NeuroscienceMolecular TherapyPeer-reviewed
Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mousemodel of Multiple Sulfatase Deficiency
Vi Pham, Lucas Tricoli, Xinying Hong…·Rebecca C. Ahrens‐Nicklas
This study shows that SUMF1 gene therapy via hematopoietic stem cell transplant improves biochemical and functional outcomes in a mouse model of Multiple Sulfatase Deficiency.
3 citations