GeneticsHuman Molecular GeneticsPeer-reviewed
MCOLN1 gene-replacement therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.
Samantha DeRosa, Monica Salani, Sierra Smith…·Yulia Grishchuk
This study demonstrates that AAV-mediated MCOLN1 gene transfer improves motor function and reduces brain pathology in a mouse model of mucolipidosis IV.
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