Cited by 32Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

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Intramuscular Delivery of Gene Therapy for Targeting the Nervous System

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Tosolini, Andrew P., Sleigh, James N.·2020·Frontiers in Molecular Neuroscience

DOI 10.3389/fnmol.2020.00129PMC PMC7379875

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Abstract

Virus-mediated gene therapy has the potential to deliver exogenous genetic material into specific cell types to promote survival and counteract disease. This is particularly enticing for neuronal conditions, as the nervous system is renowned for its intransigence to therapeutic targeting. Administration of gene therapy viruses into skeletal muscle, where distal terminals of motor and sensory neurons reside, has been shown to result in extensive transduction of cells within the spinal cord, brainstem, and sensory ganglia. This route is minimally invasive and therefore clinically relevant for gene therapy targeting to peripheral nerve soma. For successful transgene expression, viruses administered into muscle must undergo a series of processes, including host cell interaction and internalization, intracellular sorting, long-range retrograde axonal transport, endosomal liberation, and nuclear import. In this review article, we outline key characteristics of major gene therapy viruses-adenovirus, adeno-associated virus (AAV), and lentivirus-and summarize the mechanisms regulating important steps in the virus journey from binding at peripheral nerve terminals to nuclear delivery. Additionally, we describe how neuropathology can negatively influence these pathways, and conclude by discussing opportunities to optimize the intramuscular administration route to maximize gene delivery and thus therapeutic potential.

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Isolation of a Common Receptor for Coxsackie B Viruses and Adenoviruses 2 and 5
Bergelson, J. M., Cunningham, J. A., Droguett, G., Kurt‐Jones, E. A., Krithivas, A., Hong, J. S., Horwitz, M. S., Crowell, R. L., & Finberg, R. W. (1997) · Science · doi:10.1126/science.275.5304.1320 · Cited by 3,102
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381
Integrins αvβ3 and αvβ5 promote adenovirus internalization but not virus attachment
Wickham, T. J., Mathias, P., Cheresh, D. A., & Nemerow, G. R. (1993) · Cell · doi:10.1016/0092-8674(93)90231-e · Cited by 2,199
Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490
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 2×
“Due to its ability to cross the BBB, AAV serotype 9 (AAV9) has become the principal serotype for CNS-targeting upon systemic administration (Foust et al., 2009; Bevan et al., 2011; Samaranch et al., 2012), although superior serotypes, such as AAVrh10, have also emerged (Tanguy et al., 2015).”
Background
A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons
Tervo, D. G. R., Hwang, B., Viswanathan, S., Gaj, T., Lavzin, M., Ritola, K., Lindo, S. E., Michael, S. C., Кулешова, Е. П., Ojala, D. S., et al. (2016) · Neuron · doi:10.1016/j.neuron.2016.09.021 · Cited by 1,220
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
Deverman, B. E., Pravdo, P. L., Simpson, B. P., Kumar, S. R., Chan, K. Y., Banerjee, A., Wu, W.-L., Yang, B., Huber, N., Pasca, S. P., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3440 · Cited by 1,061
Clinical use of lentiviral vectors
Milone, M. C., & O’Doherty, U. (2018) · Leukemia · doi:10.1038/s41375-018-0106-0 · Cited by 930
Emerging issues in AAV-mediated in vivo gene therapy
Colella, P., Ronzitti, G., & Mingozzi, F. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2017.11.007 · Cited by 881
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Hinderer, C., Katz, N., Buza, E. L., Dyer, C., Goode, T., Bell, P., Richman, L. K., & Wilson, J. M. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.015 · Cited by 814
Engineering adeno-associated viruses for clinical gene therapy
Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761
An Adenovirus Vector for Gene Transfer into Neurons and Glia in the Brain
Salle, G. L. G. L., Robert, J. J., Berrard, S., Ridoux, V., Stratford-Perricaudet, L. D., Perricaudet, M., & Mallet, J. J. (1993) · Science · doi:10.1126/science.8382374 · Cited by 737
Adenovirus-mediated gene delivery: Potential applications for gene and cell-based therapies in the new era of personalized medicine
Lee, C. S., Bishop, E. S., Zhang, R., Yu, X., Farina, E. M., Yan, S., Zhao, C., Zeng, Z., Shu, Y., Wu, X., et al. (2017) · Genes & Diseases · doi:10.1016/j.gendis.2017.04.001 · Cited by 691
LDL receptor and its family members serve as the cellular receptors for vesicular stomatitis virus
Finkelshtein, D., Werman, A., Novick, D., Barak, S., & Rubinstein, M. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1214441110 · Cited by 656
Tissue clearing and its applications in neuroscience
Ueda, H. R., Ertürk, A., Chung, K., Gradinaru, V., Chédotal, A., Tomančák, P., & Keller, P. (2020) · Nature reviews. Neuroscience · doi:10.1038/s41583-019-0250-1 · Cited by 650
The neurotrophin receptor p75NTR: novel functions and implications for diseases of the nervous system
Dechant, G., & Barde, Y. (2002) · Nature Neuroscience · doi:10.1038/nn1102-1131 · Cited by 606
VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model
Azzouz, M., Ralph, G. S., Storkebaum, E., Walmsley, L. E., Mitrophanous, K. A., Kingsman, S. M., Carmeliet, P., & Mazarakis, N. D. (2004) · Nature · doi:10.1038/nature02544 · Cited by 588
A model system for in vivo gene transfer into the central nervous system using an adenoviral vector
Davidson, B. L., Allen, E. D., Kozarsky, K., Wilson, J. M., & Roessler, B. J. (1993) · Nature Genetics · doi:10.1038/ng0393-219 · Cited by 575
Gene Therapy
High, K. A., & Roncarolo, M. G. (2019) · New England Journal of Medicine · doi:10.1056/nejmra1706910 · Cited by 555
Altering the Tropism of Lentiviral Vectors through Pseudotyping
Cronin, J., Zhang, X.-Y., & Reiser, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054546224 · Cited by 540
Transfer of a foreign gene into the brain using adenovirus vectors
Akli, S., Caillaud, C., Vigne, E., Stratford-Perricaudet, L. D., Poënaru, L., Perricaudet, M., Kahn, A., & Peschanski, M. (1993) · Nature Genetics · doi:10.1038/ng0393-224 · Cited by 532
An essential receptor for adeno-associated virus infection
Pillay, S., Meyer, N. L., Puschnik, A. S., Davulcu, O., Diep, J., Ishikawa, Y., Jae, L. T., Wosen, J. E., Nagamine, C. M., Chapman, M. S., et al. (2016) · Nature · doi:10.1038/nature16465 · Cited by 532
Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model
Ralph, G. S., Radcliffe, P. A., Day, D. M., Carthy, J. M., Leroux, M. A., Lee, D. C. P., Wong, L., Bilsland, L. G., Greensmith, L., Kingsman, S. M., et al. (2005) · Nature Medicine · doi:10.1038/nm1205 · Cited by 507
Adenovirus triggers macropinocytosis and endosomal leakage together with its clathrin-mediated uptake
Meier, O. C., Boucke, K., Hammer, S. V., Keller, S., Stidwill, R. P., Hemmi, S., & Greber, U. F. (2002) · The Journal of Cell Biology · doi:10.1083/jcb.200112067 · Cited by 470
Microtubule-dependent Plus- and Minus End–directed Motilities Are Competing Processes for Nuclear Targeting of Adenovirus
Suomalainen, M., Nakano, M. Y., Keller, S. S., Boucke, K., Stidwill, R. P., & Greber, U. F. (1999) · The Journal of Cell Biology · doi:10.1083/jcb.144.4.657 · Cited by 468
Rab5 and Rab7 Control Endocytic Sorting along the Axonal Retrograde Transport Pathway
Deinhardt, K., Salinas, S., Verastegui, C., Watson, R., Worth, D. C., Hanrahan, S. J., Bucci, C., & Schiavo, G. (2006) · Neuron · doi:10.1016/j.neuron.2006.08.018 · Cited by 466
Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS
Raoul, C., Abbas‐Terki, T., Bensadoun, J., Guillot, S., Haase, G., Szulc, J., Henderson, C. E., & Aebischer, P. (2005) · Nature Medicine · doi:10.1038/nm1207 · Cited by 464
Deficits in axonal transport precede ALS symptoms in vivo
Bilsland, L. G., Sahai, E. A., Kelly, G. P., Golding, M., Greensmith, L., & Schiavo, G. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1006869107 · Cited by 436
Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery
Mazarakis, N. D. (2001) · Human Molecular Genetics · doi:10.1093/hmg/10.19.2109 · Cited by 425
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Hudry, E., & Vandenberghe, L. H. (2019) · Neuron · doi:10.1016/j.neuron.2019.02.017 · Cited by 383
The Neural Cell Adhesion Molecule Is a Receptor for Rabies Virus
Thoulouze, M., Lafage, M., Schachner, M., Hartmann, U., Cremer, H., & Lafon, M. (1998) · Journal of Virology · doi:10.1128/jvi.72.9.7181-7190.1998 · Cited by 379
Neuromuscular Junction Formation, Aging, and Disorders
Li, L., Xiong, W., & Mei, L. (2017) · Annual Review of Physiology · doi:10.1146/annurev-physiol-022516-034255 · Cited by 372
Recent Advances in Lentiviral Vector Development and Applications
Mátrai, J., Chuah, M., & VandenDriessche, T. (2010) · Molecular Therapy · doi:10.1038/mt.2009.319 · Cited by 365
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
Ronzitti, G., Gross, D.-A., & Mingozzi, F. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.00670 · Cited by 357
In vivo tissue-tropism of adeno-associated viral vectors
Srivastava, A. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.08.003 · Cited by 346
Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
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
Import of adenovirus DNA involves the nuclear pore complex receptor CAN/Nup214 and histone H1
Trotman, L. C., Mosberger, N., Fornerod, M. W. J., Stidwill, R. P., & Greber, U. F. (2001) · Nature Cell Biology · doi:10.1038/ncb1201-1092 · Cited by 332
Human adenovirus: Viral pathogen with increasing importance
Ghebremedhin, B. (2014) · European Journal of Microbiology and Immunology · doi:10.1556/eujmi.4.2014.1.2 · Cited by 329
ALS as a distal axonopathy: molecular mechanisms affecting neuromuscular junction stability in the presymptomatic stages of the disease
Moloney, E. B., de Winter, F., & Verhaagen, J. (2014) · Frontiers in Neuroscience · doi:10.3389/fnins.2014.00252 · Cited by 319
Axonal transport and neurological disease
Sleigh, J. N., Rossor, A. M., Fellows, A. D., Tosolini, A. P., & Schiavo, G. (2019) · Nature Reviews Neurology · doi:10.1038/s41582-019-0257-2 · Cited by 318
Self-citation
Low‐affinity nerve‐growth factor receptor (P75NTR) can serve as a receptor for rabies virus
Tuffereau, C., Bénéjean, J., Blondel, D., Kieffer, B. L., & Flamand, A. (1998) · The EMBO Journal · doi:10.1093/emboj/17.24.7250 · Cited by 309
Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306
Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
Samaranch, L., Salegio, E. A., San Sebastian, W., Kells, A. P., Foust, K. D., Bringas, J. R., Lamarre, C., Forsayeth, J., Kaspar, B. K., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.200 · Cited by 299
Adeno-Associated Virus Type 2 Capsids with Externalized VP1/VP2 Trafficking Domains Are Generated prior to Passage through the Cytoplasm and Are Maintained until Uncoating Occurs in the Nucleus
Sonntag, F., Bleker, S., Leuchs, B., Fischer, R., & Kleinschmidt, J. A. (2006) · Journal of Virology · doi:10.1128/jvi.01056-06 · Cited by 297
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291
Endocytosis and Nuclear Trafficking of Adeno-Associated Virus Type 2 Are Controlled by Rac1 and Phosphatidylinositol-3 Kinase Activation
Şanlıoğlu, S., Benson, P. K., Yang, J., Atkinson, E. M., Reynolds, T. C., & Engelhardt, J. F. (2000) · Journal of Virology · doi:10.1128/jvi.74.19.9184-9196.2000 · Cited by 272
