Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques

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 · see citing works

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses

Calcedo, R., Vandenberghe, L. H., Gao, G., Lin, J., & Wilson, J. M. (2009) · The Journal of Infectious Diseases · doi:10.1086/595830 · Cited by 808 · see citing works

Immune and inflammatory mechanisms in neuropathic pain

Moalem‐Taylor, G., & Tracey, D. J. (2006) · Brain Research Reviews · doi:10.1016/j.brainresrev.2005.11.004 · Cited by 772 · see citing works

The neuro-immune balance in neuropathic pain: Involvement of inflammatory immune cells, immune-like glial cells and cytokines

Austin, P. J., & Moalem-Taylor, G. (2010) · Journal of Neuroimmunology · doi:10.1016/j.jneuroim.2010.08.013 · Cited by 607 · see citing works

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

Gray, S. J., Nagabhushan Kalburgi, S., McCown, T. J., & Jude Samulski, R. (2013) · Gene Therapy · doi:10.1038/gt.2012.101 · Cited by 377 · cited 1× · see citing works

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

Haurigot, V., Marcó, S., Ribera, A., Garcia, M., Ruzo, A., Villacampa, P., Ayuso, E., Añor, S., Andaluz, A., Pineda, M., et al. (2013) · Journal of Clinical Investigation · doi:10.1172/jci66778 · Cited by 215 · see citing works

Vascularization of the Dorsal Root Ganglia and Peripheral Nerve of the Mouse: Implications for Chemical-Induced Peripheral Sensory Neuropathies

Jiménez‐Andrade, J. M., Herrera, M. B., Ghilardi, J. R., Vardanyan, M., Melemedjian, O. K., & Mantyh, P. W. (2008) · Molecular Pain · doi:10.1186/1744-8069-4-10 · Cited by 199 · see citing works

Immunohistochemical analysis of anti-Hu-associated paraneoplastic encephalomyelitis

Bernal, F., Graus, F., Pifarré, À., Saiz, A., Benyahia, B., & Ribalta, T. (2002) · Acta Neuropathologica · doi:10.1007/s00401-001-0498-0 · Cited by 171 · see citing works

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

Muenzer, J., Hendriksz, C. J., Fan, Z., Vijayaraghavan, S., Perry, V., Santra, S., Solanki, G. A., Mascelli, M. A., Pan, L., Wang, N., et al. (2015) · Genetics in Medicine · doi:10.1038/gim.2015.36 · Cited by 168 · 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

The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

Wang, L., Calcedo, R., Wang, H., Bell, P., Grant, R., Vandenberghe, L. H., Sanmiguel, J., Morizono, H., Batshaw, M. L., & Wilson, J. M. (2010) · Molecular Therapy · doi:10.1038/mt.2009.245 · Cited by 130 · 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

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques

Hordeaux, J., Hinderer, C., Goode, T., Katz, N., Buza, E. L., Bell, P., Calcedo, R., Richman, L. K., & Wilson, J. M. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2018.06.003 · Cited by 105 · see citing works

Adeno-Associated Virus-Mediated Gene Transfer to Nonhuman Primate Liver Can Elicit Destructive Transgene-Specific T Cell Responses

Gao, G., Wang, Q., Calcedo, R., Mays, L., Bell, P. L., Wang, L., Vandenberghe, L. H., Grant, R. L., Sanmiguel, J. C., Furth, E. E., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.060 · Cited by 103 · see citing works

Immunopathogenesis of paraneoplastic neurological syndromes associated with anti-Hu antibodies

Pignolet, B., Gebauer, C., & Liblau, R. (2013) · OncoImmunology · doi:10.4161/onci.27384 · Cited by 91 · see citing works

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

Golebiowski, D., van der Bom, I. M. J., Kwon, C.-S., Miller, A. D., Petrosky, K., Bradbury, A. M., Maitland, S., Kühn, A. L., Bishop, N., Curran, E., et al. (2017) · Human Gene Therapy · doi:10.1089/hum.2016.109 · Cited by 87 · see citing works

Neonatal systemic AAV induces tolerance to CNS gene therapy in MPS I dogs and nonhuman primates

Hinderer, C., Bell, P., Louboutin, J.-P., Zhu, Y., Yu, H., Lin, G., Choa, R., Gurda, B. L., Bagel, J., O'Donnell, P., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.99 · Cited by 85 · cited 1× · see citing works

Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients

Parini, R., Rigoldi, M., Tedesco, L., Boffi, L., Brambilla, A., Bertoletti, S., Boncimino, A., Longo, A. D., Lorenzo, P. D., Gaini, R. M., et al. (2015) · Molecular Genetics and Metabolism Reports · doi:10.1016/j.ymgmr.2015.03.011 · Cited by 80 · see citing works

Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years

Tomanin, R., Zanetti, A., D’Avanzo, F., Rampazzo, A., Gasparotto, N., Parini, R., Pascarella, A., Concolino, D., Procopio, E., Fiumara, A., et al. (2014) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-014-0129-1 · Cited by 59 · see citing works

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR ^+/− Rhesus Macaques

Greig, J. A., Limberis, M. P., Bell, P., Chen, S.-J., Calcedo, R., Rader, D. J., & Wilson, J. M. (2017) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2017.014 · Cited by 56 · see citing works

Human dorsal-root-ganglion perfusion measured in-vivo by MRI

Godel, T., Pham, M., Heiland, S., Bendszus, M., & Bäumer, P. (2016) · NeuroImage · doi:10.1016/j.neuroimage.2016.07.030 · Cited by 49 · 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

Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

Hinderer, C. J., Bell, P. L., Louboutin, J., Katz, N., Zhu, Y., Lin, G. H. Y., Choa, R., Bagel, J. H., O’Donnell, P., Fitzgerald, C. A., et al. (2016) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2016.06.006 · Cited by 39 · see citing works

Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairment

Hendriksz, C. J., Muenzer, J., Vanderver, A. L., Davis, J. M., Burton, B. K., Mendelsohn, N. J., Wang, N., Pan, L., Pano, A., & Barbier, A. (2015) · Molecular Genetics and Metabolism Reports · doi:10.1016/j.ymgmr.2015.11.001 · Cited by 31 · see citing works

Cognitive and behaviour profiles of children with mucopolysaccharidosis Type II

Crowe, L. M., Yaplito‐Lee, J., Anderson, V. A., & Peters, H. L. (2017) · Cognitive Neuropsychology · doi:10.1080/02643294.2017.1401530 · Cited by 8 · see citing works

263. Evaluation of Intrathecal rAAV Vectors in Canine Mucopolysaccharidosis VII

(2014) · Molecular Therapy · doi:10.1016/s1525-0016(16)35276-5 · Cited by 1 · see citing works

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. F., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 895 · see citing works

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

Hordeaux, J., Buza, E. L., Dyer, C., Goode, T., Mitchell, T. W., Richman, L., Denton, N., Hinderer, C., Katz, N., Schmid, R., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.167 · Cited by 257 · see citing works

Overcoming innate immune barriers that impede AAV gene therapy vectors

Muhuri, M., Maeda, Y., Ma, H., Ram, S., Fitzgerald, K. A., Tai, P. W. L., & Gao, G. (2021) · Journal of Clinical Investigation · doi:10.1172/jci143780 · Cited by 212 · see citing works

AAV-based in vivo gene therapy for neurological disorders

Ling, Q., Herstine, J. A., Bradbury, A. M., & Gray, S. J. (2023) · Nature Reviews Drug Discovery · doi:10.1038/s41573-023-00766-7 · Cited by 206 · see citing works

MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates

Hordeaux, J., Buza, E. L., Jeffrey, B., Song, C., Jahan, T., Yuan, Y., Zhu, Y., Bell, P., Li, M., Chichester, J. A., et al. (2020) · Science Translational Medicine · doi:10.1126/scitranslmed.aba9188 · Cited by 174 · see citing works

AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice

Liguore, W. A., Domire, J. S., Button, D. A., Wang, Y., Dufour, B. D., Srinivasan, S., & McBride, J. L. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.07.017 · Cited by 148 · see citing works

Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain

Chuapoco, M. R., Flytzanis, N. C., Goeden, N., Octeau, J. C., Roxas, K. M., Chan, K. Y., Scherrer, J., Winchester, J., Blackburn, R. J., Campos, L., et al. (2023) · Nature Nanotechnology · doi:10.1038/s41565-023-01419-x · Cited by 132 · see citing works

