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Gene editing muscle stem cells – a promising approach to treat muscle disease

07 April 2022

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Gene editing is a powerful technique for altering highly specific sequences, even individual letters, of the DNA code. Researchers hope to harness this power to correct specific disease-causing mutations, such as those causing muscular atrophy – a progressive and ultimately fatal deterioration of muscle. Promising results have been achieved in animal models of the disease, but several steps remain before the technique is fit for humans. For example, the editing machinery is traditionally delivered to cells via plasmid vectors – small pieces of DNA – but these can potentially integrate into the genome and cause further mutations. A safer option, because it degrades quickly and doesn’t integrate, is messenger RNA (mRNA) – like the sort used to deliver certain COVID-19 vaccines. Using mRNA, scientists have now successfully delivered gene editing machinery to human muscle cells (pictured), fixed a muscle atrophy mutation therein, and consequently brought the therapy one step closer to the clinic.

Written by Ruth Williams

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