rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3

Ana Carolina da Silva(Universidade Federal de Ouro Preto), Luís Pereira de Almeida(University of Coimbra)
bioRxiv (Cold Spring Harbor Laboratory)
October 2, 2025
Cited by 0


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