CRISPR-Cas9 Gene Editing Challenges

Recent research unveiled that a molecule named AZD7648, designed to enhance CRISPR-Cas9 gene editing efficiency, poses significant risks by unintentionally damaging other parts of the genome. While it was anticipated to streamline gene therapies, studies demonstrate that it can lead to extensive genetic changes and the deletion of thousands of DNA bases. The research team led by Jacob Korn highlighted that although localized edits appear correct initially, broader genomic analysis reveals considerable alterations. This revelation has prompted calls for alternative methods to mitigate these risks in the development of gene therapies.