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Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Open accessAbstract
Delivery of genes to the brain and spinal cord across the blood-brain barrier (BBB) has not yet been achieved. Here we show that adeno-associated virus (AAV) 9 injected intravenously bypasses the BBB and efficiently targets cells of the central nervous system (CNS). Injection of AAV9-GFP into neonatal mice through the facial vein results in extensive transduction of dorsal root ganglia and motor neurons throughout the spinal cord and widespread transduction of neurons throughout the brain, including the neocortex, hippocampus and cerebellum. In adult mice, tail vein injection of AAV9-GFP leads to robust transduction of astrocytes throughout the entire CNS, with limited neuronal transduction. This approach may enable the development of gene therapies for a range of neurodegenerative diseases, such as spinal muscular atrophy, through targeting of motor neurons, and amyotrophic lateral sclerosis, through targeting of astrocytes. It may also be useful for rapid postnatal genetic manipulations in basic neuroscience studies.
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“In addition, AAV9 vectors have the unique ability to cross the blood–brain-barrier and target the brain upon intravenous injection in adult mice and cats (Duque et al, 2009; Foust et al, 2009).”
From Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer (2012) · cites this paper as Foust et al, 2009
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“Immunohistochemistry with anti-eGFP antibodies at 1 month PI, showed a viral transduction efficiency of 20–50% of eGFP-positive cells depending on the tissue (Supporting Information Fig S1E and F) (Foust et al, 2009; Inagaki et al, 2006; Zincarelli et al, 2008).”
From Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer (2012) · cites this paper as Foust et al, 2009
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“AAV9 can cross the blood–brain barrier and transduce both neurons and astrocytes after intravenous administration.7,10–11”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [11]
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“AAV8 and AAV5 transduce a significantly greater area of the CNS in comparison with AAV2”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [11]
Experimental — accuracy not yet measuredIn the citing paper
“It was confirmed in SMA murine models that systemically delivered self-complementary AAV9 (scAAV9) crossed the blood brain barrier and achieved high levels of neuronal transduction.9,10”
From Current clinical applications of in vivo gene therapy with AAVs (2021) · cites this paper as [9]
In the citing paper
“The discovery of newer AAV capsids, such as AAV9, have permitted a much wider degree of gene transfer than multiple stereotaxic injections.9,10,67, 68, 69”
From Current clinical applications of in vivo gene therapy with AAVs (2021) · cites this paper as [9]
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Citations
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Chromatin Structure and Dynamics: Focus on Neuronal Differentiation and Pathological Implication
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Prospects for gene replacement therapies in amyotrophic lateral sclerosis
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“However, AAV9 and close relatives are currently the gold standard in clinical trials with systemic delivery approaches [23,45], including the systemic delivery to the heart [46–54].”
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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 26
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“associated virus (AAV) can also transduce muscle and even the central nervous system upon systemic injection (76).”
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Diagnosis and New Treatment Avenues in Spinal Muscular Atrophy
Kirschner, J., & Pechmann, A. (2017) · Neuropediatrics · doi:10.1055/s-0037-1603517 · Cited by 23
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“The recent advances made in gene therapy provide a promising avenue for the treatment of CNS-involved LSDs. Viral vectors such as AAV9 have been shown to cross the BBB for effective CNS delivery in MPS III A and B (Foust et al., 2009; Fu et al., 2011; Aronovich and Hackett, 2015).”
BackgroundRimoldi, M., Lucchiari, S., Pagliarani, S., Meola, G., Comi, G. P., & Abati, E. (2024) · Neurological Sciences · doi:10.1007/s10072-024-07826-9 · Cited by 27
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“These include the use of serotypes that spread further from the site of single stereotaxic injections (e.g. AAV9 and AAV10) [164]; the delivery of serotypes amenable to transport along neuronal pathways (e.g. AAV8 and AAV9) into brain nuclei with divergent connectivity [165,166]; and vector injection directly into cer…”
BackgroundInfluentialExploiting Natural Diversity of AAV for the Design of Vectors with Novel Properties
Gao, G., Li, Z., & Danos, O. (2011) · Methods in molecular biology · doi:10.1007/978-1-61779-370-7_4 · Cited by 24
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“The adenoassociated virus type 9 (AAV9) vector was found to cross the BBB of neonate mice, cats, as well as nonhuman primates after intravenous injection [5-7], and simian virus 40 (SV40) was able to cross the BBB of mice [8].”
