CRISPR/Cas System in Human Genetic Diseases

Abstract

Clustered regularly interspaced short palindromic repeats/CRISPR-associated CRISPER/Cas system, as the current most popular gene-editing technology, shows great advantages of simple composition, good specificity and high cutting efficiency compared with other gene editing technology. With the rapid development of CRISPR-Cas systems, such as Cas9, Cas12a and Cas12f, can be used to edit the DNA of eukaryotic cells, and then successively found that Cas13a, Cas13b and Cas13d are targeted to the RNA merons. Through various modifications, scientists also developed a new type of the CRISPR-Cas system. With higher DNA-cutting activity, greater specificity, and smaller size than the natural CRISPR system, these engineered gene-editing systems form a powerful tool set for DNA sequence knockout, replacement, epigenetic editing, and even the activation and suppression of gene expression. Despite the potential problems in the practical application of CRISPR technology to be solved, it is believed that with further improvement, the CRISPR treatment technology will play a more important role in the prevention and treatment of human diseases, more perfectly and precisely. This review introduces the structure, functional mechanism and application of CRISPR/Cas system in human genetic diseases, and the current status and development of CRISPR/Cas system are summarized and prospected.