A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism
Open accessPublished version of a preprint
Abstract
Adeno-associated virus (AAV) capsid modification enables the generation of recombinant vectors with tailored properties and tropism. Most approaches to date depend on random screening, enrichment, and serendipity. The approach explored here, called BRAVE (barcoded rational AAV vector evolution), enables efficient selection of engineered capsid structures on a large scale using only a single screening round in vivo. The approach stands in contrast to previous methods that require multiple generations of enrichment. With the BRAVE approach, each virus particle displays a peptide, derived from a protein, of known function on the AAV capsid surface, and a unique molecular barcode in the packaged genome. The sequencing of RNA-expressed barcodes from a single-generation in vivo screen allows the mapping of putative binding sequences from hundreds of proteins simultaneously. Using the BRAVE approach and hidden Markov model-based clustering, we present 25 synthetic capsid variants with refined properties, such as retrograde axonal transport in specific subtypes of neurons, as shown for both rodent and human dopaminergic neurons.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The capsid-cell surface interaction is the primary determinant of AAV tropism.
“Although it is possible to utilize promoter elements that limit transgene expression to specific cellular lineages, the capsid-cell surface interaction is the primary determinant of AAV tropism.”
From Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease (2021) · cites this paper as [2]
In Results, ‘Validation of the BRAVE Screening Principle’ (page unavailable), removing a capsid motif that binds a cell-surface proteoglycan eliminated HEK293T-cell tropism, and a modified capsid restored it. This supports a role for capsid–cell-surface interactions, but the paper does not establish that they are the primary determinant of AAV tropism relative to other factors. (LLM, conf=0.82)
Experimental — accuracy not yet measured
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
The Genome Analysis Toolkit: A MapReduce framework for analyzing next-generation DNA sequencing data
McKenna, A., Hanna, M., Banks, E., Sivachenko, A., Cibulskis, K., Kernytsky, A., Garimella, K., Altshuler, D., Gabriel, S., Daly, M., et al. (2010) · Genome Research · doi:10.1101/gr.107524.110 · Cited by 27,895 · see citing works
Search and clustering orders of magnitude faster than BLAST
Edgar, R. C. (2010) · Bioinformatics · doi:10.1093/bioinformatics/btq461 · Cited by 20,617 · see citing works
MapReduce: simplified data processing on large clusters
Dean, J., & Ghemawat, S. (2008) · Communications of the ACM · doi:10.1145/1327452.1327492 · Cited by 18,476 · see citing works
Site-directed mutagenesis by overlap extension using the polymerase chain reaction
Ho, S. N., Hunt, H. D., Horton, R. M., Pullen, J. K., & Pease, L. R. (1989) · Gene · doi:10.1016/0378-1119(89)90358-2 · Cited by 7,354 · see citing works
Visualization and analysis of gene expression in tissue sections by spatial transcriptomics
Ståhl, P. L., Salmén, F., Vickovic, S., Lundmark, A., Navarro, J. F., Magnusson, J., Giacomello, S., Asp, M., Westholm, J. O., Huss, M., et al. (2016) · Science · doi:10.1126/science.aaf2403 · Cited by 3,834 · see citing works
Slide-seq: A scalable technology for measuring genome-wide expression at high spatial resolution
Rodriques, S. G., Stickels, R. R., Goeva, A., Martin, C. A., Murray, E., Vanderburg, C. R., Welch, J., Chen, L. M., Chen, F., & Macosko, E. Z. (2019) · Science · doi:10.1126/science.aaw1219 · Cited by 2,424 · see citing works
Fusion protein linkers: Property, design and functionality
Chen, X., Zaro, J. L., & Shen, W. (2012) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2012.09.039 · Cited by 2,071 · see citing works
Russell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31868-8 · Cited by 1,952 · see citing works
