Beyond the Cut: Long-read sequencing reveals complex genomic and transcriptomic changes in AAV-CRISPR therapy for Duchenne Muscular Dystrophy
Mary S. Jia, Made Harumi Padmaswari, Landon A. Burcham, Shilpi Agrawal, Gabrielle N. Bulliard, Abbey L. Stokes, Christopher E. Nelson
This study uses long-read sequencing to analyze genomic and transcriptomic changes in AAV-CRISPR therapy for DMD, revealing complex editing outcomes and developing new methods for detection.
bioRxiv · Aug 1, 2025 · PreprintBioengineering