AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method

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Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

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AAV9-mediated erythropoietin gene delivery into the brain protects nigral dopaminergic neurons in a rat model of Parkinson's disease

Xue, Y.-Q., Ma, B.-F., Zhao, L.-R., Tatom, J. B., Li, B., Jiang, L.-X., Klein, R. L., & Duan, W.-M. (2009) · Gene Therapy · doi:10.1038/gt.2009.113 · Cited by 70 · see citing works

Corrigendum: Better Targeting, Better Efficiency for Wide-Scale Neuronal Transduction with the Synapsin Promoter and AAV-PHP.B

Jackson, K. L., Dayton, R. D., Deverman, B. E., & Klein, R. L. (2016) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2016.00154 · Cited by 69 · see citing works

Adeno-associated viral vector (AAV)-mediated gene transfer in the red nucleus of the adult rat brain: Comparative analysis of the transduction properties of seven AAV serotypes and lentiviral vectors

Blits, B., Derks, S., Twisk, J., Ehlert, E., Prins, J., & Verhaagen, J. (2009) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2009.10.009 · Cited by 66 · see citing works

New tools for investigating astrocyte-to-neuron communication

Li, D., Agulhon, C., Schmidt, E. M., Oheim, M., & Ropert, N. (2013) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2013.00193 · Cited by 66 · see citing works

Is Viral Vector Gene Delivery More Effective Using Biomaterials?

Wang, Y., Bruggeman, K. F., Franks, S. J., Gautam, V., Hodgetts, S. I., Harvey, A. R., Williams, R. J., & Nisbet, D. R. (2020) · Advanced Healthcare Materials · doi:10.1002/adhm.202001238 · Cited by 66 · see citing works

CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)

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Engineered antibody therapies to counteract mutant huntingtin and related toxic intracellular proteins

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Rapid, reproducible transduction of select forebrain regions by targeted recombinant virus injection into the neonatal mouse brain

Pilpel, N., Landeck, N., Klugmann, M., Seeburg, P. H., & Schwarz, M. K. (2009) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2009.05.020 · Cited by 61 · see citing works

Evaluation of the expression pattern of rAAV2/1, 2/5, 2/7, 2/8, and 2/9 serotypes with different promoters in the mouse visual cortex

Scheyltjens, I., Laramée, M., den Haute, C. V., Gijsbers, R., Debyser, Z., Baekelandt, V., Vreysen, S., & Arckens, L. (2015) · The Journal of Comparative Neurology · doi:10.1002/cne.23819 · Cited by 56 · see citing works

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

Ciron, C., Cressant, A., Roux, F., Raoul, S., Chérel, Y., Hantraye, P., Déglon, N., Schwartz, B., Barkats, M., Heard, J., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2008.155 · Cited by 60 · see citing works

Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors

Miyake, N., Miyake, K., Yamamoto, M., Hirai, Y., & Shimada, T. (2011) · Brain Research · doi:10.1016/j.brainres.2011.03.014 · Cited by 60 · see citing works

Efficient gene delivery and selective transduction of astrocytes in the mammalian brain using viral vectors

Merienne, N., Douce, J. L., Faivre, E., Déglon, N., & Bonvento, G. (2013) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2013.00106 · Cited by 58 · see citing works

Disrupting Function of FK506-Binding Protein 1b/12.6 Induces the Ca2+-Dysregulation Aging Phenotype in Hippocampal Neurons

Gant, J. C., Chen, K.-C., Norris, C. M., Kadish, I., Thibault, O., Blalock, E. M., Porter, N. M., & Landfield, P. W. (2011) · Journal of Neuroscience · doi:10.1523/jneurosci.4805-10.2011 · Cited by 55 · see citing works

Tau expression levels from various adeno‐associated virus vector serotypes produce graded neurodegenerative disease states

Klein, R. L., Dayton, R. D., Tatom, J. B., Diaczynsky, C. G., & Salvatore, M. F. (2008) · European Journal of Neuroscience · doi:10.1111/j.1460-9568.2008.06161.x · Cited by 54 · see citing works

More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose–response and comparison to AAV PHP.B

Dayton, R. D., Grames, M. S., & Klein, R. L. (2018) · Gene Therapy · doi:10.1038/s41434-018-0028-5 · Cited by 52 · see citing works

Intraganglionic AAV6 Results in Efficient and Long-Term Gene Transfer to Peripheral Sensory Nervous System in Adult Rats

