Viral vectors for therapy of neurologic diseases

Choudhury, Sourav R.; Hudry, Eloise; Maguire, Casey A.; Sena-Esteves, Miguel; Breakefield, Xandra O.; Grandi, Paola

doi:10.1016/j.neuropharm.2016.02.013

Cited by 190Virus-based gene therapy researchRNA Interference and Gene DeliveryBarrier Structure and Function Studies

Viral vectors for therapy of neurologic diseases

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Choudhury, Sourav R., Hudry, Eloise, Maguire, Casey A., Sena-Esteves, Miguel, Breakefield, Xandra O., Grandi, Paola·2017·Neuropharmacology

DOI 10.1016/j.neuropharm.2016.02.013PMC PMC5929167

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Abstract

Neurological disorders - disorders of the brain, spine and associated nerves - are a leading contributor to global disease burden with a shockingly large associated economic cost. Various treatment approaches - pharmaceutical medication, device-based therapy, physiotherapy, surgical intervention, among others - have been explored to alleviate the resulting extent of human suffering. In recent years, gene therapy using viral vectors - encoding a therapeutic gene or inhibitory RNA into a "gutted" viral capsid and supplying it to the nervous system - has emerged as a clinically viable option for therapy of brain disorders. In this Review, we provide an overview of the current state and advances in the field of viral vector-mediated gene therapy for neurological disorders. Vector tools and delivery methods have evolved considerably over recent years, with the goal of providing greater and safer genetic access to the central nervous system. Better etiological understanding of brain disorders has concurrently led to identification of improved therapeutic targets. We focus on the vector technology, as well as preclinical and clinical progress made thus far for brain cancer and various neurodegenerative and neurometabolic disorders, and point out the challenges and limitations that accompany this new medical modality. Finally, we explore the directions that neurological gene therapy is likely to evolve towards in the future. This article is part of the Special Issue entitled "Beyond small molecules for neurological disorders".

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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 [52]
Section 2 describes AAV vectors as close to ideal for CNS gene therapy, citing low toxicity and mild immunogenicity. But section 4.1 reports that clinical trials were less effective, with no significant improvement in one trial and only minor improvement in another. The paper supports a favorable safety profile, but not a generally excellent clinical efficacy profile. (LLM, conf=0.87)
Experimental — accuracy not yet measured
The AAV gene packaging size is limited to a maximum of 5.0 kb.
“Although the gene packaging size is limited (maximum 5.0 kb), typically the relevant gene coding sequences can be easily packaged for CNS delivery.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [52]
Sections 1 and 2 confirm that AAV packaging capacity is limited, but they give a maximum of about 4.5 kb, not 5.0 kb. Section 2 further specifies 4.5 kb for single-stranded vectors and 2.4 kb for self-complementary vectors. (LLM, conf=0.88)
Experimental — accuracy not yet measured
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 [52]
Section 4.1 identifies AAV9 and AAVrh10 as capsids used in more recent clinical trials. It does not identify AAV5 as commonly used in trials or establish that these three are the most commonly used serotypes; Section 3.1 discusses AAV5's cell-transduction pattern instead. The paper also says AAV2 was used in all first-generation trials. (LLM, conf=0.86)
Experimental — accuracy not yet measured
AAV vector-mediated gene therapy has become a preferred method for delivering therapeutic genes to the CNS.
“AAV vector-mediated gene therapy has become a preferred method for delivering therapeutic genes to the CNS.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [52]

