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Gene Therapy

Research from Queen Mary University of London, William Harvey Research Institute has provided new data on gene therapy

Published in Drug Week, May 1st, 2009

New investigation results, 'The ataxia protein sacsin is a functional co-chaperone that protects against polyglutamine-expanded ataxin-1,' are detailed in a study published in Human Molecular Genetics. According to recent research published in the journal Human Molecular Genetics, "An extensive protein-protein interaction network has been identified between proteins implicated in inherited ataxias. The protein sacsin, which is mutated in the early-onset neurodegenerative disease autosomal recessive spastic ataxia of Charlevoix-Saguenay, is a node in this interactome."

"Here, we have established the neuronal expression of sacsin and functionally...

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