Gene therapy for myotonic dystrophy – “We take steps every time.”

“In order to cut the error from the DNA, the entire sequence is cut out. This means that the original piece of genetic information is also gone. It is not known whether this removal will have any adverse effects. That is why we want to put the right piece in between after cutting.

What we hoped in advance seems to have been achieved. In the model of the disease, we were able to cut the hereditary defect and replace it with a healthy piece. In addition, the first results show that the transplanted muscle stem cells of the model for the disease can survive for a longer period of time. We are now looking at whether the cells also contribute to muscle recovery.