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.

This study uses base editing to correct a Pde6b mutation in a mouse model of retinitis pigmentosa, restoring photoreceptor function and visual ability.

This study demonstrates that CRISPR/Cas9-mediated gene conversion can efficiently repair genetic mutations in hematopoietic stem cells without requiring exogenous DNA templates.