Pupo A, Fernandez A, Low SH, Francois A, Suarez-Amaran L, Samulski RJ. AAV vectors: the Rubik’s cube of human gene therapy. Mol Ther. 2022;30:3515–41.
Article CAS PubMed PubMed Central Google Scholar
Li W, Asokan A, Wu Z, Van Dyke T, DiPrimio N, Johnson JS, et al. Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther. 2008;16:1252–60.
Article CAS PubMed Google Scholar
Koerber JT, Jang JH, Schaffer DV. DNA shuffling of adeno-associated virus yields functionally diverse viral progeny. Mol Ther. 2008;16:1703–9.
Article CAS PubMed Google Scholar
Herrmann AK, Bender C, Kienle E, Grosse S, El Andari J, Botta J, et al. A robust and all-inclusive pipeline for shuffling of adeno-associated viruses. ACS Synth Biol. 2019;8:194–206.
Article CAS PubMed Google Scholar
Kuklik J, Michelfelder S, Schiele F, Kreuz S, Lamla T, Muller P, et al. Development of a bispecific antibody-based platform for retargeting of capsid modified AAV vectors. Int J Mol Sci. 2021;22:8355.
Eichhoff AM, Borner K, Albrecht B, Schafer W, Baum N, Haag F, et al. Nanobody-enhanced targeting of AAV gene therapy vectors. Mol Ther Methods Clin Dev. 2019;15:211–20.
Article CAS PubMed PubMed Central Google Scholar
Hartmann J, Munch RC, Freiling RT, Schneider IC, Dreier B, Samukange W, et al. A library-based screening strategy for the identification of DARPins as ligands for receptor-targeted AAV and lentiviral vectors. Mol Ther Methods Clin Dev. 2018;10:128–43.
Article CAS PubMed PubMed Central Google Scholar
Perabo L, Buning H, Kofler DM, Ried MU, Girod A, Wendtner CM, et al. In vitro selection of viral vectors with modified tropism: the adeno-associated virus display. Mol Ther. 2003;8:151–7.
Article CAS PubMed Google Scholar
Muller OJ, Kaul F, Weitzman MD, Pasqualini R, Arap W, Kleinschmidt JA, et al. Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat Biotechnol. 2003;21:1040–6.
Varadi K, Michelfelder S, Korff T, Hecker M, Trepel M, Katus HA, et al. Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors. Gene Ther. 2012;19:800–9.
Article CAS PubMed Google Scholar
Wang Y, Yang C, Hu H, Chen C, Yan M, Ling F, et al. Directed evolution of adeno-associated virus 5 capsid enables specific liver tropism. Mol Ther Nucleic Acids. 2022;28:293–306.
Article CAS PubMed PubMed Central Google Scholar
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.
Article CAS PubMed PubMed Central Google Scholar
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.
Article CAS PubMed PubMed Central Google Scholar
Korbelin J, Dogbevia G, Michelfelder S, Ridder DA, Hunger A, Wenzel J, et al. A brain microvasculature endothelial cell-specific viral vector with the potential to treat neurovascular and neurological diseases. EMBO Mol Med. 2016;8:609–25.
Article PubMed PubMed Central Google Scholar
Krolak T, Chan KY, Kaplan L, Huang Q, Wu J, Zheng Q, et al. A high-efficiency AAV for endothelial cell transduction throughout the central nervous system. Nat Cardiovasc Res. 2022;1:389–400.
Article PubMed PubMed Central Google Scholar
Hordeaux J, Wang Q, Katz N, Buza EL, Bell P, Wilson JM. The neurotropic properties of AAV-PHP.B are limited to C57BL/6J mice. Mol Ther. 2018;26:664–8.
Article CAS PubMed PubMed Central Google Scholar
Liguore WA, Domire JS, Button D, Wang Y, Dufour BD, Srinivasan S, et al. AAV-PHP.B administration results in a differential pattern of CNS biodistribution in non-human primates compared with mice. Mol Ther. 2019;27:2018–37.
Article CAS PubMed PubMed Central Google Scholar
Batista, King AR, Reardon OD, Davis CP, Shankaracharya C, Philip V, et al. Ly6a differential expression in blood-brain barrier is responsible for strain specific central nervous system transduction profile of AAV-PHP.B. Hum Gene Ther. 2020;31:90–102.
Article CAS PubMed Google Scholar
Kremer R, Williams A. AAV-BR1 does not target endothelial cells in Sprague Dawley rats unlike in mice. MicroPubl Biol. 2024;2024. https://doi.org/10.17912/micropub.biology.001120.
Cyranoski D. Marmosets are stars of Japan’s ambitious brain project. Nature. 2014;514:151–2.
Article CAS PubMed Google Scholar
Hoshi Y, Uchida Y, Tachikawa M, Inoue T, Ohtsuki S, Terasaki T. Quantitative atlas of blood-brain barrier transporters, receptors, and tight junction proteins in rats and common marmoset. J Pharm Sci. 2013;102:3343–55.
Article CAS PubMed Google Scholar
‘t Hart BA, Abbott DH, Nakamura K, Fuchs E. The marmoset monkey: a multi-purpose preclinical and translational model of human biology and disease. Drug Discov Today. 2012;17:1160–5.
Article PubMed PubMed Central Google Scholar
Chuapoco MR, Flytzanis NC, Goeden N, Christopher Octeau J, Roxas KM, Chan KY, et al. Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain. Nat Nanotechnol. 2023;18:1241–51.
Korbelin J, Sieber T, Michelfelder S, Lunding L, Spies E, Hunger A, et al. Pulmonary targeting of adeno-associated viral vectors by next-generation sequencing-guided screening of random capsid displayed peptide libraries. Mol Ther. 2016;24:1050–61.
Article PubMed PubMed Central Google Scholar
Ravindra Kumar S, Miles TF, Chen X, Brown D, Dobreva T, Huang Q, et al. Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types. Nat Methods. 2020;17:541–50.
Article CAS PubMed Google Scholar
Lin R, Zhou Y, Yan T, Wang R, Li H, Wu Z, et al. Directed evolution of adeno-associated virus for efficient gene delivery to microglia. Nat Methods. 2022;19:976–85.
Article CAS PubMed Google Scholar
Michelfelder S, Varadi K, Raupp C, Hunger A, Korbelin J, Pahrmann C, et al. Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo. PLoS One. 2011;6:e23101.
Article CAS PubMed PubMed Central Google Scholar
Trepel M, Korbelin J, Spies E, Heckmann MB, Hunger A, Fehse B, et al. Treatment of multifocal breast cancer by systemic delivery of dual-targeted adeno-associated viral vectors. Gene Ther. 2015;22:840–7.
Article CAS PubMed Google Scholar
Naumer M, Ying Y, Michelfelder S, Reuter A, Trepel M, Muller OJ, et al. Development and validation of novel AAV2 random libraries displaying peptides of diverse lengths and at diverse capsid positions. Hum Gene Ther. 2012;23:492–507.
Article CAS PubMed Google Scholar
Perabo L, Goldnau D, White K, Endell J, Boucas J, Humme S, et al. Heparan sulfate proteoglycan binding properties of adeno-associated virus retargeting mutants and consequences for their in vivo tropism. J Virol. 2006;80:7265–9.
Article CAS PubMed PubMed Central Google Scholar
Korbelin J, Hunger A, Alawi M, Sieber T, Binder M, Trepel M. Optimization of design and production strategies for novel adeno-associated viral display peptide libraries. Gene Ther. 2017;24:470–81.
Article CAS PubMed Google Scholar
Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Viro
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