Yu, H., Fischer, G. J., Ferhatović, L., Fan, F., Light, A. R., Weihrauch, D., Sapunar, D., Nakai, H., Park, F., & Hogan, Q. H. (2013) · PLoS ONE · doi:10.1371/journal.pone.0061266 · Cited by 50 · see citing works

AAV9 supports wide-scale transduction of the CNS and TDP-43 disease modeling in adult rats

Jackson, K. L., Dayton, R. D., & Klein, R. L. (2015) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2015.36 · Cited by 49 · see citing works

Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria

Rebuffat, A., Harding, C. O., Ding, Z., & Thöny, B. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.127 · Cited by 48 · see citing works

Increased Hippocampal Neurogenesis and Accelerated Response to Antidepressants in Mice with Specific Deletion of CREB in the Hippocampus: Role of cAMP Response-Element Modulator τ

Gundersen, B. B., Briand, L. A., Onksen, J. L., Lay, J. E. L., Kaestner, K. H., & Blendy, J. A. (2013) · Journal of Neuroscience · doi:10.1523/jneurosci.1669-13.2013 · Cited by 48 · see citing works

Astrocytes are key but indirect contributors to the development of the symptomatology and pathophysiology of Huntington's disease

Meunier, C., Mérienne, N., Jollé, C., Déglon, N., & Pellerin, L. (2016) · Glia · doi:10.1002/glia.23022 · Cited by 44 · see citing works

Mouse Gender Influences Brain Transduction by Intravascularly Administered AAV9

Maguire, C. A., Crommentuijn, M. H. W., Mu, D., Hudry, E., Serrano‐Pozo, A., Hyman, B. T., & Tannous, B. A. (2013) · Molecular Therapy · doi:10.1038/mt.2013.95 · Cited by 47 · see citing works

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

Hordeaux, J., Dubreil, L., Deniaud, J., Iacobelli, F., Moreau, S., Ledevin, M., Guiner, C. L., Blouin, V., Duff, J. L., Mendes-Madeira, A., et al. (2015) · Gene Therapy · doi:10.1038/gt.2014.121 · Cited by 47 · see citing works

Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis

Polyak, S., Mach, A. S., Porvasnik, S., Dixon, L. R., Conlon, T. J., Erger, K. E., Acosta, A. J., Wright, A. J., Campbell‐Thompson, M., Zolotukhin, I., et al. (2011) · American Journal of Physiology-Gastrointestinal and Liver Physiology · doi:10.1152/ajpgi.00562.2010 · Cited by 47 · see citing works

Emerging technologies to study glial cells

Hirbec, H., Déglon, N., Foo, L. C., Goshen, I., Grutzendler, J., Hangen, E., Kreisel, T., Linck, N., Muffat, J., Regio, S., et al. (2020) · Glia · doi:10.1002/glia.23780 · Cited by 47 · see citing works

Transgenic models of Alzheimer's disease: Better utilization of existing models through viral transgenesis

Platt, T. L., Reeves, V. L., & Murphy, M. P. (2013) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2013.04.017 · Cited by 46 · see citing works

Strategies for targeting primate neural circuits with viral vectors

El-Shamayleh, Y., Ni, A. M., & Horwitz, G. D. (2016) · Journal of Neurophysiology · doi:10.1152/jn.00087.2016 · Cited by 45 · see citing works

Preventing Growth of Brain Tumors by Creating a Zone of Resistance

Maguire, C. A., Meijer, D. H., LeRoy, S. G., Tierney, L. A., Broekman, M. L. D., Costa, F. F., Breakefield, X. O., Stemmer-Rachamimov, A. O., & Sena‐Esteves, M. (2008) · Molecular Therapy · doi:10.1038/mt.2008.168 · Cited by 43 · see citing works

CRTC1 Function During Memory Encoding Is Disrupted in Neurodegeneration

Parra‐Damas, A., Chen, M., Enríquez-Barreto, L., Ortega, L., Acosta, S. P., Camats‐Perna, J., Fullana, M. N., Aguilera, J., Rodrı́guez-Álvarez, J., & Saura, C. A. (2016) · Biological Psychiatry · doi:10.1016/j.biopsych.2016.06.025 · Cited by 42 · see citing works

Adeno-Associated Viral Vectors Serotype 8 for Cell-Specific Delivery of Therapeutic Genes in the Central Nervous System

