The advent of AAV9 expands applications for brain and spinal cord gene delivery

Dayton, Robert D; Wang, David B; Klein, Ronald L

doi:10.1517/14712598.2012.681463

Cited by 144Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

The advent of AAV9 expands applications for brain and spinal cord gene delivery

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Dayton, Robert D, Wang, David B, Klein, Ronald L·2012·Expert Opinion on Biological Therapy

DOI 10.1517/14712598.2012.681463PMC PMC3361729

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Abstract

INTRODUCTION: Straightforward studies compared adeno-associated virus (AAV) serotypes to determine the most appropriate one for robust expression in the CNS. AAV9 was efficient when directly injected into the brain, but more surprisingly, AAV9 produced global expression in the brain and spinal cord after a peripheral, systemic route of administration to neonatal mice. AREAS COVERED: Topics include AAV9 gene delivery from intraparenchymal, intravenous, intrathecal and intrauterine routes of administration, and related preclinical studies and disease models. Systemic AAV9 gene transfer yields remarkably consistent neuronal expression, though only in early development. AAV9 is versatile to study neuropathological proteins: microtubule-associated protein tau and transactive response DNA-binding protein 43 kDa (TDP-43). EXPERT OPINION: AAV9 will be more widely used based on current data, although other natural serotypes and recombineered vectors may also support or improve upon wide-scale expression. A peripheral-to-central gene delivery that can affect the entire CNS without having to inject the CNS is promising for basic functional experiments, and potentially for gene therapy. Systemic or intra-cerebrospinal fluid routes of AAV9 administration should be considered for spinal muscular atrophy, lysosomal storage diseases and amyotrophic lateral sclerosis, if more neuronal expression can be achieved in adults, or if glial expression can be exploited.

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Somatic transgenesis in the mouse, achieved by vector delivery at P0, is instrumental for disease modeling.
“However, somatic transgenesis in the mouse, achieved by vector delivery at P0, is instrumental for disease modeling (Dayton et al., 2012).”
From Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes (2016) · cites this paper as Dayton et al., 2012
Section 9 describes somatic vector-based gene transfer as a way to model neurodegenerative disease, and Section 3 describes neonatal mouse AAV9 delivery as useful for mammalian modeling. Neither passage specifies delivery at P0 or establishes that P0 delivery in mice is the disease-modeling approach described; the paper’s specific disease-model examples in Section 9 are largely in rats. Page numbers were not available in our copy. (LLM, conf=0.84)
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[28]Samaranch L Salegio EA San Sebastian W AAV9 transduction in the central nervous system of non-human primates Hum Gene Ther 2011 [Epub ahead of print] PMC3327605 22201473

