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Breakthrough for genetic diseases: New tool can fix ‘nonsense’ mutations, including haemoglobin defects
A new gene-editing technique known as PERT has shown the ability to correct a wide range of disorders caused by “nonsense” mutations, including forms that interfere with haemoglobin production. The ...
Premature termination codons (PTCs) account for 10 to 20% of genetic diseases in humans. The gene inactivation resulting from PTCs can be counteracted by the use of drugs stimulating PTC readthrough, ...
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