Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Hocquemiller, Michaël; Giersch, Laura; Audrain, Mickael; Parker, Samantha; Cartier, Nathalie

doi:10.1089/hum.2016.087

Cited by 291Virus-based gene therapy researchRNA Interference and Gene DeliveryBarrier Structure and Function Studies

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

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Hocquemiller, Michaël, Giersch, Laura, Audrain, Mickael, Parker, Samantha, Cartier, Nathalie·2016·Human Gene Therapy

DOI 10.1089/hum.2016.087PMC PMC4960479

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Abstract

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and efficiency in transducing a wide range of cell types. The purpose of this review is to describe the most notable advancements in preclinical and clinical research on AAV-based CNS gene therapy and to discuss prospects for future development based on a new generation of vectors and delivery.

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The AAV serotypes most commonly used for clinical trials to date are AAV5, AAV9, and AAVrh.10.
“Of these AAV serotypes, the most commonly used for clinical trials to date are AAV5, AAV9, and AAVrh.10.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [56]
The Lysosomal storage disorders section describes clinical trials using AAV5, AAV9, and AAVrh.10. But neither that section nor the Introduction establishes that these are the most commonly used serotypes in clinical trials; the Introduction also describes AAV2 trials. The source supports their clinical use, not the claimed ranking. (LLM, conf=0.87)
Experimental — accuracy not yet measured
AAV vector administration to the CNS has an excellent safety and efficacy profile.
“AAV vector administration to the CNS has an excellent safety and efficacy profile.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [56]

