Genome Editing with the CRISPR-Cas9 System: Principles and Applications

The CRISPR-Cas9 systems are revolutionizing studies of gene functionand possibilities of gene therapy. In bacteria, natural CRISPR-Cas9systems provide adaptive immunity by cleaving foreign DNA. Cas9 isan RNA-guided endonuclease and artificial guide RNAs can easily bedesigned to cleave target any DNA sequence of interest. When used forgenome editing, the CRISPR/Cas9 system allows making a great varietyof DNA sequence modifications (deletions to inactivate gene functionor potential regulatory sequences, insertions of reporter genes, pointmutations, etc). We have developed online tools for selection of guideRNAs in more than a 300 species (crispor.org) and helped to performgenome editing in many experimental systems. Examples from our lab, incultured cells, and from collaborations, in model organisms such as therat and zebrafish, will be shown to illustrate a wide range of applicationsof genome editing for the study of gene function and genetic diseases.

کلیدواژه ها:

CRISPR-Cas9 ، Gene Therapy