CRISPR & Thalassaemia

In December, a team that includes researchers from CRISPR Therapeutics and Vertex Pharmaceuticals published promising results from a clinical trial, which is also treating patients in Germany who suffer from a related disease called ß-thalassaemia. In both groups of patients, the treatment seems to be safe, and it so far has eliminated the need for regular blood transfusions. It’s still too soon to say how long the effects will last but the consequences could be huge. Sickle cell disease and ß-thalassaemia are among the most common genetic disorders caused by mutations to a single gene, affecting millions of people worldwide.