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Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates
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Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice
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Feasibility and Safety of Systemic rAAV9-h NAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates
Murrey, D. A., Naughton, B. J., Duncan, F. J., Meadows, A. S., Bobo, T. A., Campbell, K., Bremer, W. G., Walker, C. M., Goodchild, L., Bolon, B. N., et al. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.208 · Cited by 88 · see citing works
Clinically Relevant Effects of Convection-Enhanced Delivery of AAV2-GDNF on the Dopaminergic Nigrostriatal Pathway in Aged Rhesus Monkeys
Johnston, L. C., Eberling, J. L., Pivirotto, P., Hadaczek, P., Federoff, H. J., Forsayeth, J., & Bankiewicz, K. S. (2009) · Human Gene Therapy · doi:10.1089/hum.2008.137 · Cited by 88 · 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
Differential Transduction Following Basal Ganglia Administration of Distinct Pseudotyped AAV Capsid Serotypes in Nonhuman Primates
Dodiya, H. B., Bjorklund, T., Stansell III, J., Mandel, R. J., Kirik, D., & Kordower, J. H. (2010) · Molecular Therapy · doi:10.1038/mt.2009.216 · Cited by 86 · 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 · see citing works
NGF gene transfer to intrinsic basal forebrain neurons increases cholinergic cell size and protects from age-related, spatial memory deficits in middle-aged rats
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AAV2-mediated gene delivery to monkey putamen: Evaluation of an infusion device and delivery parameters
Sanftner, L. M., Sommer, J. M., Suzuki, B. M., Smith, P. H., Vijay, S., Vargas, J. A., Forsayeth, J., Cunningham, J., Bankiewicz, K. S., Kao, H., et al. (2005) · Experimental Neurology · doi:10.1016/j.expneurol.2005.03.007 · Cited by 83 · 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
Virus-Mediated Knockdown of Nav1.3 in Dorsal Root Ganglia of STZ-Induced Diabetic Rats Alleviates Tactile Allodynia
Tan, A. M., Samad, O. A., Dib‐Hajj, S. D., & Waxman, S. G. (2015) · Molecular Medicine · doi:10.2119/molmed.2015.00063 · Cited by 80 · see citing works
Regulation of alpha-secretase ADAM10 expression and activity
Endres, K., & Fahrenholz, F. (2011) · Experimental Brain Research · doi:10.1007/s00221-011-2885-7 · Cited by 79 · see citing works
Complete Correction of Enzymatic Deficiency and Neurochemistry in the GM1-gangliosidosis Mouse Brain by Neonatal Adeno-associated Virus–mediated Gene Delivery
Broekman, M. L. D., Baek, R. C., Comer, L. A., Fernandez, J. L., Seyfried, T. N., & Sena‐Esteves, M. (2006) · Molecular Therapy · doi:10.1038/sj.mt.6300004 · Cited by 79 · see citing works
Widespread and Efficient Transduction of Spinal Cord and Brain Following Neonatal AAV Injection and Potential Disease Modifying Effect in ALS Mice
Ayers, J. I., Fromholt, S., Sinyavskaya, O., Siemienski, Z. B., Rosario, A. M., Li, A., Crosby, K. W., Cruz, P. E., DiNunno, N., Janus, C., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.180 · Cited by 77 · see citing works
Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS
Franz, C. K., Federici, T., Yang, J., Backus, C., Oh, S. S., Teng, Q., Carlton, E. F., Bishop, K. M., Gasmi, M., Bartus, R. T., et al. (2008) · Neurobiology of Disease · doi:10.1016/j.nbd.2008.12.003 · Cited by 77 · see citing works
Therapeutic Liabilities of in Vivo Viral Vector Tropism: Adeno-Associated Virus Vectors, NMDAR1 Antisense, and Focal Seizure Sensitivity
Haberman, R. P., Criswell, H. E., Snowdy, S., Ming, Z., Breese, G. R., Samulski, R. J., & McCown, T. J. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0701 · Cited by 75 · see citing works
Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
Wolf, D. A., Lenander, A. W., Nan, Z., Belur, L. R., Whitley, C. B., Gupta, P., Low, W. C., & McIvor, R. S. (2011) · Neurobiology of Disease · doi:10.1016/j.nbd.2011.02.015 · Cited by 73 · see citing works
