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

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

Pharmaceutics

Division of Cardiology, Department of Internal Medicine, McGovern Medical School, The University of Texas Health Science Center at Houston, Houston, TX 77030, USA.

Published: February 2024

AI Article Synopsis

  • Liposomes can effectively deliver CRISPR/Cas9 complexes for gene therapy targeting cardiovascular diseases, but efficiently getting these materials into heart cells, or cardiomyocytes, is still a challenge.
  • Echogenic liposomes (ELIP) loaded with specific RNA were tested both in lab settings with mouse heart cells and in live rat hearts to evaluate how well they delivered their genetic cargo with and without the use of ultrasound.
  • Results showed that using ultrasound alongside these liposomes significantly improved their ability to penetrate heart cells and successfully edit genes, highlighting a promising method for treating heart disease through CRISPR technology.

Article Abstract

Liposomes as carriers for CRISPR/Cas9 complexes represent an attractive approach for cardiovascular gene therapy. A critical barrier to this approach remains the efficient delivery of CRISPR-based genetic materials into cardiomyocytes. Echogenic liposomes (ELIP) containing a fluorescein isothiocyanate-labeled decoy oligodeoxynucleotide against nuclear factor kappa B (ELIP-NF-κB-FITC) were used both in vitro on mouse neonatal ventricular myocytes and in vivo on rat hearts to assess gene delivery efficacy with or without ultrasound. In vitro analysis was then repeated with ELIP containing Cas9-sg-IL1RL1 (interleukin 1 receptor-like 1) RNA to determine the efficiency of gene knockdown. ELIP-NF-κB-FITC without ultrasound showed limited gene delivery in vitro and in vivo, but ultrasound combined with ELIP notably improved penetration into heart cells and tissues. When ELIP was used to deliver Cas9-sg-IL1RL1 RNA, gene editing was successful and enhanced by ultrasound. This innovative approach shows promise for heart disease gene therapy using CRISPR technology.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10974117PMC
http://dx.doi.org/10.3390/pharmaceutics16030343DOI Listing

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