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Updated: Dec 11, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
Published on: August 2, 2018
CRISPR-Cas9 Applications in Cardiovascular Disease
John Paul S Khouzam1, Vijai S Tivakaran2
1University of Notre Dame, Neuroscience and Behavior Undergraduate, Notre Dame, Indiana.
Abstract:
The CRISPR-Cas9 system is an economical and accessible gene-editing technology first discovered as a naturally occurring bacterial immune system. Since its fairly recent discovery, CRISPR-Cas9 system's efficiency and simplicity have been successfully used to edit genomes of living organisms in many fields, working in vitro and in vivo in germline and somatic cells to knock-out harmful mutated genes or in some cases working to knock-in a beneficial gene. A current application of the gene-editing system works against specific mutations that cause certain cardiovascular diseases. However, there are current technical limitations as well as ethical dilemmas in introducing gene-editing to humans. Here, we explore highlights on the current state of research of the CRISPR-Cas9 system through the lens of cardiovascular disease and examine potential untouched applications of the system in the field of cardiology.
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