Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3
David Rufino‐Ramos, Patrícia Albuquerque, Kevin Leandro, Vítor Carmona, Inês M. Martins, Rita Fernandes, Carina Henriques, Diana D Lobo, Rosário Faro, Rita Perfeito, Liliana Mendonça, Dina Pereira, Célia Gomes, Rui Jorge Nobre, Luís Pereira de Almeida
Topics & Concepts
Spinocerebellar ataxiaMachado–Joseph diseaseTrinucleotide repeat expansionGene silencingBiologymicroRNAAtaxiaGeneticsCell biologyNeuroscienceGeneAlleleExtracellular vesicles in diseaseRNA Research and SplicingRNA regulation and disease