Intracellular transport of recombinant adeno-associated virus vectors
Nonnenmacher, M., & Weber, T. (2012) · Gene Therapy · doi:10.1038/gt.2012.6 · Cited by 258
Dynein- and microtubule-mediated translocation of adenovirus serotype 5 occurs after endosomal lysis
Leopold, P. L., Kreitzer, G., Miyazawa, N., Rempel, S., Pfister, K. K., Rodriguez-Boulan, E., & Crystal, R. G. (2000) · Human Gene Therapy · doi:10.1089/10430340050016238 · Cited by 254
Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo
Soudais, C., Laplace‐Builhé, C., Kissa, K., & Kremer, E. J. (2001) · The FASEB Journal · doi:10.1096/fj.01-0321fje · Cited by 250
Retroviral DNA Integration
Lesbats, P., Engelman, A., & Cherepanov, P. (2016) · Chemical Reviews · doi:10.1021/acs.chemrev.6b00125 · Cited by 250
Erratum: Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248
Adenovirus interaction with distinct integrins mediates separate events in cell entry and gene delivery to hematopoietic cells
Huang, S., Kamata, T., Takada, Y., Ruggeri, Z. M., & Nemerow, G. R. (1996) · Journal of Virology · doi:10.1128/jvi.70.7.4502-4508.1996 · Cited by 238
Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids
Huang, Q., Chan, K. Y., Tobey, I. G., Chan, Y. A., Poterba, T., Boutros, C. L., Balazs, A. B., Daneman, R., Bloom, J. M., Seed, C., et al. (2019) · PLOS ONE · doi:10.1371/journal.pone.0225206 · Cited by 234
Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression
Zhong, L., Li, B., Jayandharan, G., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Herzog, R. W., Weigel-Van Aken, K. A., Hobbs, J. A., Zolotukhin, S., et al. (2008) · Virology · doi:10.1016/j.virol.2008.08.027 · Cited by 232
Fluorescent Virions: Dynamic Tracking of the Pathway of Adenoviral Gene Transfer Vectors in Living Cells
Leopold, P. L., Ferris, B., Grinberg, I., Worgall, S., Hackett, N. R., & Crystal, R. G. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.3-367 · Cited by 231
Characterization of Adeno-Associated Virus Genomes Isolated from Human Tissues
Schnepp, B. C., Jensen, R. L., Chen, C.-L., Johnson, P. R., & Clark, K. R. (2005) · Journal of Virology · doi:10.1128/jvi.79.23.14793-14803.2005 · Cited by 231
TDP-43 and FUS: a nuclear affair
Dormann, D., & Haass, C. (2011) · Trends in Neurosciences · doi:10.1016/j.tins.2011.05.002 · Cited by 222
The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier
Hordeaux, J., Yuan, Y., Clark, P. M., Wang, Q., Martino, R. A., Sims, J. J., Bell, P., Raymond, A., Stanford, W. L., & Wilson, J. M. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.013 · Cited by 217
Capsid Serotype and Timing of Injection Determines AAV Transduction in the Neonatal Mice Brain
Chakrabarty, P., Rosario, A. M., Cruz, P. E., Siemienski, Z. B., Ceballos‐Diaz, C., Crosby, K. W., Jansen, K. R., Borchelt, D., Kim, J., Jankowsky, J. L., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0067680 · Cited by 212
Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models
Chandler, R. J., Sands, M. S., & Venditti, C. P. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.009 · Cited by 211
Adeno-Associated Virus 2 Infection Requires Endocytosis through the CLIC/GEEC Pathway
Nonnenmacher, M. E., & Weber, T. (2011) · Cell Host & Microbe · doi:10.1016/j.chom.2011.10.014 · Cited by 209
Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy
Azzouz, M., Le, T. T., Ralph, G. S., Walmsley, L. E., Monani, U. R., Lee, D. C. P., Wilkes, F. J., Mitrophanous, K. A., Kingsman, S. M., Burghes, A. H. M., et al. (2004) · Journal of Clinical Investigation · doi:10.1172/jci22922 · Cited by 209
A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity
Acsádi, G., Jáni, Á., Bernard Massie, B., Simoneau, M., Holland, P. C., Blaschuk, K., & Karpati, G. (1994) · Human Molecular Genetics · doi:10.1093/hmg/3.4.579 · Cited by 207
Adenovirus receptors: implications for targeting of viral vectors
Arnberg, N. (2012) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2012.04.005 · Cited by 200
Neuromuscular synaptogenesis: coordinating partners with multiple functions
Darabid, H., Pérez-González, A., & Robitaille, R. (2014) · Nature reviews. Neuroscience · doi:10.1038/nrn3821 · Cited by 199
AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method
Klein, R. L., Dayton, R. D., Tatom, J. B., Henderson, K. M., & Henning, P. P. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300331 · Cited by 196
Ligand-Induced Internalization of the p75 Neurotrophin Receptor: A Slow Route to the Signaling Endosome
Bronfman, F. C., Tcherpakov, M., Jovin, T. M., & Fainzilber, M. (2003) · Journal of Neuroscience · doi:10.1523/jneurosci.23-08-03209.2003 · Cited by 190
Adeno-associated Virus (AAV) Serotypes Have Distinctive Interactions with Domains of the Cellular AAV Receptor
Pillay, S., Zou, W., Cheng, F., Puschnik, A. S., Meyer, N., Ganaie, S. S., Deng, X., Wosen, J. E., Davulcu, O., Yan, Z., et al. (2017) · Journal of Virology · doi:10.1128/jvi.00391-17 · Cited by 190
Adenoviral Vector Immunity: Its Implications and Circumvention Strategies
S. Ahi, Y., S. Bangari, D., & K. Mittal, S. (2011) · Current Gene Therapy · doi:10.2174/156652311796150372 · Cited by 185
Increased Survival and Function of SOD1 Mice After Glial Cell-Derived Neurotrophic Factor Gene Therapy
Acsádi, G., Anguelov, R. A., Yang, H., Tóth, G., Thomas, R., Jáni, Á., Wang, Y., Ianakova, E., Mohammad, S., Lewis, R. A., et al. (2002) · Human Gene Therapy · doi:10.1089/104303402753812458 · Cited by 180
Cytoplasmic Trafficking, Endosomal Escape, and Perinuclear Accumulation of Adeno-Associated Virus Type 2 Particles Are Facilitated by Microtubule Network
Xiao, P.-J., & Samulski, R. J. (2012) · Journal of Virology · doi:10.1128/jvi.00935-12 · Cited by 175
