Gene Editing Extends Prion Disease Lifespan

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Gene editing

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Science

Gene editing extends lifespan in mouse model of prion disease

Summary

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79% Informative

A single-letter edit in DNA reduces levels of the disease-causing prion protein in the brain and could lead to a preventative, one -time treatment for the deadly neurodegenerative disorder.

There is currently no cure for prion disease, and the new approach could be important step towards treatments that prevent the disease or slow its progression in patients who have already developed symptoms.

The team developed a pair of adeno -associated viruses to package and deliver the base-editing machinery to brain cells.

They then administered the AAVs to mice infected with the human prion protein.

On average, the system installed the R37X edit in 37 percent of copies of the gene.

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