Breakthrough for genetic diseases: New tool can fix nonsense mutations, including haemoglobin defects

A new gene-editing technique called PERT offers a potential single-tool solution for numerous genetic disorders caused by nonsense mutations. This method, described in Nature, instructs cells to read through premature stop signals, enabling the production of functional proteins. PERT could revolutionize treatment for conditions like beta-thalassaemia, offering a more versatile and potentially long-lasting approach.