Santana, Magda M.Paixão, SusanaCunha-Santos, JaneteSilva, Teresa PereiraTrevino-Garcia, AllysonGaspar, Laetitia S.Nóbrega, ClévioNobre, Rui JorgeCavadas, CláudiaGreif, HagarPereira de Almeida, Luís2020-04-302020-04-3020201479-5876http://hdl.handle.net/10400.1/13814Machado-Joseph disease (MJD), also known as spinocerebellar ataxia type 3, is the most common of the dominantly inherited ataxias worldwide and is characterized by mutant ataxin-3 aggregation and neuronal degeneration. There is no treatment available to block or delay disease progression. In this work we investigated whether trehalose, a natural occurring disaccharide widely used in food and cosmetic industry, would rescue biochemical, behavioral and neuropathological features of an in vitro and of a severe MJD transgenic mouse model.engMachado-Joseph diseaseAutophagySpinocerebellar ataxia type 3Polyglutamine disorderTrehaloseTrehalose alleviates the phenotype of Machado–Joseph disease mouse modelsjournal article10.1186/s12967-020-02302-2