Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b
Afrooz Rashnonejad, Manal Farea, Gholamhossein Amini Chermahini, Gérald Coulis, Noah D. Taylor, Allison M. Fowler, S. Armando Villalta, Oliver D. King, 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.
bioRxiv · Dec 18, 2024 · PreprintMolecular Biology