Gene editing extends lifespan in mouse model of prion disease
Broad Institute
JANUARY 14, 2025
The treatment, which uses base editing to make a single-letter change in DNA, reduced levels of the disease-causing prion protein in the brain by as much as 60 percent. This has the potential to be a really promising strategy. In the future, the team hopes to make the base-editing cargo smaller, because dual AAVs can be costly to produce.
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