Adenovirus Transport via Direct Interaction of Cytoplasmic Dynein with the Viral Capsid Hexon Subunit
Bremner, K. H., Scherer, J., Yi, J., Vershinin, M. D., Gross, S. P., & Vallee, R. B. (2009) · Cell Host & Microbe · doi:10.1016/j.chom.2009.11.006 · Cited by 175
Adenovirus Entry: From Infection to Immunity
Greber, U. F., & Flatt, J. W. (2019) · Annual Review of Virology · doi:10.1146/annurev-virology-092818-015550 · Cited by 175
Low-pH conformational changes of rabies virus glycoprotein and their role in membrane fusion
Gaudin, Y., Ruigrok, R. W. H., Knossow, M., & Flamand, A. (1993) · Journal of Virology · doi:10.1128/jvi.67.3.1365-1372.1993 · Cited by 172
Expression of the Primary Coxsackie and Adenovirus Receptor Is Downregulated during Skeletal Muscle Maturation and Limits the Efficacy of Adenovirus-Mediated Gene Delivery to Muscle Cells
Nalbantoglu, J. N., Pari, G., Karpati, G., & Holland, P. C. (1999) · Human Gene Therapy · doi:10.1089/10430349950018409 · Cited by 169
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 166
Adeno-Associated Virus Serotypes 1, 8, and 9 Share Conserved Mechanisms for Anterograde and Retrograde Axonal Transport
Castle, M. J., Gershenson, Z. T., Giles, A. R., Holzbaur, E. L. F., & Wolfe, J. H. N. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.189 · Cited by 165
The p75 neurotrophin receptor: multiple interactors and numerous functions
Gentry, J. J., Barker, P. A., & Carter, B. D. (2004) · Progress in brain research · doi:10.1016/s0079-6123(03)46002-0 · Cited by 163
Targeting lentiviral vectors to specific cell types in vivo
Yang, L., Bailey, L., Baltimore, D., & Wang, P. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0604993103 · Cited by 161
Viral and Cellular Requirements for the Nuclear Entry of Retroviral Preintegration Nucleoprotein Complexes
Matreyek, K. A., & Engelman, A. (2013) · Viruses · doi:10.3390/v5102483 · Cited by 160
Axonal transport of adeno-associated viral vectors is serotype-dependent
Salegio, E. A., Samaranch, L., Kells, A. P., Mittermeyer, G., San Sebastian, W., Zhou, S., Beyer, J., Forsayeth, J., & Bankiewicz, K. S. (2013) · Gene Therapy · doi:10.1038/gt.2012.27 · Cited by 156
High-Capacity Adenoviral Vectors: Expanding the Scope of Gene Therapy
Ricobaraza, A., González-Aparicio, M., Mora-Jimenez, L., Lumbreras, S., & Hernández-Alcoceba, R. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21103643 · Cited by 155
Cytoplasmic Dynein Mediates Adenovirus Binding to Microtubules
Kelkar, S., Pfister, K. K., Crystal, R. G., & Leopold, P. L. (2004) · Journal of Virology · doi:10.1128/jvi.78.18.10122-10132.2004 · Cited by 153
Cytoplasmic dynein heavy chain: the servant of many masters
Schiavo, G., Greensmith, L., Hafezparast, M., & Fisher, E. (2013) · Trends in Neurosciences · doi:10.1016/j.tins.2013.08.001 · Cited by 146
Transduction Patterns of Pseudotyped Lentiviral Vectors in the Nervous System
Wong, L., Azzouz, M., Walmsley, L. E., Askham, Z., Wilkes, F. J., Mitrophanous, K. A., Kingsman, S. M., & Mazarakis, N. D. (2003) · Molecular Therapy · doi:10.1016/j.ymthe.2003.09.017 · Cited by 145
Parvovirus glycan interactions
Huang, L., Halder, S., & Agbandje‐McKenna, M. (2014) · Current Opinion in Virology · doi:10.1016/j.coviro.2014.05.007 · Cited by 143
Role of extracellular matrix proteins and their receptors in the development of the vertebrate neuromuscular junction
Singhal, N., & Martin, P. T. (2011) · Developmental Neurobiology · doi:10.1002/dneu.20953 · Cited by 141
Coxsackievirus-adenovirus receptor (CAR) is essential for early embryonic cardiac development
Dorner, A., Wegmann, F., Butz, S., Wolburg-Buchholz, K., Wolburg, H., Mack, A., Nasdala, I., August, B., Westermann, J., Rathjen, F., et al. (2005) · Journal of Cell Science · doi:10.1242/jcs.02476 · Cited by 137
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
Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice
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
Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1
Hollis, E. R., II, Kadoya, K., Hirsch, M. L., Samulski, R. J., & Tuszynski, M. H. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300367 · Cited by 126
Recombinant Adeno-Associated Virus Utilizes Host Cell Nuclear Import Machinery To Enter the Nucleus
Nicolson, S. C., & Samulski, R. J. (2014) · Journal of Virology · doi:10.1128/jvi.02660-13 · Cited by 126
Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6
Towne, C., Schneider, B. L., Kieran, D., Redmond, D. E., & Aebischer, P. (2010) · Gene Therapy · doi:10.1038/gt.2009.119 · Cited by 124
CNS gene delivery by retrograde transport of recombinant replication-defective adenoviruses.
Ghadge, G. D., Roos, R. P., Kang, U. J., Wollmann, R. L., Fishman, P. S., Kalynych, A. M., Barr, E., & Leiden, J. M. (1995) · PubMed · Cited by 124
Adeno-associated virus: fit to serve
Zinn, E. M., & Vandenberghe, L. H. (2014) · Current Opinion in Virology · doi:10.1016/j.coviro.2014.07.008 · Cited by 123
Dynamic neuromuscular remodeling precedes motor-unit loss in a mouse model of ALS
Martineau, É., Polo, A. D., Velde, C. V., & Robitaille, R. (2018) · eLife · doi:10.7554/elife.41973 · Cited by 123
Lentiviral vectors as tools to understand central nervous system biology in mammalian model organisms
Parr‐Brownlie, L. C., Bosch‐Bouju, C., Schoderboeck, L., Sizemore, R. J., Abraham, W. C., & Hughes, S. M. (2015) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2015.00014 · Cited by 121
CAR-Associated Vesicular Transport of an Adenovirus in Motor Neuron Axons
Salinas, S., Bilsland, L. G., Henaff, D., Weston, A., Keriel, A., Schiavo, G., & Kremer, E. J. (2009) · PLoS Pathogens · doi:10.1371/journal.ppat.1000442 · Cited by 120
A potential role for the p75 low-affinity neurotrophin receptor in spinal motor neuron degeneration in murine and human amyotrophic lateral sclerosis.
