Article

Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based Brain Shuttle construct

2022-04-26

Abstract excerpt

<title>Abstract</title> <p>Biallelic mutations in GBA1 that lead to reduced β-glucocerebrosidase (GCase) activity result in the monogenic lysosomal storage disease Gaucher disease (GD). Variants in one GBA1 allele are the most common genetic risk factor for multiple synucleinopathies including Parkinson’s disease (PD). Therapies to increase GCase activity in the brain hold great promise for the treatment of these...

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Literature Corpus work: c0d06eda-6d4b-5359-9c43-7e84a65f0ba7
DOI: 10.21203/rs.3.rs-1490073/v1

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