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Gene therapies for polyglutamine diseases


Matos C. et al, (2020), Polyglutamine Disorders

Extracellular microRNAs exhibit sequence-dependent stability and cellular release kinetics.

Journal article

Coenen-Stass AML. et al, (2019), RNA Biol, 16, 696 - 706

Safety profile of the intravenous administration of brain-targeted stable nucleic acid lipid particles

Journal article

Conceição M. et al, (2016), Data in Brief, 6, 700 - 705

Overexpression of Mutant Ataxin-3 in Mouse Cerebellum Induces Ataxia and Cerebellar Neuropathology

Journal article

Nóbrega C. et al, (2013), The Cerebellum, 12, 441 - 455