Therapeutic applications of CRISPR RNA-guided genome editing

Abstract

The rapid development of programmable nuclease-based genome editing technologies has enabled targeted gene disruption and correction both in vitro and in vivo. This revolution opens up the possibility of precise genome editing at target genomic sites tomodulate gene function in animals and plants. Among several programmable nucleases, the type II clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated nuclease 9 (Cas9) system has progressed remarkably in recent years, leading to its widespread use in research, medicine and biotechnology. In particular, CRISPR-Cas9 shows highly efficient gene editing activity for therapeutic purposes in systems ranging frompatient stemcells to animalmodels. However, the development of therapeutic approaches and deliverymethods remains a great challenge for biomedical applications. Herein, we reviewtherapeutic applications that use the CRISPR-Cas9 systemand discuss the possibilities and challenges ahead.