New DMD genome editing study

Published Date
Beatriz Bustillo Ramirez
Orange box stating: Breaking news research

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.

If you have any questions about this news story or any other DMD research, please contact the MDUK Research Line on 020 7803 4813 or email

Keep in touch