Lowry, K., Murray, S. S., McLean, C., Talman, P. S., Mathers, S., Lopes, E., & Cheema, S. (2001) · Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders · doi:10.1080/146608201753275463 · Cited by 120
Targeted Transduction of CNS Neurons with Adenoviral Vectors Carrying Neurotrophic Factor Genes Confers Neuroprotection That Exceeds the Transduced Population
Baumgartner, B. J., & Shine, H. D. (1997) · Journal of Neuroscience · doi:10.1523/jneurosci.17-17-06504.1997 · Cited by 119
In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy
Choudhury, S., Fitzpatrick, Z., Harris, A. F., Maitland, S. A., Ferreira, J. S., Zhang, Y., Ma, S., Sharma, R. B., Gray‐Edwards, H. L., Johnson, J. A., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.84 · Cited by 118
Long‐term in vivo transduction of neurons throughout the rat central nervous system using novel helper‐dependent CAV‐2 vectors
Soudais, C., Skander, N., & Kremer, E. J. (2003) · The FASEB Journal · doi:10.1096/fj.03-0438fje · Cited by 117
Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1 G93A Mice and Nonhuman Primates
Borel, F., Gernoux, G., Cardozo, B. H., Metterville, J., Cabrera, G. T., Song, L., Su, Q., Gao, G. P., ElMallah, M. K., Brown, R. H., et al. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.122 · Cited by 116
An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor
Dudek, A. M., Pillay, S., Puschnik, A. S., Nagamine, C. M., Cheng, F., Qiu, J., Carette, J. E., & Vandenberghe, L. H. (2018) · Journal of Virology · doi:10.1128/jvi.02213-17 · Cited by 116
Rabies Virus-Induced Membrane Fusion Pathway
Gaudin, Y. (2000) · The Journal of Cell Biology · doi:10.1083/jcb.150.3.601 · Cited by 115
Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice
Tanguy, Y., Biferi, M. G., Besse, A., Astord, S., Cohen-Tannoudji, M., Marais, T., & Barkats, M. (2015) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2015.00036 · Cited by 103
Long-distance Axonal Transport of AAV9 Is Driven by Dynein and Kinesin-2 and Is Trafficked in a Highly Motile Rab7-positive Compartment
Castle, M. J., Perlson, E., Holzbaur, E. L. F., & Wolfe, J. H. N. (2013) · Molecular Therapy · doi:10.1038/mt.2013.237 · Cited by 98
Stuck in traffic: an emerging theme in diseases of the nervous system
Neefjes, J., & van der Kant, R. (2014) · Trends in Neurosciences · doi:10.1016/j.tins.2013.11.006 · Cited by 98
Retrograde optogenetic characterization of the pontospinal module of the locus coeruleus with a canine adenoviral vector
Li, Y., Hickey, L., Perrins, R., Werlen, E., Patel, A. A., Hirschberg, S., Jones, M. W., Salinas, S., Kremer, E. J., & Pickering, A. E. (2016) · Brain Research · doi:10.1016/j.brainres.2016.02.023 · Cited by 97
Syntaxin 5-Dependent Retrograde Transport to the trans -Golgi Network Is Required for Adeno-Associated Virus Transduction
Nonnenmacher, M. E., Cintrat, J., Gillet, D., & Weber, T. (2014) · Journal of Virology · doi:10.1128/jvi.02520-14 · Cited by 96
Differential neuronal vulnerability identifies IGF-2 as a protective factor in ALS
Allodi, I., Comley, L. H., Nichterwitz, S., Nizzardo, M., Simone, C., Benitez, J. A., Cao, M., Corti, S. P., & Hedlund, E. (2016) · Scientific Reports · doi:10.1038/srep25960 · Cited by 96
Modulation of immune responses in lentiviral vector-mediated gene transfer
Annoni, A., Gregori, S., Naldini, L., & Cantore, A. (2018) · Cellular Immunology · doi:10.1016/j.cellimm.2018.04.012 · Cited by 96
Tetanus Toxin Is Transported in a Novel Neuronal Compartment Characterized by a Specialized pH Regulation
Bohnert, S., & Schiavo, G. (2005) · Journal of Biological Chemistry · doi:10.1074/jbc.m506750200 · Cited by 96
Host determinants of adeno-associated viral vector entry
Pillay, S., & Carette, J. E. (2017) · Current Opinion in Virology · doi:10.1016/j.coviro.2017.06.003 · Cited by 94
Mice Carrying ALS Mutant TDP-43, but Not Mutant FUS, Display In Vivo Defects in Axonal Transport of Signaling Endosomes
Sleigh, J. N., Tosolini, A. P., Gordon, D., Devoy, A., Fratta, P., Fisher, E., Talbot, K., & Schiavo, G. (2020) · Cell Reports · doi:10.1016/j.celrep.2020.02.078 · Cited by 93
Self-citation
Cellular transduction mechanisms of adeno-associated viral vectors
Berry, G. E., & Asokan, A. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.08.001 · Cited by 90
Neuroprotection of Spinal Motoneurons Following Targeted Transduction with an Adenoviral Vector Carrying the Gene for Glial Cell Line-Derived Neurotrophic Factor
Baumgartner, B. J., & Shine, H. D. (1998) · Experimental Neurology · doi:10.1006/exnr.1998.6878 · Cited by 89
Pseudotyped Lentiviral Vectors: One Vector, Many Guises
Joglekar, A. V., & Sandoval, S. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2017.084 · Cited by 88
A Two-hybrid Screen Identifies Cathepsins B and L as Uncoating Factors for Adeno-associated Virus 2 and 8
Akache, B., Grimm, D., Shen, X., Fuess, S., Yant, S. R., Glazer, D. S., Park, J., & Kay, M. A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300053 · Cited by 87
Divergent engagements between adeno-associated viruses with their cellular receptor AAVR
Zhang, R., Xu, G., Cao, L., Sun, Z., He, Y., Cui, M., Sun, Y., Li, S., Li, H., Qin, L., et al. (2019) · Nature Communications · doi:10.1038/s41467-019-11668-x · Cited by 86
Neuromuscular junction maturation defects precede impaired lower motor neuron connectivity in Charcot-Marie-Tooth type 2D mice
Sleigh, J. N., Grice, S. J., Burgess, R. W., Talbot, K., & Cader, M. Z. (2013) · Human Molecular Genetics · doi:10.1093/hmg/ddt659 · Cited by 86
Self-citation
The travel diaries of tetanus and botulinum neurotoxins
Surana, S., Tosolini, A. P., Meyer, I., Fellows, A. D., Novoselov, S. S., & Schiavo, G. (2017) · Toxicon · doi:10.1016/j.toxicon.2017.10.008 · Cited by 82
Self-citation
Skeletal Muscle Remodelling as a Function of Disease Progression in Amyotrophic Lateral Sclerosis
Jensen, L., Jørgensen, L. H., Bech, R. D., Frandsen, U., & Schrøder, H. D. (2016) · BioMed Research International · doi:10.1155/2016/5930621 · Cited by 82
MPP+ induced apoptotic cell death in SH-SY5Y neuroblastoma cells: An electron microscope study
Sheehan, J. P., Palmer, P. E., Helm, G. A., & Tuttle, J. B. (1997) · Journal of Neuroscience Research · doi:10.1002/(sici)1097-4547(19970501)48:3 · Cited by 81
