Here, the authors use a gene therapy approach in an MLC mouse model in an attempt to improve the disease. They inject a virus that delivers a healthy copy of the MLC1 gene into a strongly affected brain region of MLC mice. This leads to a local recovery of the myelin vacuoles in the white matter. It should be noted that applying such a gene therapy approach in the human brain is not possible at the moment. But despite this, the study shows that some MLC features are reversible if appropriate therapeutic options would be available.