Chan, K. Y., Jang, M. J., Yoo, B. B., Greenbaum, A., Ravi, N., Wu, W.-L., Sánchez-Guardado, L., Lois, C., Mazmanian, S. K., Deverman, B. E., et al. (2017) · Nature Neuroscience · doi:10.1038/nn.4593 · Cited by 1,578 · see citing works
Zolotukhin, S., Byrne, B. J., Mason, E. G., Zolotukhin, I., Potter, M., Chesnut, K. A., Summerford, C., Samulski, R. J., & Muzyczka, N. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300938 · Cited by 1,317 · see citing works
Putting a spin on the dorsal-ventral divide of the striatum
Voorn, P., Vanderschuren, L. J. M. J., Groenewegen, H. J., Robbins, T. W., & Pennartz, C. M. A. (2004) · Trends in Neurosciences · doi:10.1016/j.tins.2004.06.006 · Cited by 1,188 · see citing works
A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons
Tervo, D. G. R., Hwang, B., Viswanathan, S., Gaj, T., Lavzin, M., Ritola, K., Lindo, S. E., Michael, S. C., Кулешова, Е. П., Ojala, D. S., et al. (2016) · Neuron · doi:10.1016/j.neuron.2016.09.021 · Cited by 1,170 · see citing works
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
Deverman, B. E., Pravdo, P. L., Simpson, B. P., Kumar, S. R., Chan, K. Y., Banerjee, A., Wu, W.-L., Yang, B., Huber, N., Pasca, S. P., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3440 · Cited by 1,061 · see citing works
Emerging issues in AAV-mediated in vivo gene therapy
Colella, P., Ronzitti, G., & Mingozzi, F. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2017.11.007 · Cited by 881 · see citing works
Jordan, M., Schallhorn, A., & Wurm, F. M. (1996) · Nucleic Acids Research · doi:10.1093/nar/24.4.596 · Cited by 862 · see citing works
Spreading of pathology in neurodegenerative diseases: a focus on human studies
Brettschneider, J., Tredici, K. D., Lee, V. M.-Y., & Trojanowski, J. Q. (2015) · Nature Reviews Neuroscience · doi:10.1038/nrn3887 · Cited by 790 · see citing works
McCarty, D., Monahan, P., & Samulski, R. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301514 · Cited by 735 · see citing works
Dalkara, D., Byrne, L. C., Klimczak, R. R., Visel, M., Yin, L., Merigan, W. H., Flannery, J. G., & Schaffer, D. V. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005708 · Cited by 734 · see citing works
Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors
Grimm, D., Kern, A., Rittner, K., & Kleinschmidt, J. A. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.18-2745 · Cited by 700 · see citing works
Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696 · see citing works
McCarty, D. M., Fu, H., Monahan, P. E., Toulson, C. E., Naik, P., & Samulski, R. J. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302134 · Cited by 547 · see citing works
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
Maheshri, N., Koerber, J. T., Kaspar, B. K., & Schaffer, D. V. (2006) · Nature Biotechnology · doi:10.1038/nbt1182 · Cited by 538 · see citing works
Isolation and culture of adult neurons and neurospheres
Brewer, G. J., & Torricelli, J. R. (2007) · Nature Protocols · doi:10.1038/nprot.2007.207 · Cited by 448 · see citing works
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
Hordeaux, J., Wang, Q., Katz, N., Buza, E. L., Bell, P., & Wilson, J. M. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.01.018 · Cited by 426 · see citing works
Müller, O. J., Kaul, F., Weitzman, M. D., Pasqualini, R., Arap, W., Kleinschmidt, J. A., & Trepel, M. (2003) · Nature Biotechnology · doi:10.1038/nbt856 · Cited by 411 · see citing works
Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2
Girod, A., Ried, M., Wobus, C., Lahm, H., Leike, K., Kleinschmidt, J., Deléage, G., & Hallek, M. (1999) · Nature Medicine · doi:10.1038/12491 · Cited by 405 · see citing works
Wu, P., Xiao, W. S., Conlon, T. J., Hughes, J. A., Agbandje‐McKenna, M., Ferkol, T. W., Flotte, T. R., & Muzyczka, N. (2000) · Journal of Virology · doi:10.1128/jvi.74.18.8635-8647.2000 · Cited by 390 · see citing works
Linkers in the structural biology of protein–protein interactions
Chichili, V. P. R., Kumar, V., & Sivaraman, J. (2012) · Protein Science · doi:10.1002/pro.2206 · Cited by 366 · see citing works
Opie, S. R., Warrington, Agbandje-McKenna, M., Zolotukhin, S., & Muzyczka, N. (2003) · Journal of Virology · doi:10.1128/jvi.77.12.6995-7006.2003 · Cited by 364 · see citing works
