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Targeting ASTROcytes with cutting-edge EDITING technologies to treat the Alexander disease

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.


Net EU contribution
€ 183 473,28
Via Olgettina 60
20132 Milano

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Nord-Ovest Lombardia Milano
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00