BioengineeringMolecular TherapyPeer-reviewed
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…·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.
3 citations