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A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Tordo J, O'Leary C, Antunes ASLM, Palomar N, Aldrin-Kirk P, Basche M, Bennett A, D'Souza Z, Gleitz H, Godwin A, Holley RJ, Parker H, Liao AY, Rouse P, Youshani AS, Dridi L, Martins C, Levade T, Stacey KB, Davis DM, Dyer A, Clément N, Björklund T, Ali RR, Agbandje-McKenna M, Rahim AA, Pshezhetsky A, Waddington SN, Linden RM, Bigger BW, Henckaerts E. Tordo J, et al. Brain. 2018 Jul 1;141(7):2014-2031. doi: 10.1093/brain/awy126. Brain. 2018. PMID: 29788236 Free PMC article.
Recombinant adeno-associated viruses (AAVs) are popular in vivo gene transfer vehicles. ...Here we describe an alternative approach to capsid engineering, which draws on the natural evolution of the virus and aims to yield capsids that are bette …
Recombinant adeno-associated viruses (AAVs) are popular in vivo gene transfer vehicles. ...Here we describe an alternative app …
Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties.
Hsu HL, Brown A, Loveland AB, Lotun A, Xu M, Luo L, Xu G, Li J, Ren L, Su Q, Gessler DJ, Wei Y, Tai PWL, Korostelev AA, Gao G. Hsu HL, et al. Nat Commun. 2020 Jun 30;11(1):3279. doi: 10.1038/s41467-020-17047-1. Nat Commun. 2020. PMID: 32606306 Free PMC article.
Recombinant adeno-associated viruses (rAAVs) are currently considered the safest and most reliable gene delivery vehicles for human gene therapy. ...Here, we describe a novel capsid identified in a human clinical sample by high-throughput, long-read se …
Recombinant adeno-associated viruses (rAAVs) are currently considered the safest and most reliable gene delivery vehicles for …
A combination of mutations enhances the neurotropism of AAV-2.
Xu J, Ma C, Bass C, Terwilliger EF. Xu J, et al. Virology. 2005 Oct 25;341(2):203-14. doi: 10.1016/j.virol.2005.06.051. Epub 2005 Aug 15. Virology. 2005. PMID: 16102794 Free article.
Our objective was to generate innocuous gene vehicles that would be effectively taken up by axons and then home to the neuron cell bodies. Vectors derived from Adeno-Associated Virus (AAV), a harmless human parvovirus, offer strong starting candidates for der …
Our objective was to generate innocuous gene vehicles that would be effectively taken up by axons and then home to the neuron cell bodies. V …
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