Pignataro, D., Sucunza, D., Vanrell, L., López‐Franco, E., Dopeso‐Reyes, I. G., Vales, Á., Hommel, M., Rico, A. J., Lanciego, J. L., & González‐Aseguinolaza, G. (2017) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2017.00002 · Cited by 42 · see citing works

GFAP-Driven GFP Expression in Activated Mouse Müller Glial Cells Aligning Retinal Blood Vessels Following Intravitreal Injection of AAV2/6 Vectors

Aartsen, W. M., van Cleef, K. W. R., Pellissier, L. P., Hoek, R. M., Vos, R. M., Blits, B., Ehlert, E. M. E., Balaggan, K. S., Ali, R. R., Verhaagen, J., et al. (2010) · PLoS ONE · doi:10.1371/journal.pone.0012387 · Cited by 41 · see citing works

Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats

Bucher, T., Dubreil, L., Colle, M.-A., Maquigneau, M., Deniaud, J., Ledevin, M., Moullier, P., & Joussemet, B. (2014) · Gene Therapy · doi:10.1038/gt.2014.16 · Cited by 40 · see citing works

Overexpressing neuroglobin improves functional recovery by inhibiting neuronal apoptosis after spinal cord injury

Lan, W., Lin, J.-H., Chen, X., Wu, C., Zhong, G., Zhang, L., Lin, W., Liu, W.-N., Li, X., & Lin, J.-L. (2014) · Brain Research · doi:10.1016/j.brainres.2014.03.020 · Cited by 38 · see citing works

A simplified purification method for AAV variant by polyethylene glycol aqueous two-phase partitioning

Guo, P., Xiao, X., El‐Gohary, Y., Paredes, J. L., Prasadan, K. L., Shiota, C., Wiersch, J. C., Welsh, C., & Gittes, G. K. (2012) · Bioengineered · doi:10.4161/bioe.22293 · Cited by 38 · see citing works

Adenosine kinase, glutamine synthetase and EAAT2 as gene therapy targets for temporal lobe epilepsy

Young, D., Fong, D. M., Lawlor, P. A., Wu, A. J., Mouravlev, A., McRae, M., Glass, M., Dragunow, M., & During, M. J. (2014) · Gene Therapy · doi:10.1038/gt.2014.82 · Cited by 37 · see citing works

AAV8-mediated overexpression of mPCSK9 in liver differs between male and female mice

Vozenilek, A. E., Blackburn, C. M. R., Schilke, R. M., Chandran, S., Castore, R., Klein, R. L., & Woolard, M. D. (2018) · Atherosclerosis · doi:10.1016/j.atherosclerosis.2018.09.005 · Cited by 36 · see citing works

Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain

Swain, G. P., Prociuk, M. L., Bagel, J. H., O’Donnell, P., Berger, K., Drobatz, K., Gurda, B. L., Haskins, M. E., Sands, M. S., & Vite, C. H. (2013) · Gene Therapy · doi:10.1038/gt.2013.54 · Cited by 34 · see citing works

Adeno‐associated virus‐based Alzheimer's disease mouse models and potential new therapeutic avenues

Ittner, L. M., Klugmann, M., & Ke, Y. D. (2019) · British Journal of Pharmacology · doi:10.1111/bph.14637 · Cited by 35 · see citing works

(Pro)renin receptor knockdown in the paraventricular nucleus of the hypothalamus attenuates hypertension development and AT1 receptor-mediated calcium events

Souza, L. A. C., Worker, C. J., Li, W., Trebak, F., Watkins, T. V., Gayban, A. J. B., Yamasaki, E., Cooper, S. G., Drumm, B. T., & Feng, Y. (2019) · American Journal of Physiology-Heart and Circulatory Physiology · doi:10.1152/ajpheart.00780.2018 · Cited by 34 · see citing works

Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction

Arnett, A. L. H., Beutler, L. R., Quintana, A., Allen, J. M., Finn, E. E., Palmiter, R. D., & Chamberlain, J. S. (2012) · Gene Therapy · doi:10.1038/gt.2012.60 · Cited by 33 · see citing works

Targeting Homer genes using adeno-associated viral vector: lessons learned from behavioural and neurochemical studies

Klugmann, M., & Szumlinski, K. K. (2008) · Behavioural Pharmacology · doi:10.1097/fbp.0b013e32830c369f · Cited by 33 · see citing works

