CRISPR/Cas9 Editing for Gaucher Disease Modelling

CRISPR/Cas9 Editing for Gaucher Disease Modelling

Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by mutations in the acid β-glucosidase gene (<i>GBA1</i>). Besides causing GD, <i>GBA1</i> mutations constitute the main genetic risk factor for developing Parkinson’s disease. The molecular basi...

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Bibliographic Details
Main Authors: Eleonora Pavan, Maximiliano Ormazabal, Paolo Peruzzo, Emilio Vaena, Paula Rozenfeld, Andrea Dardis
Format: Article
Language:English
Published: MDPI AG 2020-05-01
Series:International Journal of Molecular Sciences
Subjects:
Gaucher disease
cellular model
acid β-glucosidase
CRISPR/Cas9
unfolded protein response
neuroinflammation
Online Access:https://www.mdpi.com/1422-0067/21/9/3268

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https://www.mdpi.com/1422-0067/21/9/3268

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