rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3
Ana Carolina da Silva, Carina Henriques, Diana Duarte Lobo, Ana Rita Fernandes, Miguel Monteiro Lopes, Kevin Leandro, Dina Pereira, Sónia Duarte, Sara Lopes, Magda M. Santana, Amal Dakka, Steve De Marco, Marla L. Weetall, Jana Narasimhan, Anu Bhattacharyya, , Luís Pereira de Almeida
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.
bioRxiv · Oct 2, 2025 · PreprintMolecular Biology