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

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

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Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses

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Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

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Neuroinflammation in amyotrophic lateral sclerosis: role of glial activation in motor neuron disease

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Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

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Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

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Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors

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Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

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Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

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Comparative Analysis of Adeno-Associated Viral Vector Serotypes 1, 2, 5, 7, And 8 in Mouse Brain

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Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

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Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

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Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

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Terminal N-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9

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High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

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Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

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

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The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice

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Enhanced Survival of the LINCL Mouse Following CLN2 Gene Transfer Using the rh.10 Rhesus Macaque-derived Adeno-associated Virus Vector

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Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs

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Comparison of Adeno-Associated Viral Vector Serotypes for Spinal Cord and Motor Neuron Gene Delivery

Snyder, B. R., Gray, S. J., Quach, E. T., Huang, J. W., Leung, C. H., Samulski, R. J., Boulis, N. M., & Federici, T. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.008 · Cited by 156 · see citing works

Strong Cortical and Spinal Cord Transduction After AAV7 and AAV9 Delivery into the Cerebrospinal Fluid of Nonhuman Primates

Samaranch, L., Salegio, E. A., Sebastián, W. S., Kells, A. P., Bringas, J. R., Forsayeth, J., & Bankiewicz, K. S. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.005 · Cited by 155 · see citing works

Oligodendrocytes and the early multiple sclerosis lesion

Prineas, J. W., & Parratt, J. D. E. (2012) · Annals of Neurology · doi:10.1002/ana.23634 · Cited by 149 · see citing works

Identification of the Galactose Binding Domain of the Adeno-Associated Virus Serotype 9 Capsid

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Oligodendrocyte-protection and remyelination post-spinal cord injuries: A review

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Gene therapy for leukodystrophies

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

Piguet, F., Sondhi, D., Piraud, M., Fouquet, F., Hackett, N. R., Ahouansou, O., Vanier, M.-T., Bieche, I., Aubourg, P., Crystal, R. G., et al. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.015 · Cited by 91 · see citing works

Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy

Bradbury, A. M., Cochran, J. N., McCurdy, V. J., Johnson, A. K., Brunson, B. L., Gray‐Edwards, H. L., LeRoy, S. G., Hwang, M., Randle, A. N., Jackson, L. S., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.86 · Cited by 87 · see citing works

Oligodendrocyte-specific gene expression in mouse brain: Use of a myelin-forming cell type-specific promoter in an adeno-associated virus

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Glycan Binding Avidity Determines the Systemic Fate of Adeno-Associated Virus Type 9

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Susceptibility of Domestic Cats to Chronic Wasting Disease

Mathiason, C. K., Nalls, A. V., Seelig, D. M., Kraft, S. L., Carnes, K. D., Anderson, K. R., Hayes‐Klug, J., & Hoover, E. A. (2012) · Journal of Virology · doi:10.1128/jvi.02592-12 · Cited by 59 · see citing works

AAV as a Gene Transfer Vector for the Treatment of Neurological Disorders: Novel Treatment Thoughts for ALS

Hester, M. E., Foust, K. D., Kaspar, R. W., & Kaspar, B. K. (2009) · Current Gene Therapy · doi:10.2174/156652309789753383 · Cited by 51 · see citing works

An ~140-kb deletion associated with feline spinal muscular atrophy implies an essentialLIX1function for motor neuron survivalPreprint

Fyfe, J. C., Menotti-Raymond, M. A., David, V. A., Brichta, L., Schäffer, A. A., Agarwala, R., Murphy, W. J., Wedemeyer, W. J., Gregory, B. L., Buzzell, B. G., et al. (2006) · Genome Research · doi:10.1101/gr.5268806 · Cited by 50 · see citing works

scAAV9 Intracisternal Delivery Results in Efficient Gene Transfer to the Central Nervous System of a Feline Model of Motor Neuron Disease

Bucher, T., Colle, M.-A., Wakeling, E. N., Dubreil, L., Fyfe, J. C., Briot, D., Maquigneau, M., Raoul, S., Chérel, Y., Astord, S., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.218 · Cited by 44 · see citing works

Electrodiagnostic Testing and Histopathologic Changes Confirm Peripheral Nervous System Myelin Abnormalities in the Feline Model of Niemann-Pick Disease Type C

