennett, M. L. (2013) · Science · doi:10.1126/science.1227901 · Cited by 836 · see citing works
Molecular mechanisms of calcium-dependent neurodegeneration in excitotoxicity
Arundine, M., & Tymianski, M. (2003) · Cell Calcium · doi:10.1016/s0143-4160(03)00141-6 · Cited by 827 · see citing works
GDNF, NGF and BDNF as Therapeutic Options for Neurodegeneration
Allen, S. J., Watson, J. J., Shoemark, D. K., Barua, N. U., & Patel, N. K. (2013) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2013.01.004 · Cited by 820 · see citing works
Gene therapy clinical trials worldwide to 2012 – an update
Ginn, S., Alexander, I. E., Edelstein, M. L., Abedi, M. R., & Wixon, J. (2013) · Journal of Gene Medicine · doi:10.1002/jgm.2698 · Cited by 807 · see citing works
Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector
Blömer, U., Naldini, L., Kafri, T., Trono, D., Verma, I. M., & Gage, F. H. (1997) · Journal of Virology · doi:10.1128/jvi.71.9.6641-6649.1997 · Cited by 725 · see citing works
LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., Kostyk, S. K., Thomas, K., Sarkar, A., Siddiqui, M. S., et al. (2011) · The Lancet Neurology · doi:10.1016/s1474-4422(11)70039-4 · Cited by 681 · see citing works
Dopaminergic Neurons Protected from Degeneration by GDNF Gene Therapy
Choi-Lundberg, D. L., Lin, Q., Chang, Y., Chiang, Y. L., Hay, C. M., Mohajeri, H. M., Davidson, B. L., & Bohn, M. C. (1997) · Science · doi:10.1126/science.275.5301.838 · Cited by 622 · see citing works
Begum, A. N., Jones, M. R., Lim, G. P., Morihara, T., Kim, P., Heath, D. D., Rock, C. L., Pruitt, M. A., Yang, F., Hudspeth, B., et al. (2008) · The Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.108.137455 · Cited by 604 · see citing works
Spherical Nucleic Acid Nanoparticle Conjugates as an RNAi-Based Therapy for Glioblastoma
Jensen, S. A., Day, E. S., Ko, C. H., Hurley, L. A., Luciano, J. P., Kouri, F. M., Merkel, T. J., Luthi, A. J., Patel, P. C., Cutler, J. I., et al. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3006839 · Cited by 592 · see citing works
Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
Dong, J.-Y., Fan, P.-D., & Frizzell, R. A. (1996) · Human Gene Therapy · doi:10.1089/hum.1996.7.17-2101 · Cited by 583 · see citing works
Microglial cell dysregulation in brain aging and neurodegeneration
von Bernhardi, R., Eugenín-von Bernhardi, L., & Eugenín, J. (2015) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2015.00124 · Cited by 556 · see citing works
Drug delivery to the central nervous system by polymeric nanoparticles: What do we know?
Kreuter, J. (2014) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2013.08.008 · Cited by 554 · see citing works
RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection
Hu, W., Kaminski, R., Yang, F., Zhang, Y., Cosentino, L., Li, F., Luo, B., Alvarez-Carbonell, D., Garcia-Mesa, Y., Karn, J., et al. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1405186111 · Cited by 527 · see citing works
Aschauer, D. F., Kreuz, S., & Rumpel, S. (2013) · PLoS ONE · doi:10.1371/journal.pone.0076310 · Cited by 522 · see citing works
Wang, D. D., & Bordey, A. (2008) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2008.09.015 · Cited by 517 · see citing works
Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.72 · Cited by 501 · see citing works
Microglia Biology in Health and Disease
Garden, G. A., & Möller, T. (2006) · Journal of Neuroimmune Pharmacology · doi:10.1007/s11481-006-9015-5 · Cited by 494 · see citing works
Nanotechnological advances for the delivery of cns therapeutics.
Wong, H. L., Wu, X. Y., & Bendayan, R. (2012) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2011.10.007 · Cited by 488 · see citing works
GFAP promoter directs astrocyte-specific expression in transgenic mice
Brenner, M., Kisseberth, W., Su, Y., Besnard, F., & Messing, A. (1994) · Journal of Neuroscience · doi:10.1523/jneurosci.14-03-01030.1994 · Cited by 485 · see citing works
Dittgen, T., Nimmerjahn, A., Komai, S., Licznerski, P., Waters, J., Margrie, T. W., Helmchen, F., Denk, W., Brecht, M., & Osten, P. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0407976101 · Cited by 485 · see citing works
The life, death, and replacement of oligodendrocytes in the adult CNS
McTigue, D. M., & Tripathi, R. B. (2008) · Journal of Neurochemistry · doi:10.1111/j.1471-4159.2008.05570.x · Cited by 455 · see citing works
Palfi, S., Gurruchaga, J. M., Ralph, G. S., Lepetit, H., Lavisse, S., Buttery, P. C., Watts, C., Miskin, J., Kelleher, M., Deeley, S., et al. (2014) · The Lancet · doi:10.1016/s0140-6736(13)61939-x · Cited by 452 · see citing works
Microglia: actively surveying and shaping neuronal circuit structure and function
Wake, H., Moorhouse, A. J., Miyamoto, A., & Nabekura, J. (2013) · Trends in Neurosciences · doi:10.1016/j.tins.2012.11.007 · Cited by 448 · see citing works
N-Methyl-d-aspartate (NMDA) receptor function and excitotoxicity in Huntington's disease
FAN, M., & Raymond, L. A. (2006) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2006.11.003 · Cited by 410 · see citing works
GFAP promoter elements required for region‐specific and astrocyte‐specific expression
Lee, Y., Messing, A., Su, M., & Brenner, M. (2008) · Glia · doi:10.1002/glia.20622 · Cited by 406 · see citing works
Drug and Gene Delivery to the Brain
Pardridge, W. M. (2002) · Neuron · doi:10.1016/s0896-6273(02)01054-1 · Cited by 405 · see citing works
Jucker, M. (2010) · Nature Medicine · doi:10.1038/nm.2224 · Cited by 401 · see citing works
