Results for "Medicine & Health"

ADPKD gene therapy: A precise edit for a complex organ

Researchers demonstrated that a single dose of an adenine base editor, delivered via AAV9, successfully corrected the pathogenic mutation in a mouse model of ADPKD. The study highlights that both broad and kidney-specific gene editing can delay cyst growth and improve survival rates.

By Cheng, Li, Zhou, Harris, Calvet, Li