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

Bey, K., Deniaud, J., Dubreil, L., Joussemet, B., Cristini, J., Ciron, C., Hordeaux, J., Boulc’h, M. L., Marche, K., Maquigneau, M., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.04.001 · Cited by 115 · see citing works

Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases

Perez, B. A., Shutterly, A., Chan, Y. K., Byrne, B. J., & Corti, M. (2020) · Brain Sciences · doi:10.3390/brainsci10020119 · Cited by 112 · see citing works

A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna

Taghian, T., Marosfoi, M. G., Puri, A. S., Cataltepe, O. I., King, R. M., Diffie, E. B., Maguire, A. S., Martin, D. R., Fernau, D., Batista, A. R., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.11.012 · Cited by 111 · see citing works

Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies

Bailey, R. M., Rozenberg, A., & Gray, S. J. (2020) · Brain Research · doi:10.1016/j.brainres.2020.146832 · Cited by 83 · see citing works

Characterization of AAV-mediated dorsal root ganglionopathy

Buss, N., Lanigan, L., Zeller, J., Cissell, D., Metea, M., Adams, E., Higgins, M., Kim, K. H., Budzynski, E., Yang, L., et al. (2022) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2022.01.013 · Cited by 81 · see citing works

Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys

Hordeaux, J., Hinderer, C. J., Buza, E. L., Louboutin, J., Jahan, T., Bell, P. L., Chichester, J. A., Tarantal, A. F., & Wilson, J. M. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.012 · Cited by 76 · see citing works

High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models

Belbellaa, B., Reutenauer, L., Messaddeq, N., Monassier, L., & Puccio, H. M. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.08.018 · Cited by 75 · see citing works

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

Gross, D., Tedesco, N., Leborgne, C., & Ronzitti, G. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.857276 · Cited by 63 · see citing works

Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS

Stanton, A. C., Lagerborg, K. A., Tellez, L., Krunnfusz, A., King, E. M., Ye, S. H., Solomon, I. H., Tabebordbar, M., & Sabeti, P. C. (2022) · Med · doi:10.1016/j.medj.2022.11.002 · Cited by 61 · see citing works

Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies

Chu, W. S., & Ng, J. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.658038 · Cited by 59 · see citing works

Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration

Hinderer, C. J., Katz, N., Dyer, C., Goode, T. L., Johansson, J., Bell, P. L., Richman, L. K., Buza, E. L., & Wilson, J. M. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.04.012 · Cited by 56 · see citing works

Revisiting the “New” Inflammatory Toxicities of Adeno-Associated Virus Vectors

Flotte, T. R. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.29117.trf · Cited by 55 · see citing works

Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations

Hinderer, C. J., Miller, R. C., Dyer, C., Johansson, J., Bell, P. L., Buza, E. L., & Wilson, J. M. (2020) · Annals of Clinical and Translational Neurology · doi:10.1002/acn3.51165 · Cited by 51 · see citing works

Optimization of AAV vectors to target persistent viral reservoirs

Colón-Thillet, R., Jerome, K. R., & Stone, D. (2021) · Virology Journal · doi:10.1186/s12985-021-01555-7 · Cited by 50 · 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

Comparative Pathology of the Peripheral Nervous System

Lanigan, L. G., Russell, D. S., Woolard, K., Pardo, I. D. R., Godfrey, V., Jortner, B. S., Butt, M. T., & Bolon, B. N. (2020) · Veterinary Pathology · doi:10.1177/0300985820959231 · Cited by 47 · see citing works

Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy

Rosenberg, J. B., Chen, A., De, B. P., Dyke, J. P., Ballon, D. J., Monette, S., Ricart Arbona, R. J., Kaminsky, S. M., Crystal, R. G., & Sondhi, D. (2021) · Human Gene Therapy · doi:10.1089/hum.2020.269 · Cited by 41 · see citing works

Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies

Stone, D., Aubert, M., & Jerome, K. R. (2023) · Gene Therapy · doi:10.1038/s41434-023-00405-1 · Cited by 41 · see citing works

Circulating neurofilament light chain as a promising biomarker of AAV-induced dorsal root ganglia toxicity in nonclinical toxicology species

Fader, K. A., Pardo, I. D. R., Kovi, R. C., Somps, C. J., Wang, H. H., Vaidya, V. S., Ramaiah, S. K., & Sirivelu, M. P. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.03.017 · Cited by 40 · see citing works

Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1

Kurokawa, Y., Osaka, H., Kouga, T., Jimbo, E. F., Muramatsu, K., Nakamura, S., Takayanagi, Y., Onaka, T., Muramatsu, S., & Yamagata, T. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.175 · Cited by 40 · see citing works

Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey Central Nervous System

Galván, A., Petkau, T. L., Hill, A. M., Korecki, A. J., Lu, G., Choi, D., Rahman, K. A., Simpson, E. M., Leavitt, B. R., & Smith, Y. (2021) · Human Gene Therapy · doi:10.1089/hum.2020.301 · Cited by 38 · see citing works

Gene and Cell-Based Therapies for Parkinson's Disease: Where Are We?

Buttery, P. C., & Barker, R. A. (2020) · Neurotherapeutics · doi:10.1007/s13311-020-00940-4 · Cited by 39 · see citing works

Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal

Marcó, S., Haurigot, V., Jaén, M. L., Ribera, A., Sánchez, V., Molas, M., Garcia, M., León, X., Roca, C., Sánchez, X., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.09.017 · Cited by 36 · see citing works

Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery

Kagiava, A., Richter, J., Tryfonos, C., Leal-Julià, M., Sargiannidou, I., Christodoulou, C., Bosch, A., & Kleopa, K. A. (2021) · Scientific Reports · doi:10.1038/s41598-021-02694-1 · Cited by 34 · see citing works

Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?

Zhan, W., Muhuri, M., Tai, P. W. L., & Gao, G. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.673699 · Cited by 33 · see citing works

The Perspective of DMPK on Recombinant Adeno-Associated Virus-Based Gene Therapy: Past Learning, Current Support, and Future Contribution

Chen, N., Sun, K., Chemuturi, N. V., Cho, H., & Xia, C. Q. (2022) · The AAPS Journal · doi:10.1208/s12248-021-00678-7 · Cited by 30 · see citing works

The use of viral vectors to promote repair after spinal cord injury

Islam, A., & Tom, V. J. (2022) · Experimental Neurology · doi:10.1016/j.expneurol.2022.114102 · Cited by 29 · 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

Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase

Hordeaux, J., Ramezani, A., Tuske, S., Mehta, N., Song, C., Lynch, A., Lupino, K., Chichester, J. A., Buza, E. L., Dyer, C., et al. (2023) · Frontiers in Immunology · doi:10.3389/fimmu.2023.1094279 · Cited by 23 · see citing works

Charcot–Marie–Tooth neuropathies: Current gene therapy advances and the route toward translation

Stavrou, M., Kagiava, A., Sargiannidou, I., Georgiou, E., & Kleopa, K. A. (2023) · Journal of the Peripheral Nervous System · doi:10.1111/jns.12543 · Cited by 23 · see citing works

Intra-cisterna magna delivery of an AAV vector with the GLUT1 promoter in a pig recapitulates the physiological expression of SLC2A1

Nakamura, S., Osaka, H., Muramatsu, S., Takino, N., Ito, M., Jimbo, E. F., Watanabe, C., Hishikawa, S., Nakajima, T., & Yamagata, T. (2020) · Gene Therapy · doi:10.1038/s41434-020-00203-z · Cited by 20 · see citing works

Mammalian Sulfatases: Biochemistry, Disease Manifestation, and Therapy

Mashima, R., & Nakanishi, M. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23158153 · Cited by 20 · see citing works

Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus

Borges, B., Varthaliti, A., Schwab, M., Clarke, M. T., Pivetti, C., Gupta, N., Cadwell, C., Guibinga, G. H., Phillips, S., del Rio, T., et al. (2024) · Molecular Therapy: Methods & Clinical Development · doi:10.1016/j.omtm.2024.101263 · Cited by 18 · see citing works

Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector

Kudo, M., Sidikejiang, W., Fujiwara, M., Saito, Y., Kubota, S., Inoue, K., Takada, M., & Seki, K. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.07.009 · Cited by 18 · see citing works

AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

Hocquemiller, M., Giersch, L., Mei, X., Gross, A. L., Randle, A. N., Gray‐Edwards, H. L., Hudson, J. A., Todeasa, S. H., Stoica, L., Martin, D. R., et al. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.10.004 · Cited by 18 · see citing works

Viral Vector-Based Gene Therapy for Epilepsy: What Does the Future Hold?