Intramuscular Delivery of Gene Therapy for Targeting the Nervous System

Tosolini, A. P., & Sleigh, J. N. (2020) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.00129 · Cited by 32 · see citing works

A next step in adeno‐associated virus‐mediated gene therapy for neurological diseases: regulation and targeting

Chtarto, A., Bockstael, O., Tshibangu, T., Dewitte, O., Levivier, M., & Tenenbaum, L. (2013) · British Journal of Clinical Pharmacology · doi:10.1111/bcp.12065 · Cited by 32 · see citing works

Absence of Nicotinamide Nucleotide Transhydrogenase in C57BL/6J Mice Exacerbates Experimental Atherosclerosis

Vozenilek, A. E., Vetkoetter, M., Green, J. M., Shen, X., Traylor, J. G., Klein, R. L., Orr, A. W., Woolard, M. D., & Krzywanski, D. M. (2018) · Journal of Vascular Research · doi:10.1159/000486337 · Cited by 31 · see citing works

Lentiviral Vectors: A Powerful Tool to Target Astrocytes In Vivo

Delzor, A., Escartin, C., & Déglon, N. (2013) · Current Drug Targets · doi:10.2174/13894501113146660213 · Cited by 31 · see citing works

Gene Transfer to the CNS Using Recombinant Adeno‐Associated Virus

Stoica, L., Ahmed, S. S., Gao, G., & Sena‐Esteves, M. (2013) · Current Protocols in Microbiology · doi:10.1002/9780471729259.mc14d05s29 · Cited by 29 · see citing works

Optimization of Adeno-Associated Viral Vector-Mediated Gene Delivery to the Hypothalamus

de Backer, M. W. A., Brans, M. A. D., Luijendijk, M. C. M., Garner, K. M., & Adan, R. A. H. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.169 · Cited by 30 · see citing works

Recombinant AAV9-TLK1B Administration Ameliorates Fractionated Radiation-Induced Xerostomia

Shanmugam, P. S. T., Dayton, R. D., Palaniyandi, S., Abreo, F. W., Caldito, G. C., Klein, R. L., & Sunavala‐Dossabhoy, G. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.235 · Cited by 28 · see citing works

Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors

Hutt, J. A., Assaf, B. T., Bolon, B. N., Cavagnaro, J. A., Galbreath, E. J., Grubor, B., Kattenhorn, L. M., Romeike, A., & Whiteley, L. O. (2021) · Toxicologic Pathology · doi:10.1177/01926233211041962 · Cited by 27 · see citing works

Adeno-Associated Viral Vector Serotype DJ-Mediated Overexpression of N171-82Q-Mutant Huntingtin in the Striatum of Juvenile Mice Is a New Model for Huntington’s Disease

Jang, M., Lee, S. E., & Cho, I. (2018) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2018.00157 · Cited by 25 · see citing works

Gene delivery in tissue engineering and regenerative medicine

Fang, Y. L., Chen, X., & Godbey, W. T. (2014) · Journal of Biomedical Materials Research Part B Applied Biomaterials · doi:10.1002/jbm.b.33354 · Cited by 24 · see citing works

Reply to Dr Fricke

Aspelin, P., & Lundkvist, J. (2005) · American Heart Journal · doi:10.1016/j.ahj.2005.06.002

Comparative Analysis of Six Adeno-Associated Viral Vector Serotypes in Mouse Inferior Colliculus and Cerebellum

Witteveen, I. F., & Balmer, T. S. (2024) · eNeuro · doi:10.1523/eneuro.0391-24.2024 · Cited by 8 · see citing works

Comparative AAV-eGFP Transgene Expression Using Vector Serotypes 1–9, 7m8, and 8b in Human Pluripotent Stem Cells, RPEs, and Human and Rat Cortical Neurons

Duong, T. T., Lim, J., Vasireddy, V., Papp, T., Nguyen, H., Leo, L., Pan, J., Zhou, S., Chen, H. I., Bennett, J., et al. (2019) · Stem Cells International · doi:10.1155/2019/7281912 · Cited by 33 · see citing works

Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex

Watakabe, A., Ohtsuka, M., Kinoshita, M., Takaji, M., Isa, K., Mizukami, H., Ozawa, K., Isa, T., & Yamamori, T. (2015) · Neuroscience Research · doi:10.1016/j.neures.2014.09.002 · Cited by 303 · see citing works

Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates

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