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A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases
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“…et al, 2000; Cearley & Wolfe, 2006; de Backer et al, 2010; Markakis et al, 2010; Shen et al, 2013), and some of them (e.g., AAV9, AAVrh.8, and AAVrh.10) seem to be able to cross the mature BBB after systemic administration in adult animals (Foust et al, 2009; Dayton et al, 2012; Yang et al, 2014).”
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Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound
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“Advances in the AAV field have led to the selection of appropriate serotypes, exhibiting tropism for various organs (Pulicherla et al., 2011), and to the characterization of conditions that are important for infection efficiency (Gray et al., 2011; Dayton et al., 2012; Rapti et al., 2012).”
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“For instance, AAV serotype 9 was effective at transducing the neurons in the enteric autonomic nervous system (37,38).”
Background
Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection
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“However, compared with promising data obtained for another form of NCL, CLN2 disease (32, 33, 39), previous attempts to treat Ppt1 mice by targeting the known”
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“…system from the periphery, several groups have been successful in transducing AAV serotype 9 (AAV9) to foetal, neonatal and adult animal motor neurons in the spinal cord via intravenous administration (Benkhelifa ‐ Ziyyat et al, 2013; Dayton et al, 2012; Duque et al, 2009; Foust et al, 2009).”
Injection parameters and virus dependent choice of promoters to improve neuron targeting in the nonhuman primate brain
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“Other routes of administration are currently being evaluated for delivery of AAV vectors into different tissues by bypassing the circulating system, such as intrathecal delivery to target the CNS [29-31].”
Background
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“However, somatic transgenesis in the mouse, achieved by vector delivery at P0, is instrumental for disease modeling (Dayton et al., 2012).”
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Intramuscular Delivery of scAAV9-hIGF1 Prolongs Survival in the hSOD1G93A ALS Mouse Model via Upregulation of D-Amino Acid Oxidase
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Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment
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“More interestingly, rAAV9 has been shown to transduce neural tissue via retrograde transport after intramuscular delivery or even by crossing the blood–brain barrier itself (Foust et al., 2009; Bevan et al., 2011; Dayton et al., 2012; Dimattia et al., 2012; ElMallah et al., 2012).”
Background
Mouse Gender Influences Brain Transduction by Intravascularly Administered AAV9
Maguire, C. A., Crommentuijn, M. H. W., Mu, D., Hudry, E., Serrano‐Pozo, A., Hyman, B. T., & Tannous, B. A. (2013) · Molecular Therapy · doi:10.1038/mt.2013.95 · Cited by 47 · see citing works
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Selective Forelimb Impairment in Rats Expressing a Pathological TDP-43 25 kDa C-terminal Fragment to Mimic Amyotrophic Lateral Sclerosis
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“The advent of AAV9 expands applications for brain and spinal cord gene delivery.”
Method
Focused ultrasound–mediated brain genome editing
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“In addition, aside from the currently used stereotaxy-guided intraparenchymal delivery [170], new techniques for global brain transduction (by intravenous or intra-CSF injections) are very promising [171] [39].”
Background
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“Consistently, pTAU immunoreactivity was localized in GFAP + astrocytes in the hippocampal regions of control 3 x Tg mice, and astrocytic pTAU deposition was signi ﬁ cantly reduced in the mice treated with Nurr1 + Foxa2 (Fig.”
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“Compared with other viral vectors, such as adenovirus and lentivirus, AAV vector is highly safe, has a wide range of host cells, and can be used to express exogenous genes for a long time period in vivo([32-33]).”
Background
Neurological glycogen storage diseases and emerging therapeutics
Colpaert, M. G., Singh, P. K., Donohue, K. J., Pires, N. T., Fuller, D. D., Corti, M., Byrne, B. J., Sun, R. C., Kooi, C. W. V., & Gentry, M. S. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00446 · Cited by 23 · see citing works
“Among the AAV1-13 serotype, AAV9, AAV rh.8, and AAV rh.10, display high ef ﬁ ciency to cross the BBB after i.v. and or other administration routes [229 – 233].”
Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain
