Day: December 12, 2023
ARCUS-mediated excision of the “hot spot” region of the human dystrophin gene results in functional improvement in a mouse model of Duchenne muscular dystrophy (DMD)
Owens, G., et al. European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress.
Unique features of ARCUS nucleases enable high efficiency, targeted gene insertion in vivo
Gorsuch, C., et al. European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress.