Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders
Evan W. Kleinboehl, Kanut Laoharawee, Jacob D. Jensen, Joseph J. Peterson, Nicholas J. Slipek, Bryce J. Wick, Matthew J. Johnson, Beau R. Webber, Branden S. Moriarity
This study demonstrates that genetically engineered memory T cells can deliver enzyme replacement therapy for mucopolysaccharidosis type I, reducing GAG levels in multiple tissues for up to 22 weeks.
Molecular Therapy · Oct 4, 2024Peer-reviewedBioengineering