BackgroundTherapy Development for Spinal Muscular Atrophy in SMN Independent Targets
Tsai, L. (2012) · Neural Plasticity · doi:10.1155/2012/456478 · Cited by 22
“In contrast, intravenous AAV serotype 9 (AAV9) injection has shown success in affecting widespread gene delivery in entire spinal cord [76].”
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“Several studies already showed that injection of self-complementary scAAV9 vectors expressing full-length human SMN protein in different severe SMA mouse models remarkably ameliorated lifespan and disease severity [71-74].”
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Genetic Constructs for the Control of Astrocytes’ Activity
Borodinova, A. A., Balaban, P. M., Борисович, Б. И., Salmina, A. B., & Власова, О. Л. (2021) · Cells · doi:10.3390/cells10071600 · Cited by 22
“Unexpectedly, the cellular tropism of particular AAV serotypes can be injection route-dependent, making it impossible to combine the data obtained from different protocols of local and systemic virus delivery [69,71].”
BackgroundPerinatal systemic gene delivery using adeno-associated viral vectors
Karda, R., Buckley, S. M. K., Mattar, C. N. Z., Ng, J., Massaro, G., Hughes, M. P., Kurian, M. A., Baruteau, J., Gissen, P., Chan, J. K. Y., et al. (2014) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2014.00089 · Cited by 22
“Mattar et al. (2013) evaluated the ability of AAV9 to transduce the CNS through a single intravenous injection into a late gestation fetal macaque.”
BackgroundMethodInfluentialAdvances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders
Zhou, L., Wang, Y., Xu, Y., Zhang, Y., & Zhu, C. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101357 · Cited by 22
Impact of liver fibrosis on AAV-mediated gene transfer to mouse hepatocytes
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“Given its ability to traverse biologicalbarriers 22 , we tested AAV9-mediated gene transfer in fi brotic livers.”
Fetal gene therapy: recent advances and current challenges
Mattar, C. N. Z., Choolani, M. A., Biswas, A., Waddington, S. N., & Chan, J. K. Y. (2011) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2011.585153 · Cited by 21
“The use of different viral envelopes (pseudotyping) can enhance specific organ-targeting and improve transduction efficiency, such as the use of different AAV serotypes for liver- [49] or CNS-directed gene therapy [90].”
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NF1, Neurofibromin and Gene Therapy: Prospects of Next-Generation Therapy
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“10), have shown unique properties to target the central nervous system (CNS) in comparison to most AAV serotypes (Foust et al., 2009).”
BackgroundAAV9-mediated SH3TC2 gene replacement therapy targeted to Schwann cells for the treatment of CMT4C
Georgiou, E., Kagiava, A., Sargiannidou, I., Schiza, N., Stavrou, M., Richter, J., Tryfonos, C., Heslegrave, A., Zetterberg, H., Christodoulou, C., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.08.020 · Cited by 26
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Preferential Lentiviral Targeting of Astrocytes in the Central Nervous System
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“powerful tool for gene delivery specifically into astrocytes [61].”
Piersanti, S., Astrologo, L., Licursi, V., Costa, R., Roncaglia, E., Gennetier, A., Ibanes, S., Chillón, M., Negri, R., Tagliafico, E., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0069808 · Cited by 21
“Under these conditions, AAV2/9, which has been suggested for brain gene therapy [27], transduced differentiated hmNPCs with poor efficiency (,4%, data not shown), and therefore was unsuitable for comparative microarray analysis.”
BackgroundSomatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing
Odermatt, P., TRUEB, J., Furrer, L., Fricker, R., Marti, A., & Schümperli, D. (2016) · Molecular Therapy · doi:10.1038/mt.2016.152 · Cited by 23
Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques
Gombash, S. E., Cowley, C. J., Fitzgerald, J. A., Lepak, C. A., Neides, M. G., Hook, K., Todd, L. J., Wang, G., Mueller, C., Kaspar, B. K., et al. (2017) · Gene Therapy · doi:10.1038/gt.2017.72 · Cited by 19
“We previously reported extensive CNS astrocyte transduction in mice and non-human primates using the identical AAV9 construct as this study(22, 41).”
InfluentialSelf-citationIntravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis
Gross, A. L., Gray‐Edwards, H. L., Bebout, C. N., Ta, N. L., Nielsen, K., Brunson, B. L., Mercado, K. R. L., Osterhoudt, D. E., Batista, A. R., Maitland, S. A., et al. (2021) · Brain · doi:10.1093/brain/awab309 · Cited by 20
Engineering novel adeno-associated viruses (AAVs) for improved delivery in the nervous system
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Gene Therapy: A Promising Approach to Treating Spinal Muscular Atrophy
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“Various routes of delivery of scAAV9-SMN1, including intravenous, intracerebroventricular, intramuscular, and combined routes, have been investigated for SMA therapy (Foust et al., 2009; Dominguez et al., 2011; Glascock et al., 2012b; Benkhelifa-Ziyyat et al., 2013).”