Results from a phase I safety trial of <i>hAADC</i> gene therapy for Parkinson disease
Eberling, J. L., Jagust, W. J., Christine, C. W., Starr, P., Larson, P., Bankiewicz, K. S., & Aminoff, M. J. (2008) · Neurology · doi:10.1212/01.wnl.0000312381.29287.ff · Cited by 370 · see citing works
Ceulemans, A.-G., Zgavc, T., Kooijman, R., Hachimi-Idrissi, S., Sarre, S., & Michotte, Y. (2010) · Journal of Neuroinflammation · doi:10.1186/1742-2094-7-74 · Cited by 351 · see citing works
Synapse Pathology in Psychiatric and Neurologic Disease
van Spronsen, M., & Hoogenraad, C. C. (2010) · Current Neurology and Neuroscience Reports · doi:10.1007/s11910-010-0104-8 · Cited by 345 · see citing works
Bevan, A. K., Duque, S., Foust, K. D., Morales, P. R., Braun, L., Schmelzer, L., Chan, C. M., McCrate, M., Chicoine, L. G., Coley, B. D., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.157 · Cited by 337 · see citing works
Niranjan, R. (2013) · Molecular Neurobiology · doi:10.1007/s12035-013-8483-x · Cited by 331 · see citing works
Buffo, A., Rolando, C., & Ceruti, S. (2009) · Biochemical Pharmacology · doi:10.1016/j.bcp.2009.09.014 · Cited by 321 · see citing works
Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing
Kaminski, R., Chen, Y., Fischer, T., Tedaldi, E., Napoli, A., Zhang, Y., Karn, J., Hu, W., & Khalili, K. (2016) · Scientific Reports · doi:10.1038/srep22555 · Cited by 320 · see citing works
Regan, M. R., Huang, Y. H., Kim, Y., Dykes‐Hoberg, M., Jin, L., Watkins, A. M., Bergles, D. E., & Rothstein, J. D. (2007) · Journal of Neuroscience · doi:10.1523/jneurosci.0790-07.2007 · Cited by 314 · see citing works
TAT-conjugated nanoparticles for the CNS delivery of anti-HIV drugs
Rao, K. S., Reddy, M. K., Horning, J. L., & Labhasetwar, V. D. (2008) · Biomaterials · doi:10.1016/j.biomaterials.2008.08.004 · Cited by 314 · see citing works
Self-citationTargeting Astrocytes Ameliorates Neurologic Changes in a Mouse Model of Alzheimer's Disease
Furman, J. L., Sama, D. M., Gant, J. C., Beckett, T. L., Murphy, M. P., Bachstetter, A. D., Van Eldik, L. J., & Norris, C. M. (2012) · The Journal of Neuroscience · doi:10.1523/jneurosci.2323-12.2012 · Cited by 313 · see citing works
Xin, H., Sha, X., Jiang, X., Zhang, W., Chen, L., & Fang, X. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.07.046 · Cited by 311 · see citing works
Goula, D., Rémy, J.-S., Erbacher, P., Wąsowicz, M., Levi, G., Abdallah, B., & DEMENEIX, B. A. (1998) · Gene Therapy · doi:10.1038/sj.gt.3300635 · Cited by 309 · see citing works
Cold Spring Harbor ProtocolsPreprint
(2014) · Cold Spring Harbor Protocols · doi:10.1101/pdb/ · Cited by 303 · see citing works
Selective and Rapid Uptake of Adeno-Associated Virus Type 2 in Brain
Bartlett, J. S., Samulski, R. J., & McCown, T. J. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.8-1181 · Cited by 301 · see citing works
Carson, M. J., Cameron Thrash, J., & Walter, B. (2006) · Clinical Neuroscience Research · doi:10.1016/j.cnr.2006.09.004 · Cited by 299 · see citing works
Zhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., Wang, H., Mueller, C., Sena-Esteves, M., Brown, R., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.98 · Cited by 291 · see citing works
Peel, A., Zolotukhin, S., Schrimsher, G. W., Muzyczka, N., & Reier, P. J. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300358 · Cited by 289 · see citing works
Calcium dysregulation in amyotrophic lateral sclerosis
Großkreutz, J., Bosch, L. V. D., & Keller, B. U. (2010) · Cell Calcium · doi:10.1016/j.ceca.2009.12.002 · Cited by 284 · see citing works
Brain-specific expression of an exogenous gene after i.v. administration
Shi, N., Zhang, Y., Zhu, C., Boado, R. J., & Pardridge, W. M. (2001) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.221450098 · Cited by 273 · see citing works
Microglia and neuroprotection: From in vitro studies to therapeutic applications
Polazzi, E., & Monti, B. (2010) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2010.06.009 · Cited by 260 · see citing works
Neuronal changes in normal human aging and Alzheimer's disease.
Uylings, H., & de Brabander, J. D. (2002) · Brain and Cognition · doi:10.1006/brcg.2001.1500 · Cited by 260 · see citing works
Ascl1 Converts Dorsal Midbrain Astrocytes into Functional Neurons In Vivo
Liu, Y., Miao, Q., Yuan, J., Han, S., Zhang, P., Li, S., Rao, Z., Zhao, W., Ye, Q., Geng, J., et al. (2015) · Journal of Neuroscience · doi:10.1523/jneurosci.3975-14.2015 · Cited by 257 · see citing works
Thomas, C. E., Schiedner, G., Kochanek, S., Castro, M. G., & Lowenstein, P. R. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.120474397 · Cited by 253 · see citing works
Polymeric Nanoparticles for Nonviral Gene Therapy Extend Brain Tumor Survival in Vivo
Mangraviti, A., Tzeng, S. Y., Kozielski, K., Wang, Y., Jin, Y., Gullotti, D. M., Pedone, M., Buaron, N., Liu, A., Wilson, D. R., et al. (2015) · ACS Nano · doi:10.1021/nn504905q · Cited by 251 · see citing works
Chui, R. W., & Dorovini‐Zis, K. (2010) · Journal of Neuroinflammation · doi:10.1186/1742-2094-7-1 · Cited by 250 · see citing works
Kim, B. O., Liu, Y., Ruan, Y., Xu, Z. C., Schantz, L. D., & He, J. J. (2003) · American Journal Of Pathology · doi:10.1016/s0002-9440(10)64304-0 · Cited by 248 · see citing works
Joris, F., Manshian, B. B., Peynshaert, K., Smedt, S. C. D., Braeckmans, K., & Soenen, S. J. (2013) · Chemical Society Reviews · doi:10.1039/c3cs60145e · Cited by 235 · see citing works
Pathology of demyelinating diseases.
