In vivo base editing extends lifespan of a humanized mouse model of prion disease | Nature Medicine
An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion isolate.
Download PDF Subjects Prion diseases Targeted gene repair An Author Correction to this article was published on 30 January 2025 This article has been updated Abstract Prion disease is a fatal neurodegenerative disease caused by the misfolding of prion protein (PrP) encoded by the PRNP gene. While there is currently no cure for the disease, depleting PrP in the brain is an established strategy to prevent or stall templated misfolding of PrP. Here we developed in vivo cytosine and adenine base strategies delivered by adeno-associated viruses to permanently modify the PRNP locus to achieve PrP kn
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