Bagel, J. H., Sikora, T. U., Prociuk, M. L., Pesayco, J. P., Mizisin, A. P., Shelton, G. D., & Vite, C. H. (2013) · Journal of Neuropathology & Experimental Neurology · doi:10.1097/nen.0b013e318286587f · Cited by 25 · see citing works

Identification of Bangladeshi Domestic Cats with GM1 Gangliosidosis Caused by the c.1448G>C Mutation of the Feline GLB1 Gene: Case Study

UDDIN, M. M., HOSSAIN, M. A., Rahman, M. M., CHOWDHURY, M. A., TANIMOTO, T., Yabuki, A., Mizukami, K., Chang, H.-S., & Yamato, O. (2012) · Journal of Veterinary Medical Science · doi:10.1292/jvms.12-0307 · Cited by 9 · see citing works

Mutational analysis of the feline CLN3 gene and an ultrastructural evaluation of lysosomal storage materials in a cat with neuronal ceroid lipofuscinosis: An investigation into the molecular basis of the disease

Furusawa, Y., Mizukami, K., Yabuki, A., Kuwamura, M., Chang, H. S., Hossain, M. A., Rahman, M. M., Uddin, M. M., Mitani, S., & Yamato, O. (2012) · The Veterinary Journal · doi:10.1016/j.tvjl.2012.04.025 · Cited by 8 · see citing works

Erratum: Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Gray, S. J., Kalburgi, S. N., McCown, T. J., & Samulski, R. J. (2013) · Gene Therapy · doi:10.1038/gt.2013.11 · Cited by 2 · see citing works

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201 · see citing works

AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

Rosenberg, J. B., Kaplitt, M. G., De, B. P., Chen, A., Flagiello, T., Salami, C., Pey, E., Zhao, L., Ricart Arbona, R. J., Monette, S., et al. (2018) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2017.231 · Cited by 169 · see citing works

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Hinderer, C., Bell, P., Katz, N., Vite, C. H., Louboutin, J.-P., Bote, E., Yu, H., Zhu, Y., Casal, M. L., Bagel, J., et al. (2018) · Human Gene Therapy · doi:10.1089/hum.2017.026 · Cited by 141 · see citing works

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

Hinderer, C. J., Bell, P. L., Gurda, B. L., Wang, Q., Louboutin, J., Zhu, Y., Bagel, J. H., O’Donnell, P., Sikora, T. U., Ruane, T., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.135 · Cited by 108 · see citing works

AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy

O’Carroll, S. J., Cook, W. H., & Young, D. (2021) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.618020 · Cited by 105 · see citing works

Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters

Nieuwenhuis, B., Haenzi, B., Hilton, S., Carnicer-Lombarte, A., Hobo, B., Verhaagen, J., & Fawcett, J. W. (2021) · Gene Therapy · doi:10.1038/s41434-020-0169-1 · Cited by 114 · see citing works

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 · see citing works

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII

Gurda, B. L., de Lataillade, A. D. G., Bell, P. L., Zhu, Y., Yu, H., Wang, P., Bagel, J. H., Vite, C. H., Sikora, T. U., Hinderer, C. J., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.189 · Cited by 81 · see citing works

Gene therapy for Mucopolysaccharidoses

Sawamoto, K., Chen, H.-H., Alméciga-Díaz, C. J., Mason, R. W., & Tomatsu, S. (2017) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2017.12.434 · Cited by 76 · see citing works

Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders

Bey, K., Ciron, C., Dubreil, L., Deniaud, J., Ledevin, M., Cristini, J., Blouin, V., Aubourg, P. R., & Colle, M.-A. (2017) · Gene Therapy · doi:10.1038/gt.2017.18 · Cited by 73 · see citing works

Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results

Sevigny, J., Uspenskaya, O., Heckman, L. D., Wong, L. C., Hatch, D. A., Tewari, A., Vandenberghe, R. R., Irwin, D. J., Saracino, D., Ber, I. L., et al. (2024) · Nature Medicine · doi:10.1038/s41591-024-02973-0 · Cited by 72 · see citing works

AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials

Hardcastle, N., Boulis, N. M., & Federici, T. (2017) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2018.1416089 · Cited by 70 · see citing works

A Multifaceted Approach to Optimizing AAV Delivery to the Brain for the Treatment of Neurodegenerative Diseases