Amplification of complex gene libraries by emulsion PCR
Williams, R., Peisajovich, S. G., Miller, O. J., Magdassi, S., Tawfik, D. S., & Griffiths, A. D. (2006) · Nature Methods · doi:10.1038/nmeth896 · Cited by 358 · see citing works
In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector
Zinn, E., Pacouret, S., Khaychuk, V., Turunen, H. T., Carvalho, L. S., Andres-Mateos, E., Shah, S., Shelke, R., Maurer, A. C., Plovie, E., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.07.019 · Cited by 310 · see citing works
Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306 · see citing works
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291 · see citing works
Li, W., Asokan, A., Wu, Z., Van Dyke, T., DiPrimio, N., Johnson, J. S., Govindaswamy, L., Agbandje-McKenna, M., Leichtle, S., Eugene Redmond Jr, D., et al. (2008) · Molecular Therapy · doi:10.1038/mt.2008.100 · Cited by 261 · see citing works
Dujardin, S., Lécolle, K., Caillierez, R., Bégard, S., Zommer, N., Lachaud, C., Carrier, S., Dufour, N., Aurégan, G., Winderickx, J., et al. (2014) · Acta Neuropathologica Communications · doi:10.1186/2051-5960-2-14 · Cited by 259 · see citing works
Nolbrant, S., Heuer, A., Parmar, M., & Kirkeby, A. (2017) · Nature Protocols · doi:10.1038/nprot.2017.078 · Cited by 252 · see citing works
Self-citationErratum: Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248 · see citing works
Incorporation of Tumor-Targeting Peptides into Recombinant Adeno-associated Virus Capsids
Grifman, M., Trepel, M., Speece, P., Gilbert, L. B., Arap, W., Pasqualini, R., & Weitzman, M. D. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0345 · Cited by 241 · see citing works
Starcode: sequence clustering based on all-pairs search
Zorita, E. V., Cuscó, P., & Filion, G. J. P. (2015) · Bioinformatics · doi:10.1093/bioinformatics/btv053 · Cited by 241 · see citing works
Efficient and Selective AAV2-Mediated Gene Transfer Directed to Human Vascular Endothelial Cells
Nicklin, S. A., Buening, H., Dishart, K. L., de Alwis, M., Girod, A., Hacker, U., Thrasher, A. J., Ali, R. R., Hallek, M. J., & Baker, A. H. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0424 · Cited by 219 · see citing works
A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection
Yang, L., Jiang, J., Drouin, L. M., Agbandje‐McKenna, M., Chen, C., Qiao, C., Pu, D., Hu, X., Wang, D.-Z., Li, J., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0813207106 · Cited by 208 · see citing works
Human glia can both induce and rescue aspects of disease phenotype in Huntington disease
Benraiss, A., Wang, S., Herrlinger, S., Li, X., Chandler-Militello, D., Mauceri, J., Burm, H. B., Toner, M., Osipovitch, M., Jim Xu, Q., et al. (2016) · Nature Communications · doi:10.1038/ncomms11758 · Cited by 199 · see citing works
Hnasko, T. S., Perez, F. A., Scouras, A. D., Stoll, E. A., Gale, S. D., Luquet, S. H., Phillips, P. E. M., Kremer, E. J., & Palmiter, R. D. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0603081103 · Cited by 198 · see citing works
Gray, S. J., Blake, B. L., Criswell, H. E., Nicolson, S. C., Samulski, R. J., & McCown, T. J. (2010) · Molecular Therapy · doi:10.1038/mt.2009.292 · Cited by 185 · see citing works
Münch, R. C., Janicki, H., Völker, I., Rasbach, A., Hallek, M. J., Büning, H., & Buchholz, C. J. (2012) · Molecular Therapy · doi:10.1038/mt.2012.186 · Cited by 176 · see citing works
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
Chen, Y. H., Chang, M., & Davidson, B. L. (2009) · Nature Medicine · doi:10.1038/nm.2025 · Cited by 168 · see citing works
Adachi, K., Enoki, T., Kawano, Y., Veraz, M., & Nakai, H. (2014) · Nature Communications · doi:10.1038/ncomms4075 · Cited by 149 · see citing works
Rohr, U., Wulf, M.-A., Stahn, S., Steidl, U. G., Haas, R. R., & Kronenwett, R. (2002) · Journal of Virological Methods · doi:10.1016/s0166-0934(02)00138-6 · Cited by 144 · see citing works
Insertional Mutagenesis of AAV2 Capsid and the Production of Recombinant Virus