InfluentialGene and splicing therapies for neuromuscular diseases
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“the blood-brain barrier through the vascular system (22).”
BackgroundKnockdown of GADD34 in neonatal mutant SOD1 mice ameliorates ALS
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High rate of HDR in gene editing of p.(Thr158Met) MECP2 mutational hotspot
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“Indeed, AAVs are immune-tolerated and some serotypes are capable of crossing the BBB, an essential feature for in vivo gene therapy of CNS disorders via intravenous injection [38, 39].”
BackgroundPOLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches
Perrier, S., Michell‐Robinson, M. A., & Bernard, G. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2020.631802 · Cited by 23
“allows for intrathecal or intravenous administration (Foust et al., 2009; Mendell et al., 2017; Gessler et al., 2019).”
Safety and Efficacy of Intravenous and Intrathecal Delivery of AAV9-Mediated ARSA in Minipigs
Mullagulova, A. I., Shaimardanova, A. A., Solovyeva, V. V., Mukhamedshina, Y. O., Chulpanova, D. S., Kostennikov, A., Issa, S. S., & Rizvanov, A. A. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24119204 · Cited by 20
“When administered intravenously, AAV9 can cross the BBB and enter the CNS [52].”
BackgroundViral vector-mediated gene transfer for CNS disease
Snyder, B. R., Boulis, N. M., & Federici, T. (2010) · Expert Opinion on Biological Therapy · doi:10.1517/14712590903514074 · Cited by 22
“Two other groups reported efficient and widespread transduction of sensory and motor neurons in the spinal cord after intravascular delivery of scAAV9 to the facial vein of neonate mice, when the BBB is not completely developed [42,43].”
BackgroundResearch advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis
Nizzardo, M., Simone, C., Falcone, M., Riboldi, G. M., Rizzo, F., Magri, F., Bresolin, N., Comi, G. P., & Corti, S. P. (2011) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-011-0881-5 · Cited by 21
In Vivo Astrocytic Ca 2+ Signaling In Health and Brain Disorders
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Schober, A. L., Gagarkin, D. A., Chen, Y., Gao, G., Jacobson, L., & Mongin, A. A. (2016) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2016.00262 · Cited by 19
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The therapeutic potential of RNA regulation in neurological disorders
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“AAV9, for example, is often used to target the brain [16], while”
BackgroundAstrocytes in Motor Neuron Diseases
Valori, C. F., Guidotti, G., Brambilla, L., & Rossi, D. (2019) · Advances in experimental medicine and biology · doi:10.1007/978-981-13-9913-8_10 · Cited by 19
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Gene delivery with viral vectors for cerebrovascular diseases
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Fu, H., Zaraspe, K., Murakami, N., Meadows, A. S., Pineda, R. J., McCarty, D. M., & Muenzer, J. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.07.005 · Cited by 20
AAV9: Over the Fence and Into the Woods …
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Glioblastoma Multiforme Selective Nanomedicines for Improved Anti-Cancer Treatments
Duskey, J. T., Rinaldi, A., Ottonelli, I., Caraffi, R., Benedictis, C. A. D., Sauer, A. K., Tosi, G., Vandelli, M. A., Ruozi, B., & Grabrucker, A. M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14071450 · Cited by 21
“For this reason, although not specific for GBM, NMeds decorated with g7 and AAVF can still be valuable players in the search for improved GBM treatments, as they are known to be effective for BBB crossing [43,45,111,112].”
BackgroundAdvances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases
Li, L., Vasan, L., Kartono, B., Clifford, K. P., Attarpour, A., Sharma, R., Mandrozos, M., Kim, A., Zhao, W., Belotserkovsky, A., et al. (2023) · Biomedicines · doi:10.3390/biomedicines11102725 · Cited by 19
“These analyses highlighted AAV2 and AAV9, which are found in Clades B and F, respectively, and were originally isolated from human tissues for their ability to penetrate the BBB and preferentially target CNS neurons [23,48].”
BackgroundBorges, B., Varthaliti, A., Schwab, M. E., Clarke, M. T., Pivetti, C. D., Gupta, N. M., Cadwell, C. R., 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 20
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“The results showed better dose-dependent putaminal coverage [21% (cohort 1), 34% (cohort 2), and 42% (cohort 3)], AADC activity (13%, 56%, and 79%), and less antiparkinsonian medication use (13%, 56%, and 79%, respectively) at 6 months.”
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Organ targeted prenatal gene therapy—how far are we?
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“Recent studies using AAV9 serotype in neonatal rats has demonstrated that intravascular delivery of this vector can transduce cells of the nervous system (Foust et al., 2009).”
BackgroundAdachi, K., & Nakai, H. (2010) · Gene Therapy and Regulation · doi:10.1142/s1568558610000197 · Cited by 19
“Vectors based on AAV serotype 8 or 9 have been shown to overcome the barriers faced by earlier-generation rAAV vectors and mediate robust transduction in various organs including the liver, heart, striated muscles and central nervous system (Foust et al., 2009; Ghosh et al., 2007; Inagaki et al., 2006; Pacak et al., 2…”
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“with a single dose of AAV9 in P0/P1 mouse neonates [43].”
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“tion efficiency in neurons after intravenous injection in neonatal mice.(27) Herein, we observed widespread expres-”
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Prabhakar, S., Zhang, X., Goto, J., Han, S., Lai, C. P., Bronson, R. T., Sena‐Esteves, M., Ramesh, V., Stemmer-Rachamimov, A. O., Kwiatkowski, D. J., et al. (2015) · Neurobiology of Disease · doi:10.1016/j.nbd.2015.04.018 · Cited by 19
“liver, kidney, lung and muscle (Foust et al., 2009; Zhang et al., 2011).”
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 20
“…key event in the inflammatory response after SCI involves cooperation between the upregulation of the chemoattractant SDF-1 and MMP-9 expressed by infiltrating monocytes and suggests a synergistic relationship that enhances the transmigration of monocytes into the injured spinal cord (Zhang 2011b).”
BackgroundGenetic manipulation of brain endothelial cells in vivo
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The Dilemma of Two Innovative Therapies for Spinal Muscular Atrophy
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Gene Therapy for Misfolding Protein Diseases of the Central Nervous System
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“In this regard, AAV9 is especially interesting in view of its ability to cross the blood–brain barrier after being injected into the circulation [16, 26].”
BackgroundGene therapy for a mouse model of glucose transporter-1 deficiency syndrome
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“Intravenously injected AAV9-based vectors at neonatal period reportedly pass through the blood-brain barrier and transducewidespread neuronal targets including the striatum, cortex, anterior commissure, internal capsule, corpus callosum, hippocampus, dentate gyrus, midbrain, and cerebellum [23].”
BackgroundBanning, A., Zakrzewicz, A., Chen, X., Gray, S. J., & Tikkanen, R. (2021) · Cells · doi:10.3390/cells10051259 · Cited by 18
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“Indeed, the differential target specificity to the same AAV serotypes at different ages of animals has been reported.(45,64) In nonhuman primates, intravascular administration of the AAV9 vector in neonatal(65) or juvenile(66) animals yielded different efficacy of gene transduction into CNS neurons.”
BackgroundPietersz, K. L., Plessis, F. D., Pouw, S. M., Liefhebber, J. M. P., van Deventer, S. J. H., Martens, G. J. M., Konstantinova, P. S., & Blits, B. (2021) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2021.679483 · Cited by 20
“For example, in one of the first studies, predominant astrocyte transduction (Foust et al., 2008) was reported while another laboratory observed the transduction of neurons (Gray et al., 2011).”
BackgroundResultCell Reprogramming for Regeneration and Repair of the Nervous System
Clark, I. H., Roman, A., Fellows, E., Radha, S., Var, S. R., Roushdy, Z., Borer, S. M., Johnson, S., Chen, O. M., Borgida, J. S. W., et al. (2022) · Biomedicines · doi:10.3390/biomedicines10102598 · Cited by 20
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy
Li, Y., Zeng, H., Wei, Y., Ma, X., & He, Z. (2023) · Human Gene Therapy · doi:10.1089/hum.2022.189 · Cited by 19
“Ideal vectors are lenti-virus 46 and adeno-associated virus (AAV) 47–49 ; AAVs are the first choice because of several advantages, such as central nervous system (CNS) targeting (AAV9), 50 global biodistribution and transduction in peripheral organs, 10 and low immunogenicity.”
InfluentialMerjane, J., Chung, R. S., Patani, R., & Lisowski, L. (2023) · Medicinal Research Reviews · doi:10.1002/med.21937 · Cited by 19
“134 Although this was a significant advancement for the field, a key issue quickly arose: although AAV9 demonstrates clear ability to enter and transduce cells in the CNS, it also has strong tropism for human liver.”
