A long-running human trial has shown that CRISPR gene editing could prove to be a highly effective way of treating serious conditions.
The trial, which was kicked off in 2019 by an international team of scientists, found that a new gene-editing therapy called exagamglogene autotemcel, or ex-cel for short, was able to essentially “cure” patients with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD), two blood disorders that are conventionally treated using blood transfusions.
It’s a promising new use of the technology. Around 100,000 Americans are affected by TDT, while SCD affects an estimated 300 to 3,000. And in a broader sense, the results suggest that tinkering with genetic code could come to be a practical, widespread new area of medicine.
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