Research Progress and Application of iPSC Combined with CRISPR/Cas9 in Genetic Disease

TIAN Xinyuan^1,2, ZHANG Chuan¹, SUN Xiaohong³, HUI Ling^1
*

( ¹Gansu Provincial Clinical Research Center for Birth Defects and Rare Diseases, Medical Genetics Center, Maternity and Childcare Hospital of Gansu, Lanzhou 730050, China; ² School of Public Health, Gansu University of Chinese Medicine, Lanzhou 730000, China; ³ Maternity and Childcare Hospital of Lanzhou, Lanzhou 730000, China)

Abstract:

The CRISPR/Cas9 (regular clustering of short palindrome repeats/Cas9) technology combined with iPSC (induced pluripotent stem cell) can establish various genetic disease models to study the molecular mechanism of disease occurrence or perform high-throughput drug screening. It can also correct disease-causing mutations, laying a research foundation for the development of cell and gene therapy, personalized regenerative medicine and living biomedicine. However, there are still many problems to be solved in the combination of the two. In this paper, the latest progress of iPSC and CRISPR/Cas9 technology are summarized, as well as the combination of the two in human genetic disease modeling, cell and gene replacement therapy, and the applications of biosensor. At the same time, the great challenges and solutions are also discussed.

CSTR: 32200.14.cjcb.2023.08.0008