Molecular BiologybioRxivPreprint
Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b
Afrooz Rashnonejad, Manal Farea, Gholamhossein Amini Chermahini…·Scott Q. Harper
This study shows CRISPR-Cas13b effectively reduces DUX4 in FSHD but is limited by immune responses that decrease Cas13b levels over time.