Imagine a future where genetic anomalies can be precisely targeted and corrected using genome editing – a giant leap from our ability to sequence or read human genomes two decades ago. The world of medicine is currently abuzz with news of regulatory agencies’ approval for two highly anticipated CRISPR-based therapies for sickle-cell disease and β-thalassaemia in the U.K. and the U.S.
The approval is groundbreaking because it augurs an era that could transform the lives of millions of patients and families grappling with these inherited blood disorders. To put this in perspective, more than a million people worldwide suffer from thalassemia, of whom 100,000 depend on regular blood transfusions. Another 20 million people around the world are estimated to be suffering from sickle-cell anaemia.
