Project description
A gene-editing treatment for Alexander disease
Alexander disease (AxD), an autosomal dominant neurodegenerative disorder, is caused by missense mutations in the gene encoding the glial fibrillary acidic protein (GFAP), a key intermediate filament protein of astrocytes. This leads to the formation of GFAP aggregates and the activation of the stress response, compromising central nervous system homeostasis. The EU-funded ASTRO-EDITING project aims to recover astrocyte function by correcting GFAP mutational hotspots. For this purpose, scientists will follow a gene-editing approach based on adeno-associated virus vectors. The project will provide proof-of-concept of in vivo editing in astrocytes as a potential gene therapy strategy for AxD, offering at the same time a novel therapeutic approach for the treatment of other neurodegenerative disorders.
Fields of science
Programme(s)
Funding Scheme
MSCA-IF-EF-SE - Society and Enterprise panel
Coordinator
20132 Milano
Italy
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