Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mousemodel of Multiple Sulfatase Deficiency
Vi Pham, Lucas Tricoli, Xinying Hong, Parith Wongkittichote, Carlo Castruccio Castracani, Amaliris Guerra, Lars Schlotawa, Laura Ann Adang, Amanda Kuhs, Margaret M. Cassidy, Owen Kane, Emily Tsai, Maximiliano F Presa, Cathleen M. Lutz, Stefano Rivella, 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.
Molecular Therapy · Aug 22, 2024Peer-reviewedNeuroscience