Clinical trial shows gene editing works for β-Thalassaemia, too
Almost as soon as researchers started exploring the capabilities of the CRISPR/Cas9 system, they recognized its potential use in targeted gene editing. But the intervening decades have seen slow progress as people worked to determine how to do so in a way that would be safe for use in humans. It was only a little over two years ago, decades after CRISPR’s discovery, that the FDA approved the first CRISPR-based therapy, for sickle-cell anemia. Now, following up on […]