Advances in CRISPR-based technology for the treatment of genetic diseases

Regular clustering of short palindrome repeats (CRISPR) technology can be used to model various genetic diseases in vitro, to study the molecular mechanism of disease or high-throughput drug screening, and to correct disease-causing mutations, laying the research foundation for the development of cellular and gene replacement therapy, personalized regenerative medicine and in vivo biomedicine. However, the technology still has many problems to be solved. This article provides an overview of the development of CRISPR technology and its recent progress in modeling human genetic diseases and gene replacement therapy applications and discusses the technical challenges and solutions.