Rabinowitz, J. E., Xiao, W., & Samulski, R. J. (1999) · Virology · doi:10.1006/viro.1999.0045 · Cited by 136 · see citing works
Perabò, L., Goldnau, D., White, K., Endell, J., Bouças, J., Humme, S., Work, L. M., Janicki, H., Hallek, M. J., Baker, A. H., et al. (2006) · Journal of Virology · doi:10.1128/jvi.00076-06 · Cited by 135 · see citing works
Adeno-Associated Viral Vectors and Their Redirection to Cell-Type Specific Receptors
Michelfelder, S., & Trepel, M. (2009) · Advances in genetics · doi:10.1016/s0065-2660(09)67002-4 · Cited by 134 · see citing works
Ried, M. U., Girod, A., Leike, K., Büning, H., & Hallek, M. J. (2002) · Journal of Virology · doi:10.1128/jvi.76.9.4559-4566.2002 · Cited by 132 · see citing works
Gray, S. J., Choi, V. W., Asokan, A., Haberman, R. A., McCown, T. J., & Samulski, R. J. (2011) · Current Protocols in Neuroscience · doi:10.1002/0471142301.ns0417s57 · Cited by 130 · see citing works
Yang, Q., Mamounas, M. J., Yu, G., Kennedy, S. P., Leaker, B. R., Merson, J. R., Wong‐Staal, F., Yu, M., & Barber, J. R. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.13-1929 · Cited by 127 · see citing works
Asuri, P., Bartel, M. A., Vazin, T., Jang, J.-H., Wong, T. B., & Schaffer, D. V. (2012) · Molecular Therapy · doi:10.1038/mt.2011.255 · Cited by 119 · see citing works
Tordo, J., O’Leary, C., Antunes, A. S. L. M., Palomar, N., Aldrin-Kirk, P., Basche, M., Bennett, A., D’Souza, Z., Gleitz, H., Godwin, A., et al. (2018) · Brain · doi:10.1093/brain/awy126 · Cited by 113 · see citing works
Self-citationWang, L., Bell, P. L., Somanathan, S., Wang, Q., He, Z., Yu, H., McMenamin, D., Goode, T. L., Calcedo, R., & Wilson, J. M. (2015) · Molecular Therapy · doi:10.1038/mt.2015.179 · Cited by 107 · see citing works
Ojala, D. S., Sun, S., Santiago-Ortiz, J. L., Shapiro, M. G., Romero, P. A., & Schaffer, D. V. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.09.006 · Cited by 97 · see citing works
Körbelin, J., Sieber, T., Michelfelder, S., Lunding, L. P., Spies, E., Hunger, A., Alawi, M., Rapti, K., Indenbirken, D., Müller, O. J., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.62 · Cited by 88 · see citing works
Amygdala-ventral striatum circuit activation decreases long-term fear
Correia, S. S., McGrath, A. G., Lee, A., Graybiel, A. M., & Goosens, K. A. (2016) · eLife · doi:10.7554/elife.12669 · Cited by 82 · see citing works
Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier
Albright, B. H., Storey, C. M., Murlidharan, G., Castellanos Rivera, R. M., Berry, G. E., Madigan, V. J., & Asokan, A. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.10.017 · Cited by 82 · see citing works
Whole-rat conditional gene knockout via genome editing
Brown, A. J., Fisher, D. A., Kouranova, E., McCoy, A., Forbes, K., Wu, Y., Henry, R., Ji, D., Chambers, A., Warren, J., et al. (2013) · Nature Methods · doi:10.1038/nmeth.2516 · Cited by 77 · see citing works
Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS
Kanaan, N. M., Sellnow, R. C., Boye, S. L., Coberly, B., Bennett, A. D., Agbandje‐McKenna, M., Sortwell, C. E., Hauswirth, W. W., Boye, S. L., & Manfredsson, F. P. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.06.011 · Cited by 74 · see citing works
Design and Construction of Functional AAV Vectors
Gray, J. T., & Zolotukhin, S. (2012) · Methods in Molecular Biology · doi:10.1007/978-1-61779-370-7_2 · Cited by 63 · see citing works
Krejčí, A., Hupp, T. R., Lexa, M., et al. (2015) · Bioinformatics · doi:10.1093/bioinformatics/btv522 · Cited by 60 · see citing works
A novel method for purification of recombinant adenoassociated virus vectors on a large scale
Wu, X., Dong, X., Wu, Z., et al. (2001) · Chinese Science Bulletin · doi:10.1007/bf03187263 · Cited by 52 · see citing works
Streck, C. J., Dickson, P. V., Ng, C. Y., Zhou, J., Gray, J. T., Nathwani, A. C., & Davidoff, A. M. (2005) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-05-0502 · Cited by 47 · see citing works
Marsic, D., Méndez‐Gómez, H. R., & Zolotukhin, S. (2015) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2015.41 · Cited by 34 · see citing works