AAV.shRNA-mediated downregulation of ROCK2 attenuates degeneration of dopaminergic neurons in toxin-induced models of Parkinson's disease in vitro and in vivo
Saal, K., Koch, J. C., Tatenhorst, L., Szegő, É. M., Ribas, V. T., Michel, U., Bähr, M., Tönges, L., & Lingor, P. (2014) · Neurobiology of Disease · doi:10.1016/j.nbd.2014.09.013 · Cited by 72 · see citing works
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate
Colle, M.-A., Piguet, F., Bertrand, L., Raoul, S., Bièche, I., Dubreil, L., Sloothaak, D., Bouquet, C., Moullier, P., Aubourg, P. R., et al. (2009) · Human Molecular Genetics · doi:10.1093/hmg/ddp475 · Cited by 72 · see citing works
Self-citation
Intrajugular Vein Delivery of AAV9-RNAi Prevents Neuropathological Changes and Weight Loss in Huntington's Disease Mice
Dufour, B. D., Smith, C. A., Clark, R. L., Walker, T. R., & McBride, J. L. (2014) · Molecular Therapy · doi:10.1038/mt.2013.289 · Cited by 71 · see citing works
Mir‐142‐3p target sequences reduce transgene‐directed immunogenicity following intramuscular adeno‐associated virus 1 vector‐mediated gene delivery
Majowicz, A., Maczuga, P., Kwikkers, K. L., van der Marel, S., van Logtenstein, R., Petry, H., van Deventer, S. J. H., Konstantinova, P. S., & Ferreira, V. (2013) · The Journal of Gene Medicine · doi:10.1002/jgm.2712 · Cited by 69 · see citing works
Anticonvulsant effects and behavioural outcomes of rAAV serotype 1 vector-mediated neuropeptide Y overexpression in rat hippocampus
Noè, F. M., Vaghi, V., Balducci, C., Fitzsimons, H. L., Bland, R. J., Zardoni, D., Sperk, G., Carli, M., During, M. J., & Vezzani, A. (2010) · Gene Therapy · doi:10.1038/gt.2010.23 · Cited by 69 · see citing works
Hippocampal NPY gene transfer attenuates seizures without affecting epilepsy-induced impairment of LTP
Sørensen, A. T., Nikitidou, L., Ledri, M., Lin, E.-J. D., During, M. J., Kanter‐Schlifke, I., & Kokaia, M. (2008) · Experimental Neurology · doi:10.1016/j.expneurol.2008.10.015 · Cited by 69 · see citing works
AAV1/2-mediated CNS Gene Delivery of Dominant-negative CCL2 Mutant Suppresses Gliosis, β-amyloidosis, and Learning Impairment of APP/PS1 Mice
Kiyota, T., Yamamoto, M., Schroder, B., Jacobsen, M. T., Swan, R. J., Lambert, M. P., Klein, W. L., Gendelman, H. E., Ransohoff, R. M., & Ikezu, T. (2009) · Molecular Therapy · doi:10.1038/mt.2009.44 · Cited by 68 · see citing works
Sustained relief of neuropathic pain by AAV-targeted expression of CBD3 peptide in rat dorsal root ganglion
Fischer, G. J., Pan, B., Vilceanu, D., Hogan, Q. H., & Yu, H. (2013) · Gene Therapy · doi:10.1038/gt.2013.56 · Cited by 68 · see citing works
Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse
Passini, M. A., Bu, J., Fidler, J. A., Ziegler, R. J., Foley, J. W., Dodge, J. C., Yang, W. W., Clarke, J. L., Taksir, T. V., Griffiths, D. A., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0703509104 · Cited by 67 · see citing works
Sustained Normalization of Neurological Disease after Intracranial Gene Therapy in a Feline Model
McCurdy, V. J., Johnson, A. K., Gray‐Edwards, H. L., Randle, A. N., Brunson, B. L., Morrison, N. E., Salibi, N., Johnson, J. A., Hwang, M., Beyers, R. J., et al. (2014) · Science Translational Medicine · doi:10.1126/scitranslmed.3007733 · Cited by 67 · see citing works
An Anti-Neuroinflammatory That Targets Dysregulated Glia Enhances the Efficacy of CNS-Directed Gene Therapy in Murine Infantile Neuronal Ceroid Lipofuscinosis
Macauley, S. L., Wong, A. M. S., Shyng, C., Augner, D. P., Dearborn, J. T., Pearse, Y., Roberts, M. S., Fowler, S. C., Cooper, J. D., Watterson, D. M., et al. (2014) · Journal of Neuroscience · doi:10.1523/jneurosci.2518-14.2014 · Cited by 66 · see citing works
SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice
Dirren, E., Aebischer, J., Rochat, C., Towne, C. L., Schneider, B. L., & Aebischer, P. (2015) · Annals of Clinical and Translational Neurology · doi:10.1002/acn3.162 · Cited by 65 · see citing works
Adeno-associated virus-mediated expression and constitutive secretion of NPY or NPY13-36 suppresses seizure activity in vivo
