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Small Molecule Chaperones for the Treatment of Gaucher Disease and GBA1-Associated Parkinson Disease

Parkinson disease, the second most common movement disorder, is a complex neurodegenerative disorder hallmarked by the accumulation of alpha-synuclein, a neural-specific small protein associated with neuronal synapses. Mutations in the glucocerebrosidase gene (GBA1), implicated in the rare, autosoma...

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Auteurs principaux: Tae-Un Han, Richard Sam, Ellen Sidransky
Format: Artigo
Langue:Inglês
Publié: Frontiers Media S.A. 2020-05-01
Collection:Frontiers in Cell and Developmental Biology
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Accès en ligne:https://www.frontiersin.org/article/10.3389/fcell.2020.00271/full
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