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Frontiers in genetics
2024;15 1364742.

Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy.

Man-Ling Zhang, Hong-Bin Li, Yong Jin
Author Information
  1. Man-Ling Zhang: Department of Rheumatology and Immunology, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.
  2. Hong-Bin Li: Department of Rheumatology and Immunology, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.
  3. Yong Jin: Department of Rheumatology and Immunology, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.
PMID: 38666293 DOI: 10.3389/fgene.2024.1364742

Abstract

The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) mediated Cas9 nuclease system has been extensively used for genome editing and gene modification in eukaryotic cells. CRISPR/Cas9 technology holds great potential for various applications, including the correction of genetic defects or mutations within the human genome. The application of CRISPR/Cas9 genome editing system in human disease research is anticipated to solve a multitude of intricate molecular biology challenges encountered in life science research. Here, we review the fundamental principles underlying CRISPR/Cas9 technology and its recent application in neurodegenerative diseases, cardiovascular diseases, autoimmune related diseases, and cancer, focusing on the disease modeling and gene therapy potential of CRISPR/Cas9 in these diseases. Finally, we provide an overview of the limitations and future prospects associated with employing CRISPR/Cas9 technology for diseases study and treatment.

Keywords

CRISPR/Cas9 autoimmune-related diseases cancer cardiovascular diseases diseases modeling gene therapy neurodegenerative diseases

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