Gene Modified Stem Cells in MLD

In illnesses like MLD where there is a deficient enzyme we can use bone marrow transplantation to help the condition. In Teddy's case the Royal Manchester children's hospitals Rob Win and his colleagues have they think hopefully affected a cure. They've taken stem cells from her bone marrow and altered them in the lab to add back in a healthy copy of the gene as well as some control information to supercharge its activity. Those modified stem cells will return to Teddy where they migrate to the bone marrow and produce blood and immune cells. Some of these then migrate into the nervous system where they pump out the enzyme for other cells to use. Because more enzyme is good for the disease then we actually see that