Popescu, B. F. G., & Lucchinetti, C. (2012) · Annual Review of Pathology · doi:10.1146/annurev-pathol-011811-132443 · Cited by 228 · see citing works
Cerullo, V., Pesonen, S. A., Diaconu, I., Escutenaire, S., Arstila, P. T., Ugolini, M., Nokisalmi, P., Raki, M., Laasonen, L., Särkioja, M., et al. (2010) · Cancer Research · doi:10.1158/0008-5472.can-09-3567 · Cited by 226 · see citing works
A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer’s disease
Rafii, M. S., Baumann, T. L., Bakay, R. A. E., Ostrove, J. M., Siffert, J., Fleisher, A. S., Herzog, C. D., Barba, D., Pay, M., Salmon, D. P., et al. (2014) · Alzheimer's & Dementia · doi:10.1016/j.jalz.2013.09.004 · Cited by 223 · see citing works
Guerrero‐Cazares, H., Tzeng, S. Y., Young, N. P., Abutaleb, A., Quiñones‐Hinojosa, A., & Green, J. J. (2014) · ACS Nano · doi:10.1021/nn501197v · Cited by 221 · see citing works
Mitochondrial dysfunction and its role in motor neuron degeneration in ALS
Manfredi, G., & Xu, Z. (2005) · Mitochondrion · doi:10.1016/j.mito.2005.01.002 · Cited by 215 · see citing works
HIV-1 associated dementia: symptoms and causes
Ghafouri, M., Amini, S., Khalili, K., & Sawaya, B. E. (2006) · Retrovirology · doi:10.1186/1742-4690-3-28 · Cited by 215 · see citing works
Yang, B., Li, S., Wang, H., Guo, Y., Gessler, D. J., Cao, C., Su, Q., Kramer, J., Zhong, L., Ahmed, S. S., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.68 · Cited by 213 · see citing works
Cell number changes in Alzheimer's disease relate to dementia, not to plaques and tangles
Andrade-Moraes, C. H., de Oliveira-Pinto, A. V., Castro-Fonseca, E., da Silva, C. G., Guimarães, D. M., Szczupak, D., Parente-Bruno, D. R., Carvalho, L. R. B., Polichiso, L., Valério‐Gomes, B., et al. (2013) · Brain · doi:10.1093/brain/awt273 · Cited by 213 · see citing works
Midoux, P., Breuzard, G., Gomez, J., & Pichon, C. (2008) · Current Gene Therapy · doi:10.2174/156652308786071014 · Cited by 211 · see citing works
Шевцова, З. І., Malik, I., Michel, U., Bähr, M., & Kügler, S. (2004) · Experimental Physiology · doi:10.1113/expphysiol.2004.028159 · Cited by 207 · see citing works
Distinct neuroinflammatory profile in post-mortem human Huntington's disease
Silvestroni, A., Faull, R. L. M., Strand, A. D., & Möller, T. (2009) · Neuroreport · doi:10.1097/wnr.0b013e32832e34ee · Cited by 204 · see citing works
Ferrari, C. C., Depino, A. M., Prada, F., Muraro, N. I., Campbell, S. J., Podhajcer, O. L., Perry, V. H., Anthony, D. C., & Pitossi, F. J. (2004) · American Journal Of Pathology · doi:10.1016/s0002-9440(10)63438-4 · Cited by 201 · see citing works
Gardner, J., & Ghorpade, A. (2003) · Journal of Neuroscience Research · doi:10.1002/jnr.10835 · Cited by 199 · see citing works
Self-citationWatson, D. J., Kobinger, G., Passini, M. A., Wilson, J. M., & Wolfe, J. H. N. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0584 · Cited by 198 · see citing works
Neuroinflammation modulates distinct regional and temporal clinical responses in ALS mice
Beers, D. R., Zhao, W., Liao, B., Kano, O., Wang, J., Huang, A., Appel, S. H., & Henkel-Tigges, J. (2010) · Brain Behavior and Immunity · doi:10.1016/j.bbi.2010.12.008 · Cited by 197 · see citing works
A further update on the role of excitotoxicity in the pathogenesis of Parkinson’s disease
Ambrosi, G., Cerri, S., & Blandini, F. (2014) · Journal of Neural Transmission · doi:10.1007/s00702-013-1149-z · Cited by 195 · see citing works
The mTOR kinase inhibitor rapamycin decreases iNOS mRNA stability in astrocytes
Lisi, L., Navarra, P., Feinstein, D., & Russo, C. D. (2011) · Journal of Neuroinflammation · doi:10.1186/1742-2094-8-1 · Cited by 194 · see citing works
Alberdi, E. M., Wyssenbach, A., Alberdi, M., Sánchez‐Gómez, M. V., Cavaliere, F., Rodriguez, J. J., Verkhratsky, A., & Matute, C. (2013) · Aging Cell · doi:10.1111/acel.12054 · Cited by 188 · see citing works
Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain
Cearley, C. N., Vandenberghe, L. H., Parente, M. K., Carnish, E. R., Wilson, J. M., & Wolfe, J. H. (2008) · Molecular Therapy · doi:10.1038/mt.2008.166 · Cited by 182 · see citing works
Molecular evolution of adeno-associated virus for enhanced glial gene delivery
Koerber, J. T., Klimczak, R., Jang, J.-H., Dalkara, D., Flannery, J. G., & Schaffer, D. V. (2009) · Molecular Therapy · doi:10.1038/mt.2009.184 · Cited by 181 · see citing works
Astrogliosis as a therapeutic target for neurodegenerative diseases
Colangelo, A. M., Alberghina, L., & Papa, M. (2014) · Neuroscience Letters · doi:10.1016/j.neulet.2014.01.014 · Cited by 177 · see citing works
Schuster, D. J., Dykstra, J. A., Riedl, M. S., Kitto, K. F., Belur, L. R., McIvor, R. S., Elde, R. P., Fairbanks, C. A., & Vulchanova, L. (2014) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2014.00042 · Cited by 177 · see citing works
McMorris, F. A., & McKinnon, R. D. (1996) · Brain Pathology · doi:10.1111/j.1750-3639.1996.tb00858.x · Cited by 175 · see citing works
Science, medicine, and the future: Parkinson's disease.
