Children’s Hospital of Philadelphia researchers discover underlying mechanisms that make CRISPR an effective gene editing tool
Philadelphia, October 28, 2024 – CRISPR/Cas9 is a gene editing tool that has revolutionized biomedical research and led to the first FDA-approved CRISPR-based gene therapy. However, until now, the precise mechanism of exactly how this tool works and avoids creating detrimental off-target effects was not well understood. Now, using state-of-the-art technology, researchers from Children’s Hospital of Philadelphia (CHOP) have identified several specific steps needed for CRISPR to become active and perform its gene editing function. These preclinical findings could lead to improved designs for CRISPR-based ...