Evidence for pH-Dependent Protease Activity in the Adeno-Associated Virus Capsid
Salganik, M., Venkatakrishnan, B., Bennett, A. D., Lins, B., Yarbrough, J., Muzyczka, N., Agbandje‐McKenna, M., & McKENNA, R. V. (2012) · Journal of Virology · doi:10.1128/jvi.01717-12 · Cited by 80
Nuclear Import of Adenovirus DNA Involves Direct Interaction of Hexon with an N-Terminal Domain of the Nucleoporin Nup214
Cassany, A., Ragues, J., Guan, T., Bégu, D., Wodrich, H., Kann, M., Nemerow, G. R., & Gerace, L. (2014) · Journal of Virology · doi:10.1128/jvi.02639-14 · Cited by 80
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Hudry, E., & Vandenberghe, L. H. (2019) · Neuron · doi:10.1016/j.neuron.2019.03.020 · Cited by 77
Extracellular matrix and its receptors indrosophilaneural development
Broadie, K. S., Baumgartner, S., & Prokop, A. (2011) · Developmental Neurobiology · doi:10.1002/dneu.20935 · Cited by 77
Evidence from the anti-idiotypic network that the acetylcholine receptor is a rabies virus receptor
Hanham, C. A., Zhao, F., & Tignor, G. H. (1993) · Journal of Virology · doi:10.1128/jvi.67.1.530-542.1993 · Cited by 75
Trk receptor signaling and sensory neuron fate are perturbed in human neuropathy caused by Gars mutations
Sleigh, J. N., Dawes, J. M., West, S. J., Wei, N., Spaulding, E. L., Gómez-Martín, A., Zhang, Q. Y., Burgess, R. W., Cader, M. Z., Talbot, K., et al. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1614557114 · Cited by 75
Self-citation
Efficient Gene Transfer via Retrograde Transport in Rodent and Primate Brains Using a Human Immunodeficiency Virus Type 1-Based Vector Pseudotyped with Rabies Virus Glycoprotein
Kato, S., Inoue, K., Kobayashi, K., Yasoshima, Y., Miyachi, S., Inoue, S., Hanawa, H., Shimada, T., Takada, M., & Kobayashi, K. (2007) · Human Gene Therapy · doi:10.1089/hum.2007.082 · Cited by 74
A Viral Receptor Complementation Strategy to Overcome CAV-2 Tropism for Efficient Retrograde Targeting of Neurons
Li, S.-J., Vaughan, A., Sturgill, J. F., & Kepecs, Á. (2018) · Neuron · doi:10.1016/j.neuron.2018.05.028 · Cited by 74
Synaptic Deficits at Neuromuscular Junctions in Two Mouse Models of Charcot–Marie–Tooth Type 2d
Spaulding, E. L., Sleigh, J. N., Morelli, K. H., Pinter, M. J., Burgess, R. W., & Seburn, K. L. (2016) · Journal of Neuroscience · doi:10.1523/jneurosci.1762-15.2016 · Cited by 73
Self-citation
Differential Short-Term Transduction Efficiency of Adult versus Newborn Mouse Tissues by Adenoviral Recombinants
Huard, J., Lochmüller, H., Acsádi, G., Jáni, Á., Holland, P. C., Guérin, C., Massie, B., & Karpati, G. (1995) · Experimental and Molecular Pathology · doi:10.1006/exmp.1995.1015 · Cited by 71
Developmental distribution of coxsackie virus and adenovirus receptor localized in the nervous system
Hotta, Y., Honda, T., Naito, M., & Kuwano, R. (2003) · Developmental Brain Research · doi:10.1016/s0165-3806(03)00035-x · Cited by 69
Muscle-Specific Overexpression of the Adenovirus Primary Receptor CAR Overcomes Low Efficiency of Gene Transfer to Mature Skeletal Muscle
Nalbantoglu, J. N., Larochelle, N., Wolf, E., Karpati, G., Lochmüller, H., & Holland, P. C. (2001) · Journal of Virology · doi:10.1128/jvi.75.9.4276-4282.2001 · Cited by 69
Schwann cell targeting via intrasciatic injection of AAV8 as gene therapy strategy for peripheral nerve regeneration
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
Design of AAV Vectors for Delivery of Large or Multiple Transgenes
Patel, A., Zhao, J., Duan, D., & Lai, Y. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9139-6_2 · Cited by 67
The ubiquitous neural cell adhesion molecule (N-CAM)
Weledji, E. P., & Assob, J. C. N. (2014) · Annals of Medicine and Surgery · doi:10.1016/j.amsu.2014.06.014 · Cited by 67
Golgi fragmentation precedes neuromuscular denervation and is associated with endosome abnormalities in SOD1-ALS mouse motor neurons
van Dis, V., Kuijpers, M., Haasdijk, E. D., Teuling, E., Oakes, S. A., Hoogenraad, C. C., & Jaarsma, D. (2014) · Acta Neuropathologica Communications · doi:10.1186/2051-5960-2-38 · Cited by 66
An adenovirus vector with a chimeric fiber incorporating stabilized single chain antibody achieves targeted gene delivery
Hedley, S. J., der Maur, A. A., Hohn, S., Escher, D., Barberis, A., Glasgow, J. N., Douglas, J. T., Korokhov, N., & Curiel, D. T. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302603 · Cited by 66
Adenovirus-mediated transfer of the neurotrophin-3 gene into skeletal muscle of pmn mice: Therapeutic effects and mechanisms of action
Haase, G., Pettmann, B., Vigne, E., Castelnau, L., Schmalbruch, H., & Kahn, A. (1998) · Journal of the Neurological Sciences · doi:10.1016/s0022-510x(98)00207-x · Cited by 65
Decreased function of survival motor neuron protein impairs endocytic pathways
Dimitriadi, M., Derdowski, A., Kalloo, G., Maginnis, M. S., O’Hern, P., Bliska, B., Sorkaç, A., Nguyen, K. C. Q., Cook, S. J., Poulogiannis, G., et al. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1600015113 · Cited by 64
Specific and efficient genetransfer strategy offers new potentialities for the treatment of motor neurone diseases
Finiels, F., y Ribotta, M. G., Barkats, M., Samolyk, M.-L., Robert, J.-J., Privat, A., Revah, F., & Mallet, J. J. (1995) · Neuroreport · doi:10.1097/00001756-199512150-00009 · Cited by 64
The association of viral proteins with host cell dynein components during virus infection
Merino, J., García‐Mayoral, M. F., & Rodríguez‐Crespo, I. (2011) · FEBS Journal · doi:10.1111/j.1742-4658.2011.08252.x · Cited by 62
AAVR: A Multi-Serotype Receptor for AAV
Summerford, C., & Samulski, R. J. (2016) · Molecular Therapy · doi:10.1038/mt.2016.49 · Cited by 61
Efficient Retrograde Transport of Adeno-Associated Virus Type 8 to Spinal Cord and Dorsal Root Ganglion After Vector Delivery in Muscle
Zheng, H., Qiao, C., Wang, C., Li, J., Li, J., Yuan, Z., Zhang, C., & Xiao, X. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.131 · Cited by 56
Successful target cell transduction of capsid-engineered rAAV vectors requires clathrin-dependent endocytosis
