4.7 Review

Recent Advances in CRISPR/Cas9-Based Genome Editing Tools for Cardiac Diseases

期刊

出版社

MDPI
DOI: 10.3390/ijms222010985

关键词

genome editing; CRISPR/Cas9 & nbsp; ; base editing; prime editing; cardiac disease

资金

  1. Dutch Heart Foundation [DCVA2017-2018 ARENA-PRIME]
  2. DCVA

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In the past two decades, genome editing has emerged as a powerful tool for modeling and treating diseases, with the CRISPR/Cas9 system becoming the most common tool for both disease modeling and therapeutic studies. Novel tools like base editing and prime editing have further expanded the range and accuracy of achievable genomic modifications.
In the past two decades, genome editing has proven its value as a powerful tool for modeling or even treating numerous diseases. After the development of protein-guided systems such as zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), which for the first time made DNA editing an actual possibility, the advent of RNA-guided techniques has brought about an epochal change. Based on a bacterial anti-phage system, the CRISPR/Cas9 approach has provided a flexible and adaptable DNA-editing system that has been able to overcome several limitations associated with earlier methods, rapidly becoming the most common tool for both disease modeling and therapeutic studies. More recently, two novel CRISPR/Cas9-derived tools, namely base editing and prime editing, have further widened the range and accuracy of achievable genomic modifications. This review aims to provide an overview of the most recent developments in the genome-editing field and their applications in biomedical research, with a particular focus on models for the study and treatment of cardiac diseases.

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