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Enzyme therapy for fatal brain disorder

Updated: Oct 18, 2022

Research in animals has provided key clues for treating infantile Batten disease (CLN1 disease), an incurable brain disorder that can result in dementia, blindness and early death.

A study by Washington University School of Medicine in St Louis, Missouri, USA, and the Roslin Institute at the University of Edinburgh, UK, has shown that adding an enzyme (PPT1), which is missing in CLN1 disease, can slow brain degeneration in mice and sheep with the same genetic mutation.

Prof Jonathan Cooper, at Washington University said: “Our work has shown the potential for a new therapy to treat this devastating fatal disease.

“Not only did we improve the disease in mice, but we were successful in scaling it up to have similar partial efficacy in the much larger brain of a sheep model of the same disease.”


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