Gene editing preclinical therapy: base editing halts prion protein production

Researchers described base editing strategies for prion disease at the 2026 American Society of Gene and Cell Therapy meeting, building on a Nature Medicine paper. Using cytosine base editors to introduce an early stop codon into the PRNP gene, the approach halted PrP production in a mouse model; the work also emphasized split-base editor delivery via AAV to address packaging limits.