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irupar t1_j8mwlwz wrote

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.

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