Applications of the CRISPR-Cas-based technology to correct aneuploid and segmental aneuploid cells: The road from lab to clinic

Genetic disorders pose an enormous health threat to human public health. There are presently no disorder-reducing therapy options. The purpose of this article is to review the latest applications and prospects of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated proteins (Cas) (CRISPR-Cas) system in the treatment of human chromosomal disorders. One of the reasons for choosing this topic is because of the lack of related literature. In this review, we first briefly describe a human chromosomal disorder and then outline the molecular architecture and mechanistic basis of CRISPR-Cas9 for its treatment. Then, successful stories in this field are described. The process begins with identifying and cleaving particular sequences on the extra copy of a chromosome. This activates a repair response that guides the cell to eradicate the chromosome without harming the other healthy chromosomes. Thus, instead of editing single genes, scientists can effectively remove a chromosomal abnormality entirely, restoring normal gene expression and cellular function. Although the CRISPR-Cas method has operated well for curing chromosomal disorders in lab settings, the clinical applications are years away because several mechanistic questions have to be answered. Before clinical applications, the methods could be tested in vivo in animal models and adapted to become a keystone for future cell-replacement therapies. Challenges and limitations have to be addressed by geneticists, bioethicists, and policymakers before human translation.