Foti, S., Haberman, R. P., Samulski, R. J., & McCown, T. J. (2007) · Gene Therapy · doi:10.1038/sj.gt.3303013 · Cited by 64 · see citing works
Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9‐ADAR2 delivery to motor neurons
Yamashita, T., Chai, H., Teramoto, S., Tsuji, S., Shimazaki, K., Muramatsu, S., & Kwak, S. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201302935 · Cited by 64 · see citing works
AAV–BDNF mediated attenuation of quinolinic acid-induced neuropathology and motor function impairment
Kells, A., Henry, R. A., & Connor, B. J. (2008) · Gene Therapy · doi:10.1038/gt.2008.23 · Cited by 63 · see citing works
Enhanced Secretion and Uptake of β-Glucuronidase Improves Adeno-associated Viral-Mediated Gene Therapy of Mucopolysaccharidosis Type VII Mice
Elliger, S. S., Elliger, C. A., Chen, L., & Watson, G. L. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0594 · Cited by 63 · see citing works
Sustained Mobilization of Endogenous Neural Progenitors Delays Disease Progression in a Transgenic Model of Huntington’s Disease
Benraiss, A., Toner, M. J., Xu, Q., Bruel‐Jungerman, E., Rogers, E., Wang, F., Economides, A. N., Davidson, B. L., Kageyama, R., Nedergaard, M., et al. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.04.014 · Cited by 63 · see citing works
Selective overexpression of excitatory amino acid transporter 2 (EAAT2) in astrocytes enhances neuroprotection from moderate but not severe hypoxia–ischemia
Weller, M. L., Stone, I. M., Goss, A., Rau, T., Rova, C. R. L., & Poulsen, D. J. (2008) · Neuroscience · doi:10.1016/j.neuroscience.2008.05.059 · Cited by 62 · see citing works
Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB
Heldermon, C. D., Ohlemiller, K. K., Herzog, E. D., Vogler, C. A., Qin, E. Y., Wozniak, D. F., Tan, Y., Orrock, J. L., & Sands, M. S. (2010) · Molecular Therapy · doi:10.1038/mt.2010.17 · Cited by 62 · see citing works
Strategies to circumvent humoral immunity to adeno-associated viral vectors
Tse, L. V., Moller-Tank, S., & Asokan, A. (2015) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2015.1035645 · Cited by 61 · see citing works
Long-Term Safety of Patients with Parkinson's Disease Receiving rAAV2-Neurturin (CERE-120) Gene Transfer
Marks, W. J., Baumann, T. L., Bartus, R. T., & the CERE-120 Study Group (2015) · Human Gene Therapy · doi:10.1089/hum.2015.134 · Cited by 61 · 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
Intrathecal AAV Serotype 9-mediated Delivery of shRNA Against TRPV1 Attenuates Thermal Hyperalgesia in a Mouse Model of Peripheral Nerve Injury
Hirai, T., Enomoto, M., Kaburagi, H., Sotome, S., Yoshida‐Tanaka, K., Ukegawa, M., Kuwahara, H., Yamamoto, M., Tajiri, M., Miyata, H., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.247 · Cited by 58 · see citing works
Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model
Nizzardo, M., Simone, C., Rizzo, F., Salani, S., Dametti, S., Rinchetti, P., Bo, R. D., Foust, K. D., Kaspar, B. K., Bresolin, N., et al. (2015) · Science Advances · doi:10.1126/sciadv.1500078 · Cited by 58 · see citing works
Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates
Rosenberg, J. B., Sondhi, D., Rubin, D. G., Monette, S., Chen, A., Cram, S., De, B. P., Kaminsky, S. M., Sevin, C., Aubourg, P., et al. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.239 · Cited by 58 · see citing works
Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease
Ramaswamy, S., McBride, J. L., Han, I., Berry‐Kravis, E., Zhou, L., Herzog, C. D., Gasmi, M., Bartus, R. T., & Kordower, J. H. (2008) · Neurobiology of Disease · doi:10.1016/j.nbd.2008.12.005 · Cited by 57 · see citing works
AAV-Mediated Gene Delivery in a Feline Model of Sandhoff Disease Corrects Lysosomal Storage in the Central Nervous System
Rockwell, H. E., McCurdy, V. J., Eaton, S. C., Wilson, D. U., Johnson, A. K., Randle, A. N., Bradbury, A. M., Gray‐Edwards, H. L., Baker, H. J., Hudson, J. A., et al. (2015) · ASN NEURO · doi:10.1177/1759091415569908 · Cited by 56 · see citing works
Adeno-Associated Viral Vector Mediated Gene Delivery of Endothelin Converting Enzyme Reduces Aβ Deposits in APP+PS-1 Transgenic Mice