Schapira, A. (1999) · British medical journal · doi:10.1136/bmj.318.7179.311 · Cited by 174 · see citing works
Inflammation and neurovascular changes in amyotrophic lateral sclerosis
Evans, M. C., Couch, Y., Sibson, N. R., & Turner, M. R. (2012) · Molecular and Cellular Neuroscience · doi:10.1016/j.mcn.2012.10.008 · Cited by 174 · see citing works
Hwang, D., Carlezon, W. A., Ole Isacson, A., & Kim, K. (2001) · Human Gene Therapy · doi:10.1089/104303401750476230 · Cited by 172 · see citing works
Abordo‐Adesida, E., Follenzi, A., Barcia, C., Sciascia, S. A., Castro, M. G., Naldini, L., & Lowenstein, P. R. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.741 · Cited by 171 · see citing works
Convection-enhanced delivery for the treatment of brain tumors
Debinski, W., & Tatter, S. B. (2009) · Expert Review of Neurotherapeutics · doi:10.1586/ern.09.99 · Cited by 170 · see citing works
Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy
Bartus, R. T., Weinberg, M. S., & Samulski, R. J. (2013) · Molecular Therapy · doi:10.1038/mt.2013.281 · Cited by 165 · see citing works
Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain
Rungta, R. L., Choi, H. B., Lin, P. J., Ko, R. W., Ashby, D., Nair, J., Manoharan, M., Cullis, P. R., & MacVicar, B. A. (2013) · Molecular Therapy—Nucleic Acids · doi:10.1038/mtna.2013.65 · Cited by 165 · cited 9× · see citing works
“Arginine-modified polyplexes (Morris and Labhasetwar 2015), transactivator of transcription-conjugated NPs (Rao et al. 2008) and AAV vector serotype 9 (Foust et al. 2009) crossed the BBB when injected intravenously in rodent models, indicating that tailor-made NPs with surface modifications and genetically modified vi…”
InfluentialBemelmans, A., Horellou, P., Pradier, L., Brunet, I., Colin, P., & Mallet, J. J. (1999) · Human Gene Therapy · doi:10.1089/10430349950016393 · Cited by 164 · see citing works
Targeted transgene expression in rat brain using lentiviral vectors
Jakobsson, J., Ericson, C., Swanberg, M., Björk, E., & Lundberg, C. (2003) · Journal of Neuroscience Research · doi:10.1002/jnr.10719 · Cited by 164 · see citing works
Biomedical Applications of Nanodiamonds in Imaging and Therapy
Perevedentseva, E. V., Lin, Y., Jani, M., & Cheng, C. (2013) · Nanomedicine · doi:10.2217/nnm.13.183 · Cited by 162 · see citing works
Persidsky, Y., Zheng, J., Miller, D. W., & Gendelman, H. E. (2000) · Journal of Leukocyte Biology · doi:10.1189/jlb.68.3.413 · Cited by 161 · see citing works
Lawlor, P. A., Bland, R. J., Mouravlev, A., Young, D., & During, M. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.170 · Cited by 161 · see citing works
The biology of CNS remyelination
Franklin, R. J. M., & Kotter, M. (2008) · Journal of Neurology · doi:10.1007/s00415-008-1004-6 · Cited by 160 · see citing works
Engineered lentiviral vector targeting astrocytes <i>In vivo</i>
Colin, A., Faideau, M., Dufour, N., Auregan, G., Hassig, R., Andrieu, T., Brouillet, E., Hantraye, P., Bonvento, G., & Déglon, N. (2009) · Glia · doi:10.1002/glia.20795 · Cited by 158 · see citing works
Samaranch, L., Sebastián, W. S., Kells, A. P., Salegio, E. A., Heller, G., Bringas, J. R., Pivirotto, P., DeArmond, S. J., Forsayeth, J., & Bankiewicz, K. S. (2013) · Molecular Therapy · doi:10.1038/mt.2013.266 · Cited by 157 · see citing works
Byrnes, A. P., MacLaren, R. E., & Charlton, H. M. (1996) · Journal of Neuroscience · doi:10.1523/jneurosci.16-09-03045.1996 · Cited by 155 · see citing works
Adeno-associated virus vectors: activity and applications in the CNS
Peel, A. L., & Klein, R. L. (2000) · Journal of Neuroscience Methods · doi:10.1016/s0165-0270(00)00183-7 · Cited by 155 · see citing works
Kügler, S., Lingor, P., Schöll, U., Zolotukhin, S., & Bähr, M. (2003) · Virology · doi:10.1016/s0042-6822(03)00162-4 · Cited by 153 · see citing works
Kim, I., Shin, J., Kim, S.-W., Choi, S., Ahn, J., Han, P., Park, J. S., & Lee, J.-K. (2012) · Molecular Therapy · doi:10.1038/mt.2011.291 · Cited by 150 · see citing works
Brain-targeted delivery of paclitaxel using glutathione-coated nanoparticles for brain cancers
Geldenhuys, W. J., Mbimba, T., Bui, T., Harrison, K. A., & Sutariya, V. B. (2011) · Journal of drug targeting · doi:10.3109/1061186x.2011.589435 · Cited by 144 · see citing works
Ezcurra, A. L. D. L., Chertoff, M., Ferrari, C. C., Graciarena, M., & Pitossi, F. J. (2009) · Neurobiology of Disease · doi:10.1016/j.nbd.2009.11.018 · Cited by 140 · see citing works
Viral vectors, tools for gene transfer in the nervous system
