Last month, a CRISPR Therapeutics clinical trial for beta-thalassemia was approved to start treating patients in Europe.
The clinical trial is one of over a dozen that have been initiated since the advent of the CRISPR-Cas9 gene editing technique. Most of these trials are in China, working on the use of CRISPR in cancer, but there are also several underway in the U.S. Globally there are around 60,000 people born each year who have beta-thalassemia, a rare blood condition that causes low oxygen levels in the body and anemia. The CRISPR Therapeutics clinical trial uses CRISPR to turn off the gene that causes this disorder, and instead helps red blood cells produce a form of hemoglobin produced in newborns that isn’t impacted by the mutation.
Read at: Genome Magazine