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“In higher species such as pigs and non-human primates, the spread of gene transfer after IT injection is much wider, with multiple cell populations (including neurons) in the brain and brainstem being transduced at high efficiency (Federici et al., 2012; Samaranch et al., 2012; Gray et al., 2013).”
Background
A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson’s disease
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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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Stereotaxic gene delivery in the rodent brain
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A Phase 1 Trial of Oncolytic HSV-1, G207, Given in Combination With Radiation for Recurrent GBM Demonstrates Safety and Radiographic Responses
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Human gene targeting by viral vectors
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In Vivo Gene Therapy of Hemophilia B: Sustained Partial Correction in Factor IX-Deficient Dogs
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Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells
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Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency
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Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
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Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease
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Aberrant insulin signaling in Alzheimer's disease: current knowledge
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Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System
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Self-citation
Nerve Growth Factor Gene Therapy: Activation of Neuronal Responses in Alzheimer Disease
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Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
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Gene therapy for lysosomal storage diseases
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Microvesicle-associated AAV Vector as a Novel Gene Delivery System
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Self-citation
Atrophy of the Cholinergic Basal Forebrain Over the Adult Age Range and in Early Stages of Alzheimer's Disease
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Terminal N-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9
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Hot spots of retroviral integration in human CD34+ hematopoietic cells
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Effective delivery of large genes to the retina by dual AAV vectors
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An Oncolytic HSV-1 Mutant Expressing ICP34.5 under Control of a Nestin Promoter Increases Survival of Animals even when Symptomatic from a Brain Tumor
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A recombinant retrovirus encoding alkaline phosphatase confirms clonal boundary assignment in lineage analysis of murine retina.
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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
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Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene ( ASPA ) to the Human Brain
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Delivery of neurturin by AAV2 (CERE‐120)‐mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP‐treated monkeys
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Intracerebroventricular Viral Injection of the Neonatal Mouse Brain for Persistent and Widespread Neuronal Transduction
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Gene Therapy for Aromatic <scp>l</scp> -Amino Acid Decarboxylase Deficiency
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Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals
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Immune responses to AAV in a phase I study for Canavan disease
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Mammalian Endogenous Retroviruses
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Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome
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Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10
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A Single Injection of an Adeno-Associated Virus Vector into Nuclei with Divergent Connections Results in Widespread Vector Distribution in the Brain and Global Correction of a Neurogenetic Disease
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Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
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TALE nucleases: tailored genome engineering made easy
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Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS
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Capsid Serotype and Timing of Injection Determines AAV Transduction in the Neonatal Mice Brain
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Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease
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Efficient Neuronal Gene Transfer with AAV8 Leads to Neurotoxic Levels of Tau or Green Fluorescent Proteins
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Transport and egress of herpes simplex virus in neurons
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Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice
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Adeno-associated Virus Gene Therapy With Cholesterol 24-Hydroxylase Reduces the Amyloid Pathology Before or After the Onset of Amyloid Plaques in Mouse Models of Alzheimer's Disease
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Self-citation
Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery
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Large‐scale molecular characterization of adeno‐associated virus vector integration in mouse liver
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The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice
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Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo
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Gene Transfer of Human Apoe Isoforms Results in Differential Modulation of Amyloid Deposition and Neurotoxicity in Mouse Brain
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Self-citation
shRNA targeting α-synuclein prevents neurodegeneration in a Parkinson’s disease model