Hermens, W. T. J. M. C., & Verhaagen, J. (1998) · Progress in Neurobiology · doi:10.1016/s0301-0082(98)00007-0 · Cited by 140 · see citing works
Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain
Wang, C., Wang, C.-M., Clark, K. R., & Sferra, T. J. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302011 · Cited by 139 · see citing works
Potential Use of Polymeric Nanoparticles for Drug Delivery Across the Blood-Brain Barrier
Tosi, G., Bortot, B., Ruozi, B., Dolcetta, D., Vandelli, M. A., Forni, F., & Severini, G. M. (2013) · Current Medicinal Chemistry · doi:10.2174/0929867311320170006 · Cited by 136 · see citing works
Pilakka‐Kanthikeel, S., Atluri, V. S. R., Sagar, V., Saxena, S. K., & Nair, M. (2013) · PLoS ONE · doi:10.1371/journal.pone.0062241 · Cited by 135 · see citing works
Nicchia, G. P., Frigeri, A., Liuzzi, G. M., & Svelto, M. (2003) · The FASEB Journal · doi:10.1096/fj.02-1183fje · Cited by 135 · see citing works
Zhang, Y., Schlachetzki, F., Zhang, Y.-F., Boado, R. J., & Pardridge, W. M. (2004) · Human Gene Therapy · doi:10.1089/104303404322959498 · Cited by 134 · see citing works
Glial promoter selectivity following AAV-delivery to the immature brain
von Jonquieres, G., Mersmann, N., Klugmann, C. B., Harasta, A. E., Lutz, B., Teahan, O., Housley, G. D., Fröhlich, D., Krämer-Albers, E.-M., & Klugmann, M. (2013) · PLoS ONE · doi:10.1371/journal.pone.0065646 · Cited by 133 · 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
Microglia as a Pharmacological Target in Infectious and Inflammatory Diseases of the Brain
Rock, R. B., & Peterson, P. K. (2006) · Journal of Neuroimmune Pharmacology · doi:10.1007/s11481-006-9012-8 · Cited by 130 · see citing works
Trophic Factor Gene Therapy for Parkinson's Disease
Kordower, J. H., & Björklund, A. (2013) · Movement Disorders · doi:10.1002/mds.25344 · Cited by 129 · see citing works
Benkhelifa‐Ziyyat, S., Besse, A., Roda, M., Duqué, S. I., Astord, S., Carcenac, R., Marais, T., & Barkats, M. (2013) · Molecular Therapy · doi:10.1038/mt.2012.261 · Cited by 128 · see citing works
Hu, X., Zhang, D., Pang, H., Caudle, W. M., Li, Y., Gao, H., Liu, Y., Li, Q., Wilson, B. C., Monte, D. A. D., et al. (2008) · The Journal of Immunology · doi:10.4049/jimmunol.181.10.7194 · Cited by 125 · see citing works
Towne, C., Schneider, B. L., Kieran, D., Redmond, D. E., & Aebischer, P. (2010) · Gene Therapy · doi:10.1038/gt.2009.119 · Cited by 124 · see citing works
Tuinstra, H. M., Avilés, M. O., Shin, S., Holland, S. J., Zelivyanskaya, M. L., Fast, A. G., Ko, S. Y., Margul, D. J., Bartels, A. K., Boehler, R. M., et al. (2011) · Biomaterials · doi:10.1016/j.biomaterials.2011.11.002 · Cited by 122 · see citing works
Sandhu, J. K., Gardaneh, M., Iwasiow, R. M., Lanthier, P. H., Gangaraju, S., Ribecco‐Lutkiewicz, M., Tremblay, R. G., Kiuchi, K., & Sikorska, M. (2008) · Neurobiology of Disease · doi:10.1016/j.nbd.2008.11.016 · Cited by 112 · see citing works
Su, X., Kells, A. P., Huang, E. J., Lee, H. S., Hadaczek, P., Beyer, J., Bringas, J., Pivirotto, P., Penticuff, J., Eberling, J. L., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.103 · Cited by 112 · see citing works
Mechanisms of damage to myelin and oligodendrocytes and their relevance to disease
Merrill, & Scolding, N. J. (1999) · Neuropathology and Applied Neurobiology · doi:10.1046/j.1365-2990.1999.00200.x · Cited by 111 · see citing works
Enhanced transfection of primary cortical cultures using arginine-grafted PAMAM dendrimer, PAMAM-Arg
Kim, J., Choi, J. S., Nam, K., Lee, M., Park, J., & Lee, J.-K. (2006) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.05.011 · Cited by 111 · see citing works
Ciesielska, A., Hadaczek, P., Mittermeyer, G., Zhou, S., Wright, J. F., Bankiewicz, K. S., & Forsayeth, J. (2013) · Molecular Therapy · doi:10.1038/mt.2012.167 · Cited by 108 · see citing works
Giralt, A., Friedman, H. C., Caneda-Ferrón, B., Urbán, N., Moreno, E., Rubio, N., Blanco, J., Peterson, A. C., Canals, J. M., & Alberch, J. (2010) · Gene Therapy · doi:10.1038/gt.2010.71 · Cited by 106 · see citing works
Accumulation of silver nanoparticles by cultured primary brain astrocytes
Luther, E. M., Koehler, Y., Diendorf, J., Epple, M., & Dringen, R. (2011) · Nanotechnology · doi:10.1088/0957-4484/22/37/375101 · Cited by 106 · see citing works
In vivo delivery of siRNA to the brain by carbosilane dendrimer
Serramía, M. J., Álvarez, S., Fuentes‐Paniagua, E., Clemente, M. I., Sánchez‐Nieves, J., Gómez, R., de la Mata, J., & Muñoz‐Fernández, M. Á. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2014.12.042 · Cited by 105 · see citing works