Fischell, J. M., & Fishman, P. S. (2021) · Frontiers in Neuroscience · doi:10.3389/fnins.2021.747726 · Cited by 66 · see citing works

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

Motas, S., Haurigot, V., Garcia, M., Marcó, S., Ribera, A., Roca, C., Sánchez, X., Sánchez, V., Molas, M., Bertolin, J., et al. (2016) · JCI Insight · doi:10.1172/jci.insight.86696 · Cited by 63 · see citing works

Lysosomal storage disease: Gene therapy on both sides of the blood–brain barrier

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Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease

Hordeaux, J., Dubreil, L., Robveille, C., Deniaud, J., Pascal, Q., Déqueant, B., Pailloux, J., Lagalice, L., Ledevin, M., Babarit, C., et al. (2017) · Acta Neuropathologica Communications · doi:10.1186/s40478-017-0464-2 · Cited by 59 · see citing works

Efficacy and Safety of a Krabbe Disease Gene Therapy

Hordeaux, J., Jeffrey, B. A., Jian, J., Choudhury, G. R., Michalson, K. T., Mitchell, T. W., Buza, E. L., Chichester, J. A., Dyer, C., Bagel, J. H., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2021.245 · Cited by 51 · see citing works

The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System

Piguet, F., Saint-Denis, T. D., Audouard, E., Beccaria, K., André, A., Wurtz, G., Schatz, R., Alves, S., Sevin, C., Zérah, M., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.105 · Cited by 44 · see citing works

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice

Hinderer, C. J., Katz, N., Louboutin, J., Bell, P. L., Yu, H., Nayal, M., Kozarsky, K., O'Brien, W. T., Goode, T. L., & Wilson, J. M. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.101 · Cited by 44 · see citing works

Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion

Castle, M. J., Cheng, Y., Asokan, A., & Tuszynski, M. H. (2018) · Science Advances · doi:10.1126/sciadv.aau9859 · Cited by 41 · see citing works

Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates

Ballon, D., Rosenberg, J. B., Fung, E. K., Nikolopoulou, A., Kothari, P. J., De, B. P., He, B., Chen, A., Heier, L., Sondhi, D., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.116 · Cited by 37 · see citing works

Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1

Yoon, S. Y., Bagel, J. H., O’Donnell, P. A., Vite, C. H., & Wolfe, J. H. N. (2015) · Molecular Therapy · doi:10.1038/mt.2015.168 · Cited by 31 · see citing works

Lot-to-Lot Variation in Adeno-Associated Virus Serotype 9 (AAV9) Preparations

O'Connor, D. M., Lutomski, C. A., Jarrold, M. F., Boulis, N. M., & Donsante, A. (2019) · Human Gene Therapy Methods · doi:10.1089/hgtb.2019.105 · Cited by 24 · see citing works

Allele-specific silencing as treatment for gene duplication disorders: proof-of-principle in autosomal dominant leukodystrophy

Giorgio, E., Lorenzati, M., Cervo, P. R. D. V., Brussino, A., Cernigoj, M., Sala, E. D., Stella, A. B., Ferrero, M., Caiazzo, M., Capellari, S., et al. (2019) · Brain · doi:10.1093/brain/awz139 · Cited by 25 · see citing works

Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis

Gross, A. L., Gray‐Edwards, H. L., Bebout, C. N., Ta, N. L., Nielsen, K., Brunson, B. L., Mercado, K. R. L., Osterhoudt, D. E., Batista, A. R., Maitland, S. A., et al. (2021) · Brain · doi:10.1093/brain/awab309 · Cited by 21 · see citing works

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

De, B. P., Rosenberg, J. B., Selvan, N., Wilson, I., Yusufzai, N., Greco, A., Kaminsky, S. M., Heier, L., Arbona, R. J. R., Miranda, I. C., et al. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.067 · Cited by 12 · see citing works

Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid

Rosenberg, J. B., Fung, E. K., Dyke, J. P., De, B. P., Lou, H. H., Kelly, J. M., Reejhsinghani, L., Arbona, R. J. R., Sondhi, D., Kaminsky, S. M., et al. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.060 · Cited by 11 · see citing works

The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA

Christie-Brown, V., Mitchell, J., & Talbot, K. (2017) · Gene Therapy · doi:10.1038/gt.2017.47 · Cited by 10 · see citing works