Bettegazzi, B., Cattaneo, S., Simonato, M., Zucchini, S., & Soukupovà, M. (2023) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-023-00687-6 · Cited by 15 · see citing works

Pre-clinical delivery of gene therapy products to the cerebrospinal fluid: challenges and considerations for clinical translation

Salegio, E. A., Hancock, K., & Korszen, S. (2023) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2023.1248271 · Cited by 13 · see citing works

Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47

Wiseman, J. P., Scarrott, J. M., Alves-Cruzeiro, J., Saffari, A., Böger, C., Karyka, E., Dawes, E., Davies, A. K., Marchi, P. M., Graves, E. J., et al. (2024) · EMBO Molecular Medicine · doi:10.1038/s44321-024-00148-5 · Cited by 13 · see citing works

Scientific and Regulatory Policy Committee Technical Review: Biology and Pathology of Ganglia in Animal Species Used for Nonclinical Safety Testing

Bennet, B. M., Pardo, I. D. R., Assaf, B. T., Buza, E. L., Cramer, S. D., Crawford, L. K., Engelhardt, J. A., Galbreath, E. J., Grubor, B., Morrison, J. P., et al. (2023) · Toxicologic Pathology · doi:10.1177/01926233231213851 · Cited by 13 · see citing works

Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy

Nasir, G., Chopra, R., Elwood, F., & Ahmed, S. S. (2021) · Frontiers in Medicine · doi:10.3389/fmed.2021.760236 · Cited by 13 · see citing works

Neuropathological Findings in Nonclinical Species Following Administration of Adeno-Associated Virus (AAV)-Based Gene Therapy Vectors

Bolon, B. N., Buza, E. L., Galbreath, E. J., Wicks, J. R., Cargnin, F., & Hordeaux, J. (2024) · Toxicologic Pathology · doi:10.1177/01926233241300314 · Cited by 12 · see citing works

Society of Toxicologic Pathology Neuropathology Interest Group Article: Neuropathologic Findings in Nonhuman Primates Associated With Administration of Biomolecule-Based Test Articles

Bangari, D. S., Lanigan, L. G., Goulet, F., Sisó, S., & Bolon, B. N. (2022) · Toxicologic Pathology · doi:10.1177/01926233221101314 · Cited by 10 · see citing works

Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies

Minno, G. D., Miesbach, W., Castaman, G., & Peyvandi, F. (2024) · Haematologica · doi:10.3324/haematol.2023.284622 · Cited by 11 · see citing works

The potential of gene delivery for the treatment of traumatic brain injury

Dooley, J., Hughes, J. G., Needham, E., Palios, K. A., & Liston, A. (2024) · Journal of Neuroinflammation · doi:10.1186/s12974-024-03156-x · Cited by 9 · see citing works

Non-clinical safety considerations on genome editing using the CRISPR/Cas system

Toofan, P., Singh, M., Brööks, A. G., & McLuckie, K. (2025) · Genes & Diseases · doi:10.1016/j.gendis.2025.101785 · Cited by 8 · see citing works

VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF

Carrell, E. M., Chen, Y. H., Ranum, P. T., Coffin, S. L., Singh, L. N., Tecedor, L., Keiser, M. S., Hudry, E., Hyman, B. T., & Davidson, B. L. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.07.016 · Cited by 7 · see citing works

AAV8 vector induced gliosis following neuronal transgene expression

McLeod, F., McDermott, E., Mak, S., Walsh, D. A., Turnbull, M., LeBeau, F. E. N., Jackson, A., Trevelyan, A. J., & Clowry, G. J. (2024) · Frontiers in Neuroscience · doi:10.3389/fnins.2024.1287228 · Cited by 7 · see citing works

A dose escalation and safety study of AAVrh10-mediated Schwann cell-targeted gene therapy for CMT1X

Christou, M., Sargiannidou, I., Papacharalambous, R., Richter, J., Tryfonos, C., Christodoulou, C., Kagiava, A., & Kleopa, K. A. (2025) · Neurotherapeutics · doi:10.1016/j.neurot.2025.e00568 · Cited by 6 · see citing works

Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates

Okai, T., Sato, S., Yasuno, H., Nakayama, M., Yamamoto, S., Sjöqvist, S., Otake, K., Nakashima, M., Deshpande, M., Galbreath, E. J., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101593 · Cited by 6 · see citing works

A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP

Benatti, H. R., Anagnostakou, V., Taghian, T., Hall, E. F., Nath, S., Heilman, C. B., Beneduce, B. M., Leporati, A. M., Raskett, C. M., Epshtein, M., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.08.024 · Cited by 6 · see citing works

Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II

Catalano, F., Vlaar, E. C., Katsavelis, D., Dammou, Z., Huizer, T. F., van den Bosch, J. C., Hoogeveen‐Westerveld, M., van den Hout, J. M. P., Oussoren, E., Ruijter, G. J. G., et al. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101149 · Cited by 6 · see citing works

Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following AAV gene therapy

Grubor, B., Henry, K. L., Chan, S. J., Sheehan, M. J., Shah, A., Pellerin, A., Bai, J., Nadella, P., Bommegowda, S., Cullen, P. F., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101643 · Cited by 5 · see citing works

Restoration of peripheral neuropathy in Fabry mice via intrathecal administration of an adeno-associated virus vector encoding mGLA cDNA.

Higuchi, T., Shimada, Y., Takahashi, Y., Kato, F., Ohashi, T., & Kobayashi, H. (2024) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2024.108545 · Cited by 5 · see citing works

Absence of functional deficits in rats following systemic administration of an AAV9 vector despite moderate peripheral nerve and dorsal root ganglia findings: A clinically silent peripheral neuropathy

Tyszkiewicz, C. M., Hwang, S., DaSilva, J. K., Kovi, R. C., Fader, K. A., Sirivelu, M. P., Liu, J., Somps, C. J., Cook, J. C., Liu, C., et al. (2024) · NeuroToxicology · doi:10.1016/j.neuro.2024.02.001 · Cited by 5 · see citing works

A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice

Krause, F., Schmidtke, K., Vasconcelos, M., Schmidt, D., Cansiz, B., Theisen, F., Mark, M. D., & Rybarski, M. O. (2024) · Gene Therapy · doi:10.1038/s41434-024-00447-z · Cited by 3 · see citing works

Dynein-regulated brain transduction of AAV.CAP-B10 via cerebral lateral ventricle enhances hippocampal function after traumatic brain injury through Ngf gene delivery in mice

Wang, J.-C., Ding, S., He, Z.-H., Huang, J., Weng, W., Gao, Y.-W., Jiang, J., Lin, Y., & Feng, J. (2025) · Experimental Neurology · doi:10.1016/j.expneurol.2025.115285 · Cited by 3 · see citing works

Potent and selective repression of SCN9A by engineered zinc finger repressors for the treatment of neuropathic painPreprint

Samie, M., Parman, T., Jalan, M., Lee, J., Dunn, P. J., Eshleman, J. A., Vidales, D. B., Holter, J., Jones, B., Pan, Y., et al. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.09.06.609976 · Cited by 3 · see citing works

Reader Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial

Palma, J. (2022) · Neurology · doi:10.1212/wnl.0000000000201004 · Cited by 2 · see citing works

Engineered zinc finger repressors induce a prolonged and selective repression of SCN9A in nociceptors of nonhuman primates

Samie, M., Parman, T., Jalan, M., Lee, J., Dunn, P. J., Eshleman, J. A., Vidales, D. B., Kennard, K., Holter, J., Jones, B., et al. (2026) · Science Translational Medicine · doi:10.1126/scitranslmed.adu0217 · Cited by 5 · see citing works

The dose makes the poison: Mechanisms of cytotoxicity of high-dose AAV vectors

Flotte, T. R. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201746 · Cited by 1 · see citing works

Intra-CNS AAV9-GBA1 delivery yields species and route of administration differences in safety and transgene expression

Amaral, A. C. S., Grubor, B., Gianni, D., Koetzner, L., Abraham, N., Bourque, S., Brown, D. C., Chen, Y., Chicoine, K.-E., Clarner, P., et al. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201779 · Cited by 1 · see citing works

Huntingtin lowering therapeutics

D, N. A. M., Sena‐Esteves, M., Khvorova, A., DiFiglia, M., & Brodsky, M. (2024) · Elsevier eBooks · doi:10.1016/b978-0-323-95672-7.00004-2 · Cited by 1 · see citing works

