Clinical trials and promising preclinical applications of CRISPR/Cas gene editing

Treatment of genetic disorders by genomic manipulation has been the unreachable goal of researchers for many decades. Although our understanding of the genetic basis of genetic diseases has advanced tremendously in the last few decades, the tools developed for genomic editing were not efficient and practical for their use in the clinical setting until now. The recent advancements in the research of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein (Cas) systems offered an easy and efficient way to edit the genome and accelerated the research on their potential use in the treatment of genetic disorders. In this review, we summarize the clinical trials that evaluate the CRISPR/Cas systems for treating different genetic diseases and highlight promising preclinical research on CRISPR/Cas mediated treatment of a great diversity of genetic disorders. Ultimately, we discuss the future of CRISPR/Cas mediated genome editing in genetic diseases.

Automated summary

Treatment of genetic disorders through genomic manipulation has long been a goal for researchers. Recent advancements in the CRISPR/Cas systems have provided a more efficient and practical approach for editing the genome and have accelerated research on their potential use in treating genetic diseases. This review summarizes clinical trials and promising preclinical research on CRISPR/Cas-mediated treatment of various genetic disorders, and discusses the future implications of genome editing using CRISPR/Cas in the field of genetic diseases.