Since 2013, several international teams have been studying how to use CRISPR against Duchenne dystrophy. For now, the therapy has been shown to work on guinea pigs with the disease. When is experimentation on humans? The researchers talked about it during the XVII International Conference on Duchenne and Becker muscular dystrophy.
The gene responsible for dystrophy is very cumbersome, which makes gene therapy more complicated. Transporting a healthy copy to sick cells using the usual viruses is impossible. As a result, scientists are developing different alternatives to classical gene therapy. However, all this work requires a large number of experiments, above all to understand what the impact on human beings could be.
There are currently three alternative gene therapies being developed.
- Modify the expression of the gene responsible for the disease.
- Act only on deletion mutations, those in which a stroke is missing.
- In case of duplication mutations, remove too many portions.
For the time being, only 13 human patients have tested gene therapies in progress. The results seem positive for the moment, but the analyzes and tests are still in progress. Furthermore, only one treatment has been approved so far and could only help a slice of the sick. It works only on patients with a particular genetic mutation, not on the others.
The Italian Giulio Cossu offered an alternative to CRISPR during the conference. With his Manchester University team he is testing stem cell transplantation. The treatment is still under study.