Biomarker and Bioanalytical Readouts for the Development of AAV Gene Therapy

Lu, Y., & Lembke, W. (2024) · Wiley eBooks · doi:10.1002/9781119852810.ch3 · Cited by 1 · see citing works

Gene Therapy for Sjögren’s Syndrome

Yin, H., & Chiorini, J. A. (2021) · Springer eBooks · doi:10.1007/978-3-030-72029-2_12

Gene Therapy and Gene Editing

Assaf, B. T., Harper, C. G., & Phillips, J. A. (2023) · Elsevier eBooks · doi:10.1016/b978-0-12-821047-5.00034-8

A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice.Preprint

Mark, M. D., Krause, F., Schmidtke, K., Vasconcelos, M., Schmidt, D., Cansiz, B., Theisen, F., & Rybarski, M. O. (2023) · Research Square · doi:10.21203/rs.3.rs-3459293/v1

Intrathecal Delivery of Viral Vector-Mediated Gene Therapy

Keifer, O. P. (2023) · Springer eBooks · doi:10.1007/978-3-031-39558-1_16

Current Regulatory Landscape for Gene Therapy Product Development and the Role of Biomarkers

Salazar‐Fontana, L. I., & Havert, M. (2024) · doi:10.1002/9781119852810.ch18

Pre-clinical evaluation of a gene therapy candidate for SOD1 -ALS shows improved survival and signs of inflammation in the CNS of treated micePreprint

Pezet, S., Hua, J., Marais, T., Delamare, M., Elouej, S., Lemos, J. P., Castiglione, A., Astord, S., Cohen-Tannoudji, M., Pêche, G. A., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.10.16.682744

Translational importance of non-human primates for the study of the immune response in gene therapy procedures involving in vivo administration of rAAV vectors

Gaston, C., Fayard, A., Dufour, N., Hantraye, P., Badin, R. A., & Bemelmans, A. (2025) · Revue de primatologie · doi:10.4000/15jub

Deciphering the role and contribution of AAV immune responses to DRG toxicity

Hordeaux, J., & Chan, Y. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201675

Adeno-associated virus-induced neurotoxicity is prevented by CpG depletion

Dunn, C. E., Carvajal, B. V., Ma, J., Castaneda-Hernandez, G., Nguyen, A. V., Jung, C., Suriano, C. M., & Boulanger, L. M. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201703

A dose-escalation and safety gene therapy study in a model of CMT4C neuropathy

Georgiou, E., Kagiava, A., Hentschel, A., Sargiannidou, I., Papacharalampous, R., Stavrou, M., Tryfonos, C., Richter, J., Roos, A., & Kleopa, K. A. (2026) · Gene Therapy · doi:10.1038/s41434-026-00616-2

Inner-ear delivery of AAV2-retro robustly labels lateral olivocochlear efferents but results in off-target transfection in the contralateral cochlea and the brain

Khalil, M., Bisignani, M., & Kandler, K. (2026) · Frontiers in Genome Editing · doi:10.3389/fgeed.2026.1946992

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques

Hordeaux, J., Hinderer, C., Goode, T., Katz, N., Buza, E. L., Bell, P., Calcedo, R., Richman, L. K., & Wilson, J. M. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2018.06.003 · Cited by 105 · see citing works

Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys

Hordeaux, J., Hinderer, C. J., Buza, E. L., Louboutin, J., Jahan, T., Bell, P. L., Chichester, J. A., Tarantal, A. F., & Wilson, J. M. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.012 · Cited by 76 · see citing works

Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome

Laoharawee, K., Podetz-Pedersen, K. M., Nguyen, T. T. T. N., Singh, S. M., Smith, M. C., Belur, L. R., Low, W. C., Kozarsky, K., & McIvor, R. S. (2023) · Molecular Genetics and Metabolism Reports · doi:10.1016/j.ymgmr.2023.100956 · Cited by 6 · see citing works

In vitro correction of iduronate-2-sulfatase deficiency by adenovirus-mediated gene transfer

di Francesco, C., Cracco, C., Tomanin, R., Picci, L., Ventura, L., Zacchello, F., Natale, P. D., Anson, D. S., Hopwood, J. J., Graham, F., et al. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300411 · Cited by 20 · 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