Making CRISPR hype more of a reality
The latest CRISPR-based genome editing systems function more like molecular taxis rather than a pair of molecular scissors. This makes them much safer for patients who will soon benefit from gene therapies, as Eric Aird explains.
This year we celebrate 10 years of genome editing with CRISPR. The system is often referred to as molecular scissors, and this designation is quite accurate for its first applications. These short 10 years were marked by stunningly swift development and a great promise to cure thousands of genetic diseases with relative ease – with a single treatment dose that specifically corrects disease- causing DNA mutations in the body’s cells. Sickle cell anaemia and muscular dystrophy are two such diseases. And indeed, a decade later, we are now delivering on that promise in the form of many therapies currently being tested in human clinical trials.