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CYP46A1- gene therapy improves Machado-Joseph disease in mouse models
Publication . Nóbrega, Clévio; Mendonca, L. S.; Marcelo, Adriana; Lamaziere, A.; Tome, S.; Despres, G.; Matos, C.; Mechmet, Fatich; Langui, D.; den Dunnen, W.; de Almeida, L. P.; Cartier, N.; Alves, S.
Aims/Context: Machado-Joseph Disease (MJD) is a neurodegenerative disease associated with extensive neuronal death. Defects in brain cholesterol metabolism may contribute to neurodegenerative diseases.

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Funding agency

Fundação para a Ciência e a Tecnologia

Funding programme

SFRH

Funding Award Number

SFRH/BPD/97414/2013

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