The new gene (I don't think they are editing a gene, just inserting one) expresses the enzyme in the cells that were edited. These cells can lower the serum levels of sulphatides thereby reducing the levels else where in the body reducing the damage done by them and hopefully effectively curing the disease. It remains to be seen if it prevents lifelong impact from MLD.
irupar t1_j8mwlwz wrote
Reply to Recently a gene therapy for a rare inherited neurodegenerative disorder was approved. How come cure is achieved through bone marrow transplantation? by nickoskal024
The new gene (I don't think they are editing a gene, just inserting one) expresses the enzyme in the cells that were edited. These cells can lower the serum levels of sulphatides thereby reducing the levels else where in the body reducing the damage done by them and hopefully effectively curing the disease. It remains to be seen if it prevents lifelong impact from MLD.