Current avenues of gene therapy in Pompe disease
Leon‐Astudillo, C., Trivedi, P. D., Sun, R. C., Gentry, M. S., Fuller, D. D., Byrne, B. J., & Corti, M. (2023) · Current Opinion in Neurology · doi:10.1097/wco.0000000000001187 · Cited by 19
“While intravenous administration of AAV leads to widespread gene transfer in the neonatal CNS, this approach is not easily translatable to LOPD since it would require high vector doses to treat the CNS manifestations [63,64].”
BackgroundCrisafulli, S., Boccanegra, B., Vitturi, G., Trifirò, G., & Luca, A. D. (2023) · Brain Sciences · doi:10.3390/brainsci13101446 · Cited by 21
Emerging trends in virus and virus-like particle gene therapy delivery to the brain
Mandalawatta, H. P. P., Rajendra, K. C., Fairfax, K. A., & Hewitt, A. W. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102280 · Cited by 18
“…(ALS) had shown an ef fi cient transgene expression in the neonate brain, dorsal root ganglia and spinal cord motor neurons, and astrocytes, 62 whereas rAAVrh.8 has been shown to transduce glial cells and neurons located within the cortex, caudate-putamen, hippocampus, corpus callosum and…”
BackgroundTargeting neurological disease with RNAi
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Somatic Genetics Analysis of Sleep in Adult Mice
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The case for intrauterine gene therapy
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Fiber-Modified Adenovirus for Central Nervous System Parkinson’s Disease Gene Therapy
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In vivo targeting of subventricular zone astrocytes
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Recombinant Adeno-Associated Virus: Efficient Transduction of the Rat VMH and Clearance from Blood
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“Several AAVs have been shown to effectively cross the mouse blood-brain barrier after intravascular delivery [13,14].”
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“More recently, a study using AAV2-9 has demonstrated a unique ability to cross the blood brain barrier to predominately transduce astrocytes in the adult mouse brain and spinal cord (37).”
Robert, M.-A., Nassoury, N., Chahal, P. S., Venne, M.-H., Racine, T., Qiu, X., Kobinger, G., Kamen, A., Gilbert, R., & Gaillet, B. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.101 · Cited by 19
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Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach
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“Additionally, while the use of the scAAV9 serotype is attractive due to its ability to cross the blood-brain barrier, this serotype is ineffective at transducing microglia.(59,60) SSOs avoid some of these challenges in that they target and corrects endogenous CLN3 expression and SSOs are taken up broadly across the CN…”
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“3,4 However, systemic injection for CNS targeting lacks speci fi city and ef fi ciency, resulting in signi fi cant transduction of off-target tissues, with high risk of toxicity, particularly at high vector doses.”
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“…cells by transferrin receptor (TfR) -targeted peptide motifs (Xia et al. 2000), no previous studies have been performed in polarized BBB cell models, which have unique receptor patterns on their apical/ basolateral side, restricted paracellular passage and transcellular delivery mechanisms.”
Metadata
- Authors
- Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K.
- Affiliations
- Nationwide Children's Hospital (US)
- Corresponding author
- Kaspar, Brian K
- Venue
- Nature Biotechnology
- Published
- 2008-12-21
- Volume
- 27
- Issue
- 1
- Pages
- 59-65
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- PMC
- PMC2895694
- OpenAlex
- W2166739815
- Cited by
- 1,456
- References
- 38
- Citations by year
- 2026: 75 · 2025: 91 · 2024: 107 · 2023: 94 · 2022: 87 · 2021: 101 · 2020: 105 · 2019: 90 · 2018: 100 · 2017: 81 · 2016: 79 · 2015: 78 · 2014: 74 · 2013: 88 · 2012: 54
- FWCI
- 28.00
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Eye Institute (R21EY018491)
- National Institute of Neurological Disorders and Stroke (R21NS064328, R01NS064492)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- AAV9, adeno-associated virus, blood-brain barrier, astrocytes, motor neurons, neonatal neurons, spinal cord, dorsal root ganglia, gene therapy, spinal muscular atrophy, amyotrophic lateral sclerosis
- MeSH terms
- Dependovirus, Animals, Astrocytes, Ganglia, Spinal, Genetic Vectors, Hippocampus, Mice, Inbred C57BL, Muscular Atrophy, Spinal, Neurons, In Situ Hybridization, Gene Transfer Techniques, Transgenes, Neocortex, Green Fluorescent Proteins, Mice
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