Efficient Gene Editing for Heart Disease via ELIP-Based CRISPR Delivery System
February 2024
in “
Pharmaceutics
”
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.