Rafi, M. A., Rao, H. Z., Passini, M. A., Curtis, M. T., Vanier, M. T., Zaka, M., Luzi, P., Wolfe, J. H. N., & Wenger, D. A. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2004.12.020 · Cited by 105 · see citing works
Prakash, S., Tomaro-Duchesneau, C., Saha, S., Kahouli, & Malhotra, M. (2013) · International Journal of Nanomedicine · doi:10.2147/ijn.s43683 · Cited by 104 · see citing works
Gerdes, C. A., Castro, M. G., & Lowenstein, P. R. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0140 · Cited by 102 · see citing works
Intrathecal polymer-based interleukin-10 gene delivery for neuropathic pain
Milligan, E. D., Soderquist, R. G., Malone, S. M., Mahoney, J. H., Hughes, T., Langer, S. J., Sloane, E. M., Maier, S. F., Leinwand, L. A., Watkins, L. R., et al. (2006) · Neuron Glia Biology · doi:10.1017/s1740925x07000488 · Cited by 102 · see citing works
Weismann, C. M., Ferreira, J. S., Keeler, A. M., Su, Q., Qui, L., Shaffer, S. A., Xu, Z., Gao, G., & Sena‐Esteves, M. (2015) · Human Molecular Genetics · doi:10.1093/hmg/ddv168 · Cited by 102 · see citing works
Rahim, A. A., Wong, A. M. S., Howe, S. J., Buckley, S. M. K., Acosta-Saltos, A. D., Elston, K. E., Ward, N. J., Philpott, N. J., Cooper, J. D., Anderson, P. N., et al. (2009) · Gene Therapy · doi:10.1038/gt.2008.186 · Cited by 101 · see citing works
Neuron‐specific delivery of nucleic acids mediated by Tet1‐modified poly(ethylenimine)
Park, I., Lasiene, J., Chou, S.-H. S., Horner, P. J., & Pun, S. H. (2007) · The Journal of Gene Medicine · doi:10.1002/jgm.1062 · Cited by 100 · see citing works
Cucchiarini, M., Ren, X., Perides, G., & Terwilliger, E. F. (2003) · Gene Therapy · doi:10.1038/sj.gt.3301925 · Cited by 100 · see citing works
McDowell, F. (1962) · Archives of Neurology · doi:10.1001/archneur.1962.04210060109014 · Cited by 99 · see citing works
Cisneros, I. E., & Ghorpade, A. (2014) · Neuropharmacology · doi:10.1016/j.neuropharm.2014.06.011 · Cited by 98 · see citing works
Self-citationThe potential of adeno-associated viral vectors for gene delivery to muscle tissue
Wang, D., Li, Z., Nahid, M. A., & Gao, G. (2014) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2014.871258 · Cited by 97 · see citing works
The Involvement of Neuroinflammation and Kynurenine Pathway in Parkinson's Disease
Zinger, A., Barcia, C., Herrero, M. T., & Guillemin, G. J. (2011) · Parkinson s Disease · doi:10.4061/2011/716859 · Cited by 95 · see citing works
Drinkut, A., Tereshchenko, Y., Schulz, J. B., Bähr, M., & Kügler, S. (2011) · Molecular Therapy · doi:10.1038/mt.2011.249 · Cited by 95 · see citing works
Escartin, C., Brouillet, E., Gubellini, P., Trioulier, Y., Jacquard, C., Smadja, C., Knott, G. W., Goff, L. K., Déglon, N., Hantraye, P., et al. (2006) · Journal of Neuroscience · doi:10.1523/jneurosci.0302-06.2006 · Cited by 95 · see citing works
Rahim, A. A., Wong, A. M. S., Hoefer, K., Buckley, S. M. K., Mattar, C. N. Z., Cheng, S. H., Chan, J. K. Y., Cooper, J. D., & Waddington, S. N. (2011) · The FASEB Journal · doi:10.1096/fj.11-182311 · Cited by 95 · see citing works
A molecular and genetic arsenal for systems neuroscience
Callaway, E. M. (2005) · Trends in Neurosciences · doi:10.1016/j.tins.2005.01.007 · Cited by 94 · see citing works
MicroRNA‐145 as one negative regulator of astrogliosis
Wang, C., Yang, S., & Tzeng, S. (2014) · Glia · doi:10.1002/glia.22743 · Cited by 93 · see citing works
Hermann, D. M., Kılıç, E., Kügler, S., Isenmann, S., & Bähr, M. (2001) · Neurobiology of Disease · doi:10.1006/nbdi.2001.0399 · Cited by 92 · see citing works
Wang, J., Chen, Q., Wang, X., Wang, Q.-C., Wang, Y., Cheng, H., Guo, C., Sun, Q., Chen, Q., & Tang, T.-S. (2012) · Journal of Biological Chemistry · doi:10.1074/jbc.m112.407726 · Cited by 92 · see citing works
Jaffer, H., Morris, V. B., Stewart, D., & Labhasetwar, V. D. (2011) · Drug Delivery and Translational Research · doi:10.1007/s13346-011-0046-y · Cited by 92 · see citing works
Self-citationApplications of gene therapy to the CNS
Blömer, U., Naldini, L., Verma, I. M., Trono, D., & Gage, F. H. (1996) · Human Molecular Genetics · doi:10.1093/hmg/5.supplement_1.1397 · Cited by 88 · see citing works
Chen, H., McCarty, D., Bruce, A., Suzuki, K., & Suzuki, K. (1998) · Gene Therapy · doi:10.1038/sj.gt.3300547 · Cited by 87 · cited 4× · see citing works
“An AAV2 vector encoding a MBP promoter precisely targeted oligodendrocytes in vitro and in vivo, in rat cells and mice, respectively (Chen et al. 1998).”
