DELAYING NEURODEGENERATION BY CALORIC RESTRICTION APPROACHES: FROM MECHANISMS TO MOLECULAR THERAPY.

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Trehalose alleviates the phenotype of Machado–Joseph disease mouse models

Publication . Santana, Magda M.; Paixão, Susana; Cunha-Santos, Janete; Silva, Teresa Pereira; Trevino-Garcia, Allyson; Gaspar, Laetitia S.; Nóbrega, Clévio; Nobre, Rui Jorge; Cavadas, Cláudia; Greif, Hagar; Pereira de Almeida, Luís

Machado-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.

2020Journal article

Open Access