CRISPR-Cas systems: Overview, innovations and applications in human disease research and gene therapy

• Main Authors: Yuanyuan Xu, Zhanjun Li
• Format: Article
• Language: English
• Published: Elsevier 2020-01-01
• Series: Computational and Structural Biotechnology Journal
• Subjects: CRISPR; Cas9; Genome editing; Human disease models; Rabbit; Gene therapy
• Online Access: http://www.sciencedirect.com/science/article/pii/S2001037020303846

Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems.