CRISPR/Cas9 Editing for Gaucher Disease Modelling
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DOI: 10.3390/ijms21093268
Abstract: Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by mutations in the acid β-glucosidase gene (GBA1). Besides causing GD, GBA1 mutations constitute the main genetic risk factor for developing Parkinson’s disease. The… read more here.
Keywords: gaucher disease; crispr cas9; cas9 editing; disease ... See more keywords