Ana Carolina da Silva

This paper develops and tests rAAV-delivered bicistronic artificial microRNAs to selectively silence mutant ATXN3 in SCA3 mouse models, showing improved motor function and reduced mutant protein aggregates.

This study evaluates AAV9-miR-ATXN3 in a non-human primate model of SCA3, showing reduced neuropathology through allele-specific silencing of mutant ATXN3.

This paper optimizes size exclusion chromatography (SEC) for isolating biologically active EV-AAVs for non-invasive brain gene delivery, showing higher recovery and transgene expression efficiency compared to differential ultracentrifugation.