Article

Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model

2024-04-19

Abstract excerpt

<title>Abstract</title> <p>Almost all attempts to date at gene therapy approaches for monogenetic disease have used the amino acid sequences of the natural protein. In the current study, we use a designed, thermostable form of glucocerebrosidase (GCase), the enzyme defective in Gaucher disease (GD), to attempt to alleviate neurological symptoms in a GD mouse that models type 3 disease, i.e. the chronic neuronopat...

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Literature Corpus work: b2ad9e53-94e9-5628-9e1d-a9ff73139f10
DOI: 10.21203/rs.3.rs-4210702/v1

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