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

Autor: Man-Ling Zhang, Hong-Bin Li, Yong Jin
Předmět:
Zdroj: Frontiers in Genetics; 2024, p1-14, 14p
Abstrakt: 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. [ABSTRACT FROM AUTHOR]
Databáze: Complementary Index