Carty, N. C., Nash, K., Lee, D. C., Mercer, M., Gottschall, P. E., Meyers, C., Muzyczka, N., Gordon, M. N., & Morgan, D. (2008) · Molecular Therapy · doi:10.1038/mt.2008.148 · Cited by 55 · see citing works
A QUANTITATIVE STUDY OF CELL PROLIFERATION IN EPENDYMA AND CHOROID PLEXUS IN THE POSTNATAL RAT BRAIN
CHAUHAN, A. N., & Lewis, P. D. (1979) · Neuropathology and Applied Neurobiology · doi:10.1111/j.1365-2990.1979.tb00629.x · Cited by 55 · see citing works
Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease
Doerfler, P. A., Todd, A. G., Clément, N., Falk, D. J., Nayak, S., Herzog, R. W., & Byrne, B. J. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.103 · Cited by 54 · see citing works
Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy
Ribera, A., Haurigot, V., Garcia, M., Marcó, S., Motas, S., Villacampa, P., Maggioni, L., León, X., Molas, M., Sánchez, V., et al. (2014) · Human Molecular Genetics · doi:10.1093/hmg/ddu727 · Cited by 53 · see citing works
Restoration of Cytoskeleton Homeostasis After Gigaxonin Gene Transfer for Giant Axonal Neuropathy
Mussche, S., Devreese, B. V., Kalburgi, S. N., Bachaboina, L., Fox, J. C., Shih, H.-J. S., Coster, R. V., Samulski, R. J., & Gray, S. J. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.107 · Cited by 52 · see citing works
Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features
Rafi, M. A., Rao, H. Z., Luzi, P., Luddi, A., Curtis, M. T., & Wenger, D. A. (2014) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2014.12.300 · Cited by 47 · see citing works
Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain
Sargeant, T. J., Wang, S. S., Bradley, J. M., Smith, N. J. C., Raha, A. A., McNair, R., Ziegler, R. J., Cheng, S. H., Cox, T. M., & Cachón-González, M. B. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr364 · Cited by 47 · see citing works
Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type 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) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1413645111 · Cited by 46 · see citing works
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 · see citing works
Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and long-term enzyme replacement via the CSF
Yamazaki, Y., Hirai, Y., Miyake, K., & Shimada, T. (2014) · Scientific Reports · doi:10.1038/srep05506 · Cited by 46 · see citing works
Intracranial Injection of AAV Expressing NEP but Not IDE Reduces Amyloid Pathology in APP+PS1 Transgenic Mice
Carty, N., Nash, K. R., Brownlow, M. L., Cruite, D., Wilcock, D. M., Selenica, M. B., Lee, D. C., Gordon, M. N., & Morgan, D. (2013) · PLoS ONE · doi:10.1371/journal.pone.0059626 · Cited by 45 · see citing works
Efficacy of a Combined Intracerebral and Systemic Gene Delivery Approach for the Treatment of a Severe Lysosomal Storage Disorder
Spampanato, C., Leonibus, E. D., Dama, P., Gargiulo, A., Fraldi, A., Sorrentino, N. C., Russo, F., Nusco, E., Auricchio, A., Surace, E. M., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2010.299 · Cited by 44 · see citing works
Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120)
Herzog, C. D., Brown, L., Kruegel, B. R., Wilson, A., Tansey, M. G., Gage, F. H., Johnson, E. M., & Bartus, R. T. (2013) · Neurobiology of Disease · doi:10.1016/j.nbd.2013.04.011 · Cited by 44 · see citing works
Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene
Sondhi, D., Scott, E., Chen, A., Hackett, N. R., Wong, A. M. S., Kubiak, A., Nelvagal, H. R., Pearse, Y., Cotman, S. L., Cooper, J. D., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.253 · Cited by 44 · see citing works
A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome
Winner, L. K., Beard, H., Hassiotis, S., Lau, A. A., Luck, A. J., Hopwood, J. J., & Hemsley, K. M. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.170 · Cited by 43 · see citing works
IDS Crossing of the Blood-Brain Barrier Corrects CNS Defects in MPSII Mice
Polito, V. A., & Cosma, M. P. (2009) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2009.07.011 · Cited by 40 · see citing works

