CORDIS Project
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This project focuses on developing a gene therapy for Alexander disease, a neurodegenerative disorder caused by mutations in the GFAP gene. The aim is to use gene editing techniques to correct these mutations in astrocytes, potentially restoring normal function and offering a new treatment avenue for this orphan diseas…
Alexander disease (AxD) is an autosomal dominant neurodegenerative disorder caused by missense mutations in the gene encoding the glial fibrillary acidic protein (GFAP), the major intermediate filament protein in astrocytes.
Accumulation of GFAP aggregates in Rosenthal fibres leads to impairment of proteasomal activity and hyperactivation of the stress response, thus compromising astrocyte functions and altering the homeostasis of the central nervous system (CNS).
Currently, this orphan disease…
OSPEDALE SAN RAFFAELE SRL
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