CORDIS Project
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This project aims to develop gene therapy vectors using the CRISPR/Cas9 system to target and eliminate a genetic mutation linked to amyotrophic lateral sclerosis and frontotemporal dementia. The goal is to create potential therapies that could also benefit other neurodegenerative diseases.
Expansion of a hexanucleotide repeat G4C2 in the non-coding region of chromosome 9 open reading frame 72 (C9orf72) is the most common genetic cause for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). ALS is a fatal condition characterized by progressive motor deficits, degeneration of upper and lower motor neurons (MNs) and death from neuromuscular respiratory failure in the majority of afflicted individuals within 3-5 years.
Currently, the economic burden of care and trea…
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