GeneticsMolecular BiomedicinePeer-reviewed
In vivo adenine base editing corrects newborn murine model of Hurler syndrome
Jing Su, Xiu Jin, Kaiqin She…·Yang Yang
This study corrects a mutation in a mouse model of Hurler syndrome using in vivo adenine base editing, showing reduced GAG accumulation and neurobehavioral deficits.
12 citations