Zharikov, A., Cannon, J. R., Tapias, V., Bai, Q., Horowitz, M. P., Shah, V., Ayadi, A. E., Hastings, T. G., Greenamyre, J. T., & Burton, E. A. (2015) · Journal of Clinical Investigation · doi:10.1172/jci64502 · Cited by 184 · see citing works
Recombinant Semliki Forest virus and Sindbis virus efficiently infect neurons in hippocampal slice cultures
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Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain
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Robust ZFN-mediated genome editing in adult hemophilic mice
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Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse
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Early BDNF Treatment Ameliorates Cell Loss in the Entorhinal Cortex of APP Transgenic Mice
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Large-Scale Analysis of Adeno-Associated Virus Vector Integration Sites in Normal Human Cells
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Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs
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Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy
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Facile methods for generating human somatic cell gene knockouts using recombinant adeno-associated viruses
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Cytoplasmic Trafficking, Endosomal Escape, and Perinuclear Accumulation of Adeno-Associated Virus Type 2 Particles Are Facilitated by Microtubule Network
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Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors
Brooks, A. I., Stein, C. S., Hughes, S. M., Heth, J., McCray, P. B., Sauter, S. L., Johnston, J., Cory‐Slechta, D. A., Federoff, H. J., & Davidson, B. L. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.082011999 · Cited by 160 · see citing works
AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction
Samaranch, L., Sebastián, W. S., Kells, A. P., Salegio, E. A., Heller, G., Bringas, J. R., Pivirotto, P., DeArmond, S. J., Forsayeth, J., & Bankiewicz, K. S. (2013) · Molecular Therapy · doi:10.1038/mt.2013.266 · Cited by 157 · see citing works
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
Striatal Delivery of Neurturin by CERE-120, an AAV2 Vector for the Treatment of Dopaminergic Neuron Degeneration in Parkinson's Disease
Gasmi, M., Herzog, C. D., Brandon, E. P., Cunningham, J. J., Ramirez, G. A., Ketchum, E. T., & Bartus, R. T. (2006) · Molecular Therapy · doi:10.1038/sj.mt.6300010 · Cited by 153 · see citing works
Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery in vivo
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Self-citation
Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy
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The Status ofRPE65Gene Therapy Trials: Safety and EfficacyPreprint
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Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids
Castle, M. J., Turunen, H. T., Vandenberghe, L. H., & Wolfe, J. H. (2016) · Methods in Molecular Biology · doi:10.1007/978-1-4939-3271-9_10 · Cited by 151 · cited 2× · see citing works
“Recent efforts at capsid reengineering (Castle et al., 2016) have led to the generation of two AAV capsid variants, AAV-AS (Choudhury et al.”
Background
Effective gene therapy in an authentic model of Tay-Sachs-related diseases
Cachón-González, M. B., Wang, S. Z., Lynch, A. G., Ziegler, R. J., Cheng, S. H., & Cox, T. M. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0603765103 · Cited by 148 · see citing works
Insulin‐like growth factor 2 reverses memory and synaptic deficits in APP transgenic mice
Pascual‐Lucas, M., da Silva, S. V., Scala, M. D., García, C., González‐Aseguinolaza, G., Mulle, C., Alberini, C. M., Cuadrado‐Tejedor, M., & García‐Osta, A. (2014) · EMBO Molecular Medicine · doi:10.15252/emmm.201404228 · Cited by 147 · see citing works
Exosome-associated AAV vector as a robust and convenient neuroscience tool
Hudry, E., Martin, C., Gandhi, S., György, B., Scheffer, D. I., Mu, D., Merkel, S. F., Mingozzi, F., Fitzpatrick, Z., Dimant, H., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.11 · Cited by 146 · see citing works
Self-citation
Transfer and expression of the lacZ gene in rat brain neurons mediated by herpes simplex virus mutants.
Chiocca, E. A., Choi, B. B., Cai, W. Z., et al. (1990) · PubMed · Cited by 144 · see citing works
Gene therapy in the CNS
Costantini, L. C., Bakowska, J. C., Breakefield, X. O., & Ole Isacson, A. (2000) · Gene Therapy · doi:10.1038/sj.gt.3301119 · Cited by 142 · see citing works
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Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
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Authors: Choudhury, S. R., Hudry, E., Maguire, C. A., Sena-Esteves, M., Breakefield, X. O., & Grandi, P.
Affiliations: University of Massachusetts Chan Medical School (US)
Harvard University (US)
UPMC Hillman Cancer Center (US)
Corresponding author: Breakefield, Xandra O.
ORCID: Choudhury, Sourav R.
Maguire, Casey A.
Breakefield, Xandra O.
Grandi, Paola
Venue: Neuropharmacology
Publisher: Elsevier BV
Published: 2017-07
Volume: 120
Pages: 63-80
ISSN: 0028-3908 (ISSN-L)
DOI: 10.1016/j.neuropharm.2016.02.013
PMC: PMC5929167
OpenAlex: W2280490150
Cited by: 190
References: 234
Citations by year: 2026: 7 · 2025: 19 · 2024: 14 · 2023: 20 · 2022: 20 · 2021: 24 · 2020: 23 · 2019: 15 · 2018: 21 · 2017: 17 · 2016: 10
FWCI: 2.81
Type: journal article
Language: en
Open access: bronze
doi.org
Licence: www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
www.elsevier.com/open-access/userlicense/1.0/ (am, 344 d)
doi.org/10.15223/policy-017 (stm-asf)
doi.org/10.15223/policy-037 (stm-asf)
doi.org/10.15223/policy-012 (stm-asf)
doi.org/10.15223/policy-029 (stm-asf)
doi.org/10.15223/policy-004 (stm-asf)
Locations: Neuropharmacology (acceptedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Funders: Foundation for the National Institutes of Health (U19 CA179563, NIH, NS066310, HD060576)
David O. McKay School of Education, Brigham Young University
American Brain Tumor Association
NIH Office of the Director
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: viral vectors, gene therapy, neurological disorders, central nervous system delivery, brain cancer, neurodegenerative diseases, neurometabolic disorders, antisense RNA, therapeutic target
MeSH terms: Animals, Brain Neoplasms, Genetic Vectors, Humans, Nervous System Diseases, Genetic Therapy, Gene Transfer Techniques

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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

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Viral vectors for therapy of neurologic diseases

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