Uhrig, S., Coutelle, O., Wiehe, T., Perabò, L., Hallek, M. J., & Büning, H. (2011) · Gene Therapy · doi:10.1038/gt.2011.78 · Cited by 56
At the “Junction” of Spinal Muscular Atrophy Pathogenesis: The Role of Neuromuscular Junction Dysfunction in SMA Disease Progression
Goulet, B., Kothary, R., & Parks, R. J. (2013) · Current Molecular Medicine · doi:10.2174/15665240113139990044 · Cited by 55
Intraneural Colchicine Inhibition of Adenoviral and Adeno-associated Viral Vector Remote Spinal Cord Gene Delivery
Boulis, N. M., Willmarth, N. E., Song, D. K., Feldman, E. L., & Imperiale, M. J. (2003) · Neurosurgery · doi:10.1227/01.neu.0000044459.24519.3e · Cited by 55
The many disguises of the signalling endosome
Villarroel‐Campos, D., Schiavo, G., & Lazo, O. M. (2018) · FEBS Letters · doi:10.1002/1873-3468.13235 · Cited by 53
CAV-2 Vector Development and Gene Transfer in the Central and Peripheral Nervous Systems
Rio, D. D., Beucher, B., Lavigne, M., Wehbi, A., Dopeso‐Reyes, I. G., Saggio, I., & Kremer, E. J. (2019) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2019.00071 · Cited by 53
Highly Efficient Retrograde Gene Transfer into Motor Neurons by a Lentiviral Vector Pseudotyped with Fusion Glycoprotein
Hirano, M., Kato, S., Kobayashi, K., Okada, T., Yaginuma, H., & Kobayashi, K. (2013) · PLoS ONE · doi:10.1371/journal.pone.0075896 · Cited by 53
Isoform-specific expression of the Coxsackie and adenovirus receptor (CAR) in neuromuscular junction and cardiac intercalated discs
Shaw, C. A., Holland, P. C., Sinnreich, M., Allen, C. A., Sollerbrant, K., Karpati, G., & Nalbantoglu, J. N. (2004) · BMC Cell Biology · doi:10.1186/1471-2121-5-42 · Cited by 53
Manipulation of Adenovirus Interactions with Host Factors for Gene Therapy Applications
Duffy, M. R., Parker, A. L., Bradshaw, A. C., & Baker, A. H. (2012) · Nanomedicine · doi:10.2217/nnm.11.186 · Cited by 51
Transduction of motor neurons and muscle fibers by intramuscular injection of HIV-1-based vectors pseudotyped with select rabies virus glycoproteins
Mentis, G. Z., Gravell, M., Hamilton, R. S., Shneider, N. A., O'Donovan, M. J., & Schubert, M. (2006) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2006.04.011 · Cited by 50
Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis
Tosolini, A. P., & Sleigh, J. N. (2017) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2017.00405 · Cited by 48
Self-citation
Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease
Chen, Y. H., Claflin, K. E., Geoghegan, J. C., & Davidson, B. L. (2012) · Molecular Therapy · doi:10.1038/mt.2012.100 · Cited by 46
In Vivo Imaging of Anterograde and Retrograde Axonal Transport in Rodent Peripheral Nerves
Sleigh, J. N., Tosolini, A. P., & Schiavo, G. (2020) · Methods in molecular biology · doi:10.1007/978-1-0716-0585-1_20 · Cited by 45
Self-citation
Disruption of the Coxsackievirus and Adenovirus Receptor-Homodimeric Interaction Triggers Lipid Microdomain- and Dynamin-dependent Endocytosis and Lysosomal Targeting
Salinas, S., Zussy, C., Loustalot, F., Henaff, D., Menéndez, G., Morton, P. E., Parsons, M., Schiavo, G., & Kremer, E. J. (2013) · Journal of Biological Chemistry · doi:10.1074/jbc.m113.518365 · Cited by 44
Presynaptic NCAM Is Required for Motor Neurons to Functionally Expand Their Peripheral Field of Innervation in Partially Denervated Muscles
Chipman, P., Schachner, M., & Rafuse, V. F. (2014) · Journal of Neuroscience · doi:10.1523/jneurosci.0697-14.2014 · Cited by 44
Recombinant Adeno-Associated Virus Utilizes Cell-Specific Infectious Entry Mechanisms
Weinberg, M. S., Nicolson, S. C., Bhatt, A. P., McLendon, M., Li, C., & Samulski, R. J. (2014) · Journal of Virology · doi:10.1128/jvi.01971-14 · Cited by 43
Adenovirus Tales: From the Cell Surface to the Nuclear Pore Complex
Kremer, E. J., & Nemerow, G. R. (2015) · PLoS Pathogens · doi:10.1371/journal.ppat.1004821 · Cited by 43
FGF binding proteins (FGFBPs): Modulators of FGF signaling in the developing, adult, and stressed nervous system
Taetzsch, T., Brayman, V. L., & Valdez, G. (2018) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2018.06.009 · Cited by 42
Morphological variability is greater at developing than mature mouse neuromuscular junctions
Mech, A. M., Brown, A., Schiavo, G., & Sleigh, J. N. (2020) · Journal of Anatomy · doi:10.1111/joa.13228 · Cited by 42
Self-citation
rAAV2-Retro Enables Extensive and High-Efficient Transduction of Lower Motor Neurons following Intramuscular Injection
Chen, Z., Fan, G., Li, A., Yuan, J., & Xu, T. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.11.006 · Cited by 41
Rabies Virus Envelope Glycoprotein Targets Lentiviral Vectors to the Axonal Retrograde Pathway in Motor Neurons
Hislop, J. N., Islam, T. A., Eleftheriadou, I., Carpentier, D. C. J., Trabalza, A., Parkinson, M. D., Schiavo, G., & Mazarakis, N. D. (2014) · Journal of Biological Chemistry · doi:10.1074/jbc.m114.549980 · Cited by 39
Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction
Mano, M., Ippodrino, R., Zentilin, L., Zacchigna, S., & Giacca, M. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1503607112 · Cited by 39
Role of kinesins in directed adenovirus transport and cytoplasmic exploration
Zhou, J., Scherer, J., Yi, J., & Vallee, R. B. (2018) · PLoS Pathogens · doi:10.1371/journal.ppat.1007055 · Cited by 39
Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV
Nault, J., Mami, I., Bella, T. L., Datta, S., Imbeaud, S., Franconi, A., Mallet, M., Couchy, G., Letouzé, É., Pilati, C., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.47 · Cited by 38
Engineering AAV receptor footprints for gene therapy
Madigan, V. J., & Asokan, A. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.05.001 · Cited by 37
Coxsackievirus Adenovirus Receptor Loss Impairs Adult Neurogenesis, Synapse Content, and Hippocampus Plasticity
Zussy, C., Loustalot, F., Junyent, F., Gardoni, F., Bories, C., Valero, J., Desarménien, M. G., Bernex, F., Henaff, D., Bayó-Puxan, N., et al. (2016) · Journal of Neuroscience · doi:10.1523/jneurosci.0132-16.2016 · Cited by 37
Interaction of the Coxsackie and adenovirus receptor (CAR) with the cytoskeleton: Binding to actin
Huang, K.-C., Yasruel, Z., Guérin, C., Holland, P. C., & Nalbantoglu, J. N. (2007) · FEBS Letters · doi:10.1016/j.febslet.2007.05.019 · Cited by 37
Dyslexia-Associated Kiaa0319-Like Protein Interacts with Axon Guidance Receptor Nogo Receptor 1
Poon, M.-W., Tsang, W.-H., Chan, S. O., Li, H., Ng, H., & Waye, M. M. Y. (2010) · Cellular and Molecular Neurobiology · doi:10.1007/s10571-010-9549-1 · Cited by 36
DRG-targeted helper-dependent adenoviruses mediate selective gene delivery for therapeutic rescue of sensory neuronopathies in mice
Terashima, T., Oka, K., Kritz, A., Kojima, H., Baker, A. H., & Chan, L. (2009) · Journal of Clinical Investigation · doi:10.1172/jci39038 · Cited by 36
Quantitative 3D Tracing of Gene-delivery Viral Vectors in Human Cells and Animal Tissues
Xiao, P.-J., Li, C., Neumann, A., & Samulski, R. J. (2012) · Molecular Therapy · doi:10.1038/mt.2011.250 · Cited by 33
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Scherer, J., Yi, J., & Vallee, R. B. (2020) · FEBS Letters · doi:10.1002/1873-3468.13777 · Cited by 32
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Millecamps, S., Nicolle, D. M., Ceballos-Picot, I., Mallet, J. J., & Barkats, M. (2001) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.131031098 · Cited by 30
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He, T., Itano, M. S., Earley, L. F., Hall, N. E., Riddick, N. V., Samulski, R. J., & Li, C. (2019) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2019.030 · Cited by 26
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Loustalot, F., Kremer, E. J., & Salinas, S. (2015) · International review of cytology · doi:10.1016/bs.ircmb.2015.10.006 · Cited by 25
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Pérez, V., Bermedo‐García, F., Zelada, D., Court, F. A., Pérez, M. Á., Fuenzalida, M., Ábrigo, J., Cabello‐Verrugio, C., Moya‐Alvarado, G., Tapia, J. C., et al. (2019) · Acta Neuropathologica Communications · doi:10.1186/s40478-019-0802-7 · Cited by 25
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Finiels, F., y Ribotta, M. G., Barkats, M., Samolyk, M.-L., Robert, J.-J., Privat, A., Revah, F., & Mallet, J. J. (1995) · Neuroreport · doi:10.1097/00001756-199512290-00088 · Cited by 23
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Gostić, M., Martinelli, A. M., Tucker, C. S., Yang, Z., Gasparoli, F. M., Ewart, J., Dholakia, K., Sillar, K. T., Tello, J. A., & Paracchini, S. (2019) · The Journal of Comparative Neurology · doi:10.1002/cne.24696 · Cited by 17
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Sinnreich, M., Shaw, C. A., Pari, G., Nalbantoglu, J. N., Holland, P. C., & Karpati, G. (2005) · Neuromuscular Disorders · doi:10.1016/j.nmd.2005.05.007 · Cited by 16
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Morris, R., & Tosolini, A. P. (2016) · Neural Regeneration Research · doi:10.4103/1673-5374.182698 · Cited by 14
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Han, S., Li, D., Kou, Y., Fu, Z.-G., & Yin, X. (2019) · Artificial Cells Nanomedicine and Biotechnology · doi:10.1080/21691401.2019.1687493 · Cited by 13
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Eleftheriadou, I., Manolaras, I., Irvine, E. E., Dieringer, M., Trabalza, A., & Mazarakis, N. D. (2016) · Annals of Clinical and Translational Neurology · doi:10.1002/acn3.335 · Cited by 12
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Kim, S., Lee, S., Lee, H., Cho, M., Schaffer, D. V., & Jang, J. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.09.015 · Cited by 12
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Gene Therapy
Worgall, S., & Crystal, R. G. (2013) · Elsevier eBooks · doi:10.1016/b978-0-12-398358-9.00034-3 · Cited by 12

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Drug delivery to the central nervous system
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Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic/pharmacodynamic (PK/PD) models
Chowdhury, E. A., Meno‐Tetang, G. M. L., Chang, H. Y., Wu, S., Huang, H. W., Jamier, T., Chandran, J. S., & Shah, D. K. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.01.017 · Cited by 80
Micro/nanosystems for controllable drug delivery to the brain
Tian, M., Ma, Z., & Yang, G. (2023) · The Innovation · doi:10.1016/j.xinn.2023.100548 · Cited by 64
Nanocarrier imaging at single-cell resolution across entire mouse bodies with deep learning
Luo, J., Molbay, M., Chen, Y., Horvath, I., Kadletz, K., Kick, B., Zhao, S., Al-Maskari, R., Singh, I., Ali, M., et al. (2025) · Nature Biotechnology · doi:10.1038/s41587-024-02528-1 · Cited by 53
Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside
Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48

See all 32

Authors: Tosolini, A. P., & Sleigh, J. N.
Affiliations: UCL Queen Square Institute of Neurology (GB)
UK Dementia Research Institute (GB)
Corresponding author: Tosolini, Andrew P.
Sleigh, James N.
ORCID: Tosolini, Andrew P.
Sleigh, James N.
Venue: Frontiers in Molecular Neuroscience
Publisher: Frontiers Media SA
Published: 2020-07-17
Volume: 13
Pages: 129-129
ISSN: 1662-5099 (ISSN-L)
DOI: 10.3389/fnmol.2020.00129
PMC: PMC7379875
OpenAlex: W3043715250
Cited by: 32
References: 205
Citations by year: 2026: 4 · 2025: 4 · 2024: 5 · 2023: 8 · 2022: 5 · 2021: 6
FWCI: 2.79
Type: journal article
Language: en
Open access: gold
doi.org
Licence: cc-by
creativecommons.org/licenses/by/4.0/ (vor)
Locations: Frontiers in Molecular Neuroscience (publishedVersion, cc-by)
PubMed Central (publishedVersion, cc-by)
PubMed (publishedVersion)
DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
UCL Discovery (University College London) (submittedVersion)
Funders: Wellcome Trust (107116/Z/15/Z)
Medical Research Council (MR/S006990/1)
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
Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Keywords: intramuscular gene delivery, gene therapy vectors, retrograde axonal transport, peripheral nerve terminals, motor neurons, sensory neurons, adeno-associated virus, adenovirus, lentivirus, viral transduction, endosomal escape, nuclear import, neuropathology

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Intramuscular Delivery of Gene Therapy for Targeting the Nervous System

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