Zhang, X., Chai, Z., Lee Dobbins, A., Itano, M. S., Askew, C., Miao, Z., Niu, H., Samulski, R. J., & Li, C. (2022) · Biomaterials · doi:10.1016/j.biomaterials.2021.121340 · Cited by 23 · see citing works
Diagnosis and New Treatment Avenues in Spinal Muscular Atrophy
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Podocyte-specific expression of Cre recombinase promotes glomerular basement membrane thickening
Balkawade, R. S., Chen, C., Crowley, M. R., Crossman, D. K., Clapp, W. L., Verlander, J. W., & Marshall, C. B. (2019) · American Journal of Physiology-Renal Physiology · doi:10.1152/ajprenal.00359.2018 · Cited by 23 · see citing works
Recombinant adeno-associated virus vectors in the treatment of rare diseases
Hastie, E. L., & Samulski, R. J. (2015) · Expert Opinion on Orphan Drugs · doi:10.1517/21678707.2015.1039511 · Cited by 22 · see citing works
“Mutation to the aromatic L-amino acid decarboxylase (AADC) gene impairs biosynthesis of neurotransmitters such as serotonin or dopamine that are required for signaling between cells of the CNS.”
Background
Site Specific Modification of Adeno-Associated Virus Enables Both Fluorescent Imaging of Viral Particles and Characterization of the Capsid Interactome
Chandran, J. S., Sharp, P., Karyka, E., Aves-Cruzeiro, J. M. D. C., Coldicott, I., Castelli, L., Hautbergue, G., Collins, M., & Azzouz, M. (2017) · Scientific Reports · doi:10.1038/s41598-017-15255-2 · Cited by 17 · see citing works
CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model
Amrani, N., Luk, K., Singh, P., Shipley, M. A., Isik, M., Donadoni, M., Bellizzi, A., Khalili, K., Sariyer, I. K., Neumann, D. M., et al. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101303 · Cited by 20 · see citing works
“30,31 The AAV9 vector carrying SaCas9-g2g1 was administered to the rabbit keratitis model at an AAV dose of 6E+12 vg/kg (low dose) or 3E+13 vg/kg (high dose), through ear vein injection.”
Gene Therapy: A Promising Approach to Treating Spinal Muscular Atrophy
Mulcahy, P. J., Iremonger, K., Karyka, E., Herranz-Martín, S., Shum, K., Tam, J. K. V., & Azzouz, M. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.186 · Cited by 20 · see citing works
“The family of AAV contains more than 100 serotypes, and AAV9 stands out for the treatment of neurological diseases such as SMA because of its ability to cross the blood–brain barrier through the vascular system (Dayton et al. , 2012).”
Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice
Chapdelaine, P., Gérard, C., Sanchez, N., Cherif, K., Rousseau, J., Ouellet, D. L., Jauvin, D., & Tremblay, J. P. (2016) · Gene Therapy · doi:10.1038/gt.2016.36 · Cited by 21 · see citing works
Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec—Preliminary Results
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“One of the latest advances in drug therapy of SMA is gene therapy, the effects of which are delivered through an adeno-associated viral serotype 9 (AAV9) vector [16,17].”
Background
Spinal cord injury induced neuropathic pain: Molecular targets and therapeutic approaches
Schomberg, D. T., Miranpuri, G. S., Duellman, T., Crowell, A. D., Vemuganti, R., & Resnick, D. K. (2015) · Metabolic Brain Disease · doi:10.1007/s11011-014-9642-0 · Cited by 19 · see citing works
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Authors: Dayton, R. D., Wang, D. B., & Klein, R. L.
Affiliations: Louisiana State University Health Sciences Center Shreveport (US)
University of Washington (US)
Corresponding author: Klein, Ronald L
ORCID: Dayton, Robert D
Venue: Expert Opinion on Biological Therapy
Publisher: Informa Healthcare
Published: 2012-04-20
Volume: 12
Issue: 6
Pages: 757-766
ISSN: 1471-2598 (ISSN-L)
1744-7682
DOI: 10.1517/14712598.2012.681463
PMC: PMC3361729
OpenAlex: W2109349667
Cited by: 144
References: 75
Citations by year: 2026: 7 · 2025: 13 · 2024: 11 · 2023: 13 · 2022: 8 · 2021: 4 · 2020: 7 · 2019: 7 · 2018: 8 · 2017: 9 · 2016: 14 · 2015: 12 · 2014: 14 · 2013: 13 · 2012: 4
FWCI: 1.68
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Locations: Expert Opinion on Biological Therapy (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Institute of Neurological Disorders and Stroke (R01NS048450, NS048450)
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
Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
Keywords: AAV9, adeno-associated virus serotypes, intrathecal administration, intravenous administration, intraparenchymal injection, intrauterine administration, neonatal mice, neuronal expression, tau protein, TDP-43, spinal muscular atrophy, lysosomal storage disease, amyotrophic lateral sclerosis, glial expression, gene therapy vectors
MeSH terms: Dependovirus, Animals, Brain, Gene Expression Regulation, Genetic Vectors, Humans, Injections, Intravenous, Injections, Intraventricular, Injections, Spinal, Neurons, Spinal Cord, Genetic Therapy, Gene Transfer Techniques

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The advent of AAV9 expands applications for brain and spinal cord gene delivery

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