Efficient Gene Editing for Heart Disease via ELIP-Based CRISPR Delivery System

    February 2024 in “ Pharmaceutics
    Xing Yin, Romain Harmancey, Brion Frierson, Jean Wu, Melanie R. Moody, David D. McPherson, Shaoling Huang
    The study explores the use of echogenic liposomes (ELIP) as carriers for CRISPR/Cas9 complexes to improve gene delivery in cardiovascular therapy. In experiments conducted on mouse neonatal ventricular myocytes and rat hearts, ELIP containing a decoy oligodeoxynucleotide against NF-κB showed limited gene delivery without ultrasound. However, the combination of ELIP and ultrasound significantly enhanced gene penetration into heart cells and tissues. Further tests with ELIP delivering Cas9-sg-IL1RL1 RNA demonstrated successful gene editing, which was further improved by ultrasound. This method holds potential for advancing CRISPR-based gene therapy for heart disease.
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