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Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3

Rufino-Ramos, David, Albuquerque, Patrícia R., Leandro, Kevin, Carmona, Vitor, Martins, Inês M., Fernandes, Rita, Henriques, Carina, Lobo, Diana, Faro, Rosário, Perfeito, Rita, Mendonça, Liliana S., Pereira, Dina, Gomes, Célia M., Nobre, Rui Jorge, Pereira de Almeida, Luís·2023·Molecular Therapy
DOI 10.1016/j.ymthe.2023.04.001
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Figure 1 Endogenous miRNAs with ExoMotifs are extensively loaded into EVs
Figure 1 Endogenous miRNAs with ExoMotifs are extensively loaded into EVsCC-BY-NC-ND · From the PDF
Figure 2 ExoMotif and hnRNPA2B1 association with miRNA mutant ataxin-3 (mutATXN3) silencer drives its packaging into EVs and reduces mutATXN3 mRNA levels
Figure 2 ExoMotif and hnRNPA2B1 association with miRNA mutant ataxin-3 (mutATXN3) silencer drives its packaging into EVs and reduces mutATXN3 mRNA levelsCC-BY-NC-ND · From the PDF
Figure 3 RVG peptide expression on the surface of EVs promotes internalization into neurons
Figure 3 RVG peptide expression on the surface of EVs promotes internalization into neuronsCC-BY-NC-ND · From the PDF
Figure 4 Engineered EVs significantly reduce mutATXN3 mRNA in vitro
Figure 4 Engineered EVs significantly reduce mutATXN3 mRNA in vitroCC-BY-NC-ND · From the PDF
Figure 5 Delivery of mirSilencer to the brain significantly decreases mutATXN3 mRNA in dual-luminescent MJD mouse model
Figure 5 Delivery of mirSilencer to the brain significantly decreases mutATXN3 mRNA in dual-luminescent MJD mouse modelCC-BY-NC-ND · From the PDF

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Citations

Cites

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Metadata

Authors
Rufino-Ramos, D., Albuquerque, P. R., Leandro, K., Carmona, V., Martins, I. M., Fernandes, R., Henriques, C., Lobo, D., Faro, R., Perfeito, R., Mendonça, L. S., Pereira, D., Gomes, C. M., Nobre, R. J., & Pereira de Almeida, L.
ORCID
Pereira de Almeida, Luís
Venue
Molecular Therapy
Publisher
Elsevier BV
Published
2023-05
Volume
31
Issue
5
Pages
1275-1292
ISSN
1525-0016
DOI
10.1016/j.ymthe.2023.04.001
Cited by
39
References
11
Language
en
Licence
Funders
  • IMI
  • Fundacao para a Ciencia e a Tecnologia

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