Adeno-associated virus-binding antibodies detected in cats living in the Northeastern United States lack neutralizing activity

Adachi, K., Dissen, G. A., Lomniczi, A., Xie, Q., Ojeda, S. R., & Nakai, H. (2020) · Scientific Reports · doi:10.1038/s41598-020-66596-4 · Cited by 10 · see citing works

Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy

Özgür-Günes, Y., Chedik, M., Le Stunff, C., Fovet, C.-M., & Bougnères, P. (2022) · Human Gene Therapy · doi:10.1089/hum.2021.293 · Cited by 10 · see citing works

Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space

Wang, D., Li, J., Tran, K., Burt, D. R., Li, Z., & Gao, G. (2018) · Human Gene Therapy Methods · doi:10.1089/hgtb.2017.250 · Cited by 10 · see citing works

Cre-recombinase systems for induction of neuron-specific knockout models: a guide for biomedical researchers

Shcholok, T., & Eftekharpour, E. (2022) · Neural Regeneration Research · doi:10.4103/1673-5374.346541 · Cited by 9 · see citing works

Adeno-associated virus vectors for human gene therapy

Chen, H. (2015) · World Journal of Medical Genetics · doi:10.5496/wjmg.v5.i3.28 · Cited by 7 · see citing works

Intravascular injections of adenoassociated viral vector serotypes rh10 and PHP.B transduce murine sciatic nerve axons

Anderson, H. E., Schaller, K., Caldwell, J. H., & Weir, R. F. F. (2019) · Neuroscience Letters · doi:10.1016/j.neulet.2019.05.010 · Cited by 4 · see citing works

In vivo studies of redox biochemistry in vertebrate tissues - present successes and future horizons

Naumov, L. L., Sergeeva, A. D., Katrukha, V. A., Kostyuk, A. I., Brazhe, N. A., Bilan, D. S., & Khramova, Y. V. (2025) · Redox Biochemistry and Chemistry · doi:10.1016/j.rbc.2025.100069 · Cited by 2 · see citing works

An evaluation of distinct adeno-associated virus vector strategies for driving transgene expression in spinal inhibitory neurons of the rat

Klinger, L., Siegert, A., Holzinger, R., Trofimova, L., Ada, S., & Drdla-Schutting, R. (2025) · Frontiers in Neuroscience · doi:10.3389/fnins.2025.1558581 · Cited by 2 · see citing works

Intra-arterial Gene Delivery for Neurodegenerative Diseases

Fischell, J. M. (2025) · Springer eBooks · doi:10.1007/978-3-031-80173-0_14

Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in mice

Mole, A. J., Sander, C. F., Parmar, A., Williams, A. J., Azzouz, M., Hautbergue, G. M., Shaw, P. J., Ferraiuolo, L., & Mead, R. J. (2026) · Scientific Reports · doi:10.1038/s41598-026-38039-z

Étude moléculaire de la propagation de l’agrégation de l’alpha-synucléine dans un modèle transgénique de la maladie de Parkinson : impact de la surexpression de la beta-synucléine à l’aide de vecteurs viraux adéno-associés (AAV)

Sargent, D. (2018) · theses.fr (ABES) · doi:10.70675/2cd4ead6z0cd7z476dz8376z54bb3bd9a0ea

Preclinical gene therapy using recombinant AAV vectors in mouse models of two human diseases

Günes, Y. Ö. (2022) · theses.fr (ABES) · doi:10.70675/52c9a808z1652z42c4zae82zc812b95f1723

Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in micePreprint

Mole, A. J., Sander, C. F., Parmar, A., Williams, A. J., Azzouz, M., Hautbergue, G. M., Shaw, P. J., Ferraiuolo, L., & Mead, R. J. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.07.01.662642 · Cited by 1 · 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

In Brief

(2009) · Nature Reviews Genetics · doi:10.1038/nrg2533

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 · see citing works

scAAV9 Intracisternal Delivery Results in Efficient Gene Transfer to the Central Nervous System of a Feline Model of Motor Neuron Disease

Bucher, T., Colle, M.-A., Wakeling, E. N., Dubreil, L., Fyfe, J. C., Briot, D., Maquigneau, M., Raoul, S., Chérel, Y., Astord, S., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.218 · Cited by 44 · see citing works