Davidsson, M., Díaz-Fernández, P., Schwich, O. D., Torroba, M., Wang, G., & Björklund, T. (2016) · Scientific Reports · doi:10.1038/srep37563 · Cited by 27 · see citing works
Self-citationXu, M., Li, J., Xie, J., He, R., Su, Q., Gao, G., & Tai, P. W. L. (2019) · Human Gene Therapy · doi:10.1089/hum.2018.253 · Cited by 11 · see citing works
Molecular barcoding of viral vectors enables mapping and optimization of mRNAtrans-splicing
Davidsson, M., Díaz-Fernández, P., Torroba, M., Schwich, O. D., Aldrin-Kirk, P., Quintino, L., Heuer, A., Wang, G., Lundberg, C., & Björklund, T. (2018) · RNA · doi:10.1261/rna.063925.117 · Cited by 7 · see citing works
Self-citationShortland, P. J., Wang, H. F., & Molander, C. (1998) · Journal of Neurocytology · doi:10.1023/a:1006980814321 · Cited by 6 · see citing works
Flagged first, then by citations
Research works printed but not linked: 1
- [40]Davidsson M., BRAVE AAV library for in vivo screening of retrograde transport in the CNS. National Center for Biotechnology Information (NCBI) Sequence Read Archive (SRA). https://www.ncbi.nlm.nih.gov/bioproject/PRJNA473475. Deposited 29 May 2018.
Cited by
Sorted by citations
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
Ronzitti, G., Gross, D.-A., & Mingozzi, F. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.00670 · Cited by 357 · see citing works
Weinmann, J., Weis, S., Sippel, J., Tulalamba, W., Remes, A., El Andari, J., Herrmann, A.-K., Pham, Q. H., Borowski, C., Hille, S., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-19230-w · Cited by 259 · see citing works
Adeno-Associated Virus Toolkit to Target Diverse Brain Cells
Challis, R. C., Ravindra Kumar, S., Chen, X., Goertsen, D., Coughlin, G. M., Hori, A. M., Chuapoco, M. R., Otis, T. S., Miles, T. F., & Gradinaru, V. (2022) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-111020-100834 · Cited by 148 · see citing works
Journey to the Center of the Cell: Tracing the Path of AAV Transduction
Dhungel, B. P., Bailey, C. G., & Rasko, J. E. J. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2020.09.010 · Cited by 122 · see citing works
Francis, J. S., Markov, V., Wojtas, I. D., Gray, S., McCown, T., Samulski, R. J., Figueroa, M., & Leone, P. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2021.01.009 · Cited by 41 · see citing works
“Although it is possible to utilize promoter elements that limit transgene expression to specific cellular lineages, the capsid-cell surface interaction is the primary determinant of AAV tropism.(1,2) Most native AAV serotypes are inherently neurotropic in the main, but engineered capsid variants have the potential to”
Background
- Authors
- Davidsson, M., Wang, G., Aldrin-Kirk, P., Cardoso, T., Nolbrant, S., Hartnor, M., Mudannayake, J., Parmar, M., & Björklund, T.
- Affiliations
- Lund University (SE)
- Corresponding author
- Björklund, Tomas
- ORCID
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2019-12-09
- Volume
- 116
- Issue
- 52
- Pages
- 27053-27062
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC6936499
- OpenAlex
- W2992974653
- Cited by
- 142
- References
- 72
- Citations by year
- 2026: 15 · 2025: 7 · 2024: 16 · 2023: 20 · 2022: 23 · 2021: 32 · 2020: 24 · 2019: 2 · 2018: 1 · 2013: 1
- FWCI
- 12.16
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion, cc-by-nc-nd)
- bioRxiv (Cold Spring Harbor Laboratory) (acceptedVersion)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Lund University Publications (Lund University) (submittedVersion, other-oa)
- Funders
- Parkinson's Disease Foundation (PDF-IRG-1303)
- Åke Wiberg Stiftelse
- Tore Nilsons Stiftelse för Medicinsk Forskning
- Vetenskapsrådet (ÄR-MH-2016-01997, K2014-79X-22510-01-1)
- Åhlén-stiftelsen
- Magnus Bergvalls Stiftelse
- Science for Life Laboratory
- Parkinsonfonden
- 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, AAV capsid engineering, Brave, in vivo selection, DNA barcoding, peptide display, retrograde axonal transport, dopaminergic neurons, viral tropism, gene therapy vectors
About these checks
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.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
