Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders
E. Kleinboehl, K. Laoharawee, J. D. Jensen, J. J. Peterson, N. J. Slipek, B. J. Wick, M. J. Johnson, B. R. Webber, B. 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.
bioRxiv · Apr 23, 2024 · PreprintBioengineering