CRISPR-Cas9 From bacterial immune defense to genetic engineering

Abstract

Awarded by the Nobel Prize in Chemistry, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)-Cas9 (CRISPR-associated) technology has become a standard in basic research in less than 10 years. Derived from a natural bacterial defense mechanism, the CRISPR-Cas technique enables to modify the genome easily and very precisely. The original technique, which includes knock-out and knock-in approaches, has led to CRISPRa and CRISPRi variants, which respectively activate or repress specific gene. In basic research, CRISPR can be used to generate genetically modified cells and animal models to study gene function and reproduce genetic diseases. In clinical research, CRISPR opens up the possibility of new gene therapies to treat a wide range of genetic, oncological and infectious diseases. However, this revolutionary technique raises a number of ethical questions, and remains closely regulated, both scientifically and ethically.