Researchers at Duke University, USA, have shown that a genome editing technique can restore dystrophin production in a mouse model of Duchenne muscular dystrophy. The effect lasted over a year in the mice, suggesting that genome editing could be a one-off, permanent treatment. However there were some unexpected results related to the precision of the genome editing and the immune response to the adeno-associated virus (AAV) used. These will need further investigation before human trials could be considered.
For further information, read Duke University’s press release.