ABSTRACT
The discovery of clustered regularly interspaced short palindromic repeats, or CRISPR for short, is a major breakthrough both for basic biomedical research and for therapeutics, with many long-term implications. This chapter explains how over the course of more than 30 years, the discovery of CRISPR—a novel method for the editing of genes in cells or an organism—began with a series of interesting observations that were followed up by curiosity-based research without any initial clinical goal in mind. Through the persistence and innovation of multiple researchers from countries around the globe, the study of CRISPR gene editing has culminated in a 2020 Nobel award for two researchers, Jennifer Doudna and Emmanuelle Charpentier, who made (and are still making) major contributions to the development of this method of gene editing. This chapter highlights how basic discoveries, coupled with the dogged persistence of teams of researchers, led to the co-opting of this basic bacterial defense system against invading pathogens so that it could be used with exquisite accuracy to target and modify genes. This chapter discusses not only the recent history of the discovery of CRISPR, but also how a basic understanding of the science has within 30 years led to a slew of highly promising clinical trials for many diseases.
