De Almeida and colleagues engineered extracellular vesicles (EVs) with neuronal targeting properties carrying artificial miRNAs targeting mutant ataxin-3 (mutATXN3) mRNA. Therapeutic EVs were shown to silence mutATXN3 in vitro and downregulate mutant ataxin-3 species in an MJD/SCA3 animal model upon daily intranasal administration.
All Keywords
【저자키워드】 miRNA, Extracellular vesicles, Gene therapy, intranasal, ataxin-3, ExoMotif, MJD, SCA3, spinocerebellar ataxia type 3,
【저자키워드】 miRNA, Extracellular vesicles, Gene therapy, intranasal, ataxin-3, ExoMotif, MJD, SCA3, spinocerebellar ataxia type 3,