InfluentialTargeting Beta Amyloid: A Clinical Review of Immunotherapeutic Approaches in Alzheimer's Disease
Lobello, K., Ryan, J. M., Liu, E., Rippon, G. A., & Black, R. S. (2012) · International Journal of Alzheimer s Disease · doi:10.1155/2012/628070 · Cited by 86 · see citing works
Liu, G., Martins, I. H., Chiorini, J. A., & Davidson, B. L. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302554 · Cited by 84 · see citing works
D, N. A. M., & DiFiglia, M. (2014) · Movement Disorders · doi:10.1002/mds.26020 · Cited by 80 · see citing works
Uptake of dimercaptosuccinate-coated magnetic iron oxide nanoparticles by cultured brain astrocytes
Geppert, M., Hohnholt, M. C., Thiel, K., Nürnberger, S., Grunwald, I., Rezwan, K., & Dringen, R. (2011) · Nanotechnology · doi:10.1088/0957-4484/22/14/145101 · Cited by 80 · see citing works
Highly PEGylated DNA Nanoparticles Provide Uniform and Widespread Gene Transfer in the Brain
Mastorakos, P., Zhang, C., Berry, S., Oh, Y., Lee, S., Eberhart, C. G., Woodworth, G. F., Suk, J. S., & Hanes, J. (2015) · Advanced Healthcare Materials · doi:10.1002/adhm.201400800 · Cited by 80 · see citing works
Long-term Transgene Expression in the Central Nervous System Using DNA Nanoparticles
Yurek, D. M., Fletcher, A. M., Smith, G. M., Seroogy, K. B., Ziady, A. G., Molter, J. P., Kowalczyk, T. H., Padegimas, L., & Cooper, M. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.2 · Cited by 79 · see citing works
Activated astrocytes: a therapeutic target in Alzheimer’s disease?
Fuller, S. L., Münch, G. W., & Steele, M. L. (2009) · Expert Review of Neurotherapeutics · doi:10.1586/ern.09.111 · Cited by 77 · see citing works
Cisneros, I. E., & Ghorpade, A. (2012) · Current HIV Research · doi:10.2174/157016212802138832 · Cited by 74 · see citing works
Self-citationRodent models for HIV-associated neurocognitive disorders
Gorantla, S., Poluektova, L. Y., & Gendelman, H. E. (2012) · Trends in Neurosciences · doi:10.1016/j.tins.2011.12.006 · Cited by 74 · see citing works
Application of recombinant adenovirus for in vivo gene delivery to spinal cord
Liu, Y., Himes, B. T., Moul, J., Huang, W., Chow, S. Y., Tessler, A. R., & Fischer, I. (1997) · Brain Research · doi:10.1016/s0006-8993(97)00587-8 · Cited by 72 · see citing works
Oxidative Stress and Therapeutic Approaches in HIV Dementia
Steiner, J. P., Haughey, N. J., Li, W., Venkatesan, A., Anderson, C., Reid, R., Malpica-Llanos, T., Pocernich, C. B., Butterfield, D. A., & Nath, A. (2006) · Antioxidants and Redox Signaling · doi:10.1089/ars.2006.8.2089 · Cited by 71 · see citing works
Dormond, E., Perrier, M., & Kamen, A. (2008) · Biotechnology Advances · doi:10.1016/j.biotechadv.2008.10.003 · Cited by 70 · see citing works
Non-viral gene therapy for neurological diseases, with an emphasis on targeted gene delivery
Rogers, M., & Rush, R. A. (2011) · Journal of Controlled Release · doi:10.1016/j.jconrel.2011.08.026 · Cited by 70 · see citing works
Lu, S., Tremblay, M., King, I. L., Qi, J., Reynolds, H. M., Marker, D. F., Varrone, J. J., Majewska, A. K., Dewhurst, S., & Gelbard, H. A. (2011) · PLoS ONE · doi:10.1371/journal.pone.0023915 · Cited by 70 · see citing works
Jiménez, J. L., Clemente, M. I., Weber, N. D., Sánchez, J. C., Ortega, P., de la Mata, F. J., Gómez, R., García, D., López-Fernández, L. A., & Muñoz‐Fernández, M. Á. (2010) · BioDrugs · doi:10.2165/11538400-000000000-00000 · Cited by 70 · see citing works
Targeted nonviral delivery vehicles to neural progenitor cells in the mouse subventricular zone
Kwon, E. J., Lasiene, J., Jacobson, B. E., Park, I., Horner, P. J., & Pun, S. H. (2009) · Biomaterials · doi:10.1016/j.biomaterials.2009.11.086 · Cited by 70 · see citing works
Xia, C., Yin, H. H., Borlongan, C. V., Chao, J., & Chao, L. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.1243 · Cited by 68 · see citing works
Kiyota, T., Yamamoto, M., Schroder, B., Jacobsen, M. T., Swan, R. J., Lambert, M. P., Klein, W. L., Gendelman, H. E., Ransohoff, R. M., & Ikezu, T. (2009) · Molecular Therapy · doi:10.1038/mt.2009.44 · Cited by 68 · see citing works
Katz, M. G., Fargnoli, A. S., Williams, R. D., & Bridges, C. R. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.2517 · Cited by 67 · see citing works
Homs, J., Ariza, L., Pagès, G., Udina, E., Navarro, X., Chillón, M., & Bosch, A. (2011) · Gene Therapy · doi:10.1038/gt.2011.7 · Cited by 67 · see citing works
Gene transfer into the central nervous system in vivo using a recombinanat lentivirus vector
Lai, Z., & Brady, R. O. (2002) · Journal of Neuroscience Research · doi:10.1002/jnr.10137 · Cited by 67 · see citing works
Andrews, J. L., Kadan, M. J., Gorziglia, M. I., Kaleko, M., & Connelly, S. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0264 · Cited by 66 · see citing works
Bellini, M. J., Hereñú, C. B., Goya, R. G., & García‐Segura, L. M. (2011) · Journal of Neuroinflammation · doi:10.1186/1742-2094-8-21 · Cited by 66 · see citing works
Chaturvedi, M., Molino, Y., Sreedhar, B., Khrestchatisky, M., & Kaczmarek, L. (2014) · International Journal of Nanomedicine · doi:10.2147/ijn.s54750 · Cited by 65 · see citing works
Nanotechnology for CNS delivery of bio-therapeutic agents
Shah, L. K., Yadav, S. K., & Amiji, M. M. (2013) · Drug Delivery and Translational Research · doi:10.1007/s13346-013-0133-3 · Cited by 64 · see citing works
