This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters
Gene Therapy Open Access 13 January 2023
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Lerner TN, Ye L, Deisseroth K. Communication in neural circuits: tools, opportunities, and challenges. Cell. 2016;164:1136–50.
Burnside ER, De Winter F, Didangelos A, James ND, Andreica EC, Layard-Horsfall H, et al. Immune-evasive gene switch enables regulated delivery of chondroitinase after spinal cord injury. Brain. 2018;141:2362–81.
Deverman BE, Pravdo PL, Simpson BP, Kumar SR, Chan KY, Banerjee A, et al. Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat Biotechnol. 2016;34:204–9.
Chan KY, Jang MJ, Yoo BB, Greenbaum A, Ravi N, Wu WL, et al. Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems. Nat Neurosci. 2017;20:1172–9.
Flytzanis NC, Goeden N, Goertsen D, Cummins A, Pickel J, Gradinaru V. Broad gene expression throughout the mouse and marmoset brain after intravenous delivery of engineered AAV capsids. BioRxiv. 2020. https://doi.org/10.1101/2020.06.16.152975
Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O’Malley KL, et al. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet. 1994;8:148–54.
Bartlett JS, Samulski RJ, McCown TJ. Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum Gene Ther. 1998;9:1181–6.
Mason MR, Ehlert EM, Eggers R, Pool CW, Hermening S, Huseinovic A, et al. Comparison of AAV serotypes for gene delivery to dorsal root ganglion neurons. Mol Ther. 2010;18:715–24.
Vulchanova L, Schuster DJ, Belur LR, Riedl MS, Podetz-Pedersen KM, Kitto KF, et al. Differential adeno-associated virus mediated gene transfer to sensory neurons following intrathecal delivery by direct lumbar puncture. Mol Pain. 2010;6:31.
Hellstrom M, Ruitenberg MJ, Pollett MA, Ehlert EM, Twisk J, Verhaagen J, et al. Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection. Gene Ther. 2009;16:521–32.
Hutson TH, Verhaagen J, Yanez-Munoz RJ, Moon LD. Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and a non-integrating lentiviral vector. Gene Ther. 2012;19:49–60.
Blits B, Derks S, Twisk J, Ehlert E, Prins J, Verhaagen J. Adeno-associated viral vector (AAV)-mediated gene transfer in the red nucleus of the adult rat brain: comparative analysis of the transduction properties of seven AAV serotypes and lentiviral vectors. J Neurosci Methods. 2010;185:257–63.
von Jonquieres G, Mersmann N, Klugmann CB, Harasta AE, Lutz B, Teahan O, et al. Glial promoter selectivity following AAV-delivery to the immature brain. PLoS One. 2013;8:e65646.
McLean JR, Smith GA, Rocha EM, Hayes MA, Beagan JA, Hallett PJ, et al. Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection. Neurosci Lett. 2014;576:73–8.
Nieuwenhuis B, Haenzi B, Hilton S, Carnicer-Lombarte A, Hobo B, Verhaagen J, et al. Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters. Gene Ther. 2020. https://doi.org/10.1038/s41434-020-0169-1.
Pietersz KL, Martier RM, Baatje MS, Liefhebber JM, Brouwers CC, Pouw SM, et al. Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery. Gene Ther. 2020. https://doi.org/10.1038/s41434-020-0178-0.
Bailey RM, Rozenberg A, Gray SJ. Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies. Brain Res. 2020;1739:146832.
He T, Itano MS, Earley LF, Hall NE, Riddick N, Samulski RJ, et al. The influence of murine genetic background in adeno-associated virus transduction of the mouse brain. Hum Gene Ther Clin Dev. 2019;30:169–81.
Watakabe A, Ohtsuka M, Kinoshita M, Takaji M, Isa K, Mizukami H, et al. Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex. Neurosci Res. 2015;93:144–57.
Lu Q, Ganjawala TH, Ivanova E, Cheng JG, Troilo D, Pan ZH. AAV-mediated transduction and targeting of retinal bipolar cells with improved mGluR6 promoters in rodents and primates. Gene Ther. 2016;23:680–9.
Acknowledgements
I thank Dr. David Tumbarello for critically reading this manuscript.
Funding
Funding from the Biotechnology and Biological Sciences Research Council to MRA.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Conflict of interest
The author declares that she has no conflict of interest.
Additional information
Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Rights and permissions
About this article
Cite this article
Andrews, M.R. Gene therapy in the CNS—one size does not fit all. Gene Ther 28, 393–395 (2021). https://doi.org/10.1038/s41434-020-00196-9
Received:
Revised:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41434-020-00196-9