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Adeno-associated virus vector as a platform for gene therapy delivery
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Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
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AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset
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Gene Therapy Leaves a Vicious Cycle
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A walk through tau therapeutic strategies
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Possible strategies to cross the blood–brain barrier
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Erratum: Gene therapy for neurological disorders: progress and prospects
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AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease
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AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice
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A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism
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AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges
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Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy
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Blood–brain barrier: emerging trends on transport models and new-age strategies for therapeutics intervention against neurological disorders
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Adeno-Associated Viral Vectors in Neuroscience Research
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Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
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Variants of the adeno-associated virus serotype 9 with enhanced penetration of the blood–brain barrier in rodents and primates
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Therapeutic Potential of Secreted Amyloid Precursor Protein APPsα
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Viral and non-viral vectors in gene therapy: current state and clinical perspectives
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BBB opening with focused ultrasound in nonhuman primates and Parkinson’s disease patients: Targeted AAV vector delivery and PET imaging
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Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future
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Gene therapy for neurological disorders: challenges and recent advancements
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Comparison of Gene Delivery to the Kidney by Adenovirus, Adeno-Associated Virus, and Lentiviral Vectors After Intravenous and Direct Kidney Injections
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Co-encapsulation of Cas9 mRNA and guide RNA in polyplex micelles enables genome editing in mouse brain
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AAV ablates neurogenesis in the adult murine hippocampus
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Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord
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Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock
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BBB-crossing adeno-associated virus vector: An excellent gene delivery tool for CNS disease treatment
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RNAi mechanisms in Huntington’s disease therapy: siRNA versus shRNA
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Immune responses and immunosuppressive strategies for adeno-associated virus-based gene therapy for treatment of central nervous system disorders: current knowledge and approaches
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Liver induced transgene tolerance with AAV vectors
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Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
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Synthetic AAV/CRISPR vectors for blocking HIV‐1 expression in persistently infected astrocytes
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Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders
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Oligonucleotide therapeutics in neurodegenerative diseases
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Sexually Divergent Mortality and Partial Phenotypic Rescue After Gene Therapy in a Mouse Model of Dravet Syndrome
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Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases
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Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer
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Advances in the treatment of neuronal ceroid lipofuscinosis
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Authors: Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N.
Affiliations: Lysogene, Neuilly sur Seine, France.
Université Paris Descartes
Université Paris-Sud (FR)
Corresponding author: Hocquemiller, Michaël
Cartier, Nathalie
ORCID: Hocquemiller, Michaël
Parker, Samantha
Cartier, Nathalie
Venue: Human Gene Therapy
Publisher: SAGE Publications
Published: 2016-06-07
Volume: 27
Issue: 7
Pages: 478-496
ISSN: 1043-0342 (ISSN-L)
1557-7422
DOI: 10.1089/hum.2016.087
PMC: PMC4960479
OpenAlex: W2484015429
Cited by: 291
References: 220
Citations by year: 2026: 17 · 2025: 26 · 2024: 24 · 2023: 16 · 2022: 27 · 2021: 40 · 2020: 37 · 2019: 44 · 2018: 36 · 2017: 23 · 2016: 1
FWCI: 4.68
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: cc-by-nc
creativecommons.org/licenses/by-nc/4.0/ (vor)
journals.sagepub.com/page/policies/text-and-data-mining-license (tdm)
Locations: Human Gene Therapy (publishedVersion, cc-by-nc)
Europe PMC (PubMed Central) (publishedVersion, cc-by-nc)
PubMed Central (publishedVersion, cc-by-nc)
PubMed (publishedVersion)
Funders: Agence Nationale de la Recherche (13-BSV1-022-01)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
Keywords: adeno-associated virus, adeno-associated virus vectors, gene therapy, central nervous system disorders, gene delivery, vector design, clinical trials, preclinical research, neurodegenerative diseases
MeSH terms: Dependovirus, Animals, Central Nervous System Diseases, Genetic Vectors, Humans, Genetic Therapy

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