Navarro, V., Millecamps, S., Geoffroy, M.-C., Robert, J. J., Valin, A., Mallet, J. J., & Salle, G. L. G. L. (1999) · Gene Therapy · doi:10.1038/sj.gt.3301008 · Cited by 63 · see citing works
Shen, F., Fan, Y., Su, H., Zhu, Y., Chen, Y., Liu, W., Young, W. L., & Yang, G. (2007) · Gene Therapy · doi:10.1038/sj.gt.3303048 · Cited by 63 · see citing works
Segovia, J., Aragón, P. V., & Brenner, M. (1998) · Gene Therapy · doi:10.1038/sj.gt.3300776 · Cited by 63 · see citing works
Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain
San Sebastian, W., Samaranch, L., Heller, G., Kells, A. P., Bringas, J., Pivirotto, P., Forsayeth, J., & Bankiewicz, K. S. (2013) · Gene Therapy · doi:10.1038/gt.2013.48 · Cited by 62 · see citing works
Pseudotype-dependent lentiviral transduction of astrocytes or neurons in the rat substantia nigra
Cannon, J. R., Sew, T., Montero, L. M., Burton, E. A., & Greenamyre, J. T. (2010) · Experimental Neurology · doi:10.1016/j.expneurol.2010.10.016 · Cited by 61 · see citing works
Gholizadeh, S., Tharmalingam, S., MacAldaz, M. E., & Hampson, D. R. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2013.076 · Cited by 61 · see citing works
Adam, S. A., Schnell, O., Pöschl, J., Eigenbrod, S., Kretzschmar, H. A., Tonn, J., & Schüller, U. (2012) · Brain Pathology · doi:10.1111/j.1750-3639.2012.00592.x · Cited by 60 · see citing works
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Helmschrodt, C., Höbel, S., Schöniger, S., Bauer, A., Bonicelli, J., Gringmuth, M., Fietz, S. A., Aigner, A. M., Richter, A., & Richter, F. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.08.013 · Cited by 154 · see citing works
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Wu, J., Ji, A., Wang, Z., Qiang, G.-H., Qu, Z., Wu, J., & Jiang, C. (2018) · Scientific Reports · doi:10.1038/s41598-018-20505-y · Cited by 91 · see citing works
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Thuringer, D., Solary, É., & Garrido, C. (2017) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2017.00246 · Cited by 8 · see citing works
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Russo, A., Maiorano, G., Cortese, B., D’Amone, S., Invidia, A., Quattrini, A., Romano, A., Gigli, G., & Palamà, I. E. (2024) · Nanoscale · doi:10.1039/d4nr03159h · Cited by 7 · see citing works
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Abdallah, K., Nadeau, F., Bergeron, F., Blouin, S., Blais, V., Bradbury, K. M., Lavoie, C. L., Parent, J., & Gendron, L. (2018) · Scientific Reports · doi:10.1038/s41598-018-25626-y · Cited by 8 · see citing works
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Rudd, P. A., & Herrero, L. J. (2018) · InTech eBooks · doi:10.5772/intechopen.71071 · Cited by 4 · see citing works
Ghali, G. Z., & Ghali, M. G. Z. (2020) · Molecular Biology Reports · doi:10.1007/s11033-020-05427-1 · Cited by 3 · see citing works
RetractedEffective Gene Transfer with Non‐Viral Vectors
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Wu, L., Sun, W., Huang, L., Sun, L., Dou, J., & Lu, G. (2025) · Organogenesis · doi:10.1080/15476278.2025.2489667 · Cited by 2 · see citing works
Ghali, M. G. Z., Arborelius, U. P., & Yasargil, M. G. (2020) · Journal of Receptors and Signal Transduction · doi:10.1080/10799893.2020.1759091 · Cited by 2 · see citing works
RetractedLockowandt, M., Günther, D. M., Quintino, L., Breger, L. S., Isaksson, C., & Lundberg, C. (2020) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2019.108542 · Cited by 2 · see citing works
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Parhi, R., Jena, B. R., Ratha, B. N., & Swain, S. (2025) · Elsevier eBooks · doi:10.1016/b978-0-443-29066-4.00025-1 · Cited by 1 · see citing works
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Sharma, M., Gupta, T., Gupta, N., Rawat, R., Badoniya, N., Pandey, E., & Sharma, S. (2024) · Smart nanomaterials technology · doi:10.1007/978-981-97-0308-1_7
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van der Knaap, M. S., & Abbink, T. E. M. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-19176-3.00016-9
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Koduru, T. S., Osmani, R. A. M., & Madhuchandra, K. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-28822-7.00027-1
- Authors
- Joshi, C. R., Labhasetwar, V., & Ghorpade, A.
- Affiliations
- University of North Texas Health Science Center (US)
- Cleveland Clinic (US)
- Corresponding author
- Ghorpade, Anuja
- Venue
- Journal of Neuroimmune Pharmacology
- Published
- 2017-02-03
- Volume
- 12
- Issue
- 1
- Pages
- 51-83
- ISSN
- 1557-1890 (ISSN-L)
- 1557-1904
- PMC
- PMC5393046
- OpenAlex
- W2585557706
- Cited by
- 55
- References
- 259
- Citations by year
- 2025: 4 · 2024: 8 · 2023: 4 · 2022: 2 · 2021: 5 · 2020: 10 · 2019: 5 · 2018: 10 · 2017: 7
- FWCI
- 1.13
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- Locations
- Journal of Neuroimmune Pharmacology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute of Neurological Disorders and Stroke (NS048837, NS070896)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- gene therapy, gene delivery to the brain, central nervous system, neurological disorders, blood-brain barrier, viral vectors, nanoparticles, RNA interference, genome editing, cell-specific promoters, clinical trials, translational medicine, neuropathogenesis
- MeSH terms
- Animals, Blood-Brain Barrier, Brain, Forecasting, Humans, Genetic Therapy, Gene Transfer Techniques, Neurodegenerative Diseases
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