Sara M. Lopes

Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders.

Protocol for the Characterization of the Cytosine-Adenine-Guanine Tract and Flanking Polymorphisms in Machado-Joseph Disease: Impact on Diagnosis and Development of Gene-Based Therapies.

ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease.

miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3.

A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification.