The study is exciting as it used the technique to boost dystrophin production, key to combatting the condition – but the study needs to be followed up with a lot more research, as outlined below.
Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, said:
It is exciting to see advances in how the technique can be applied to Duchenne muscular dystrophy, but as the authors recognise, there are limits to this study. The sample size was small and the study duration too short to know whether the gene editing was safe and effective. Although it seems to have largely boosted dystrophin production, which is key to tackling this condition, the team weren’t looking to record improvements in function.
The next step will be to conduct larger, longer-term studies to see if the gene editing approach does help to slow the progression of the condition and improve muscle strength. This won’t be a cure, but that shouldn’t obscure that this is a key step forward in proving the CRISPR/Cas9 technology could work for Duchenne.