CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases.

ABSTRACT: At present, the idea of genome modification has revolutionized the modern therapeutic research era. Genome modification studies have traveled a long way from gene modifications in primary cells to genetic modifications in animals. The targeted genetic modification may result in the modulation (i.e., either upregulation or downregulation) of the predefined gene expression. Clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated nuclease 9 (Cas9) is a promising genome-editing tool that has therapeutic potential against incurable genetic disorders by modifying their DNA sequences. In comparison with other genome-editing techniques, CRISPR-Cas9 is simple, efficient, and very specific. This enabled CRISPR-Cas9 genome-editing technology to enter into clinical trials

SUBMITTER: Sharma G 

PROVIDER: S-EPMC7854284 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature