Functional Correction of Mucopolysaccharidosis Type I (Hurler Syndrome) Using Genetically Modified Autologous Memory T Cells

This study aims to develop safer and more effective therapies for patients with a class of genetic diseases, called enzymopathies resulting from missing or defective enzymes. Current therapeutic approaches fail to stop disease progression and adequately replace deficient enzymes. Researchers are developing a novel cell-based therapeutic approach using genetically modified autologous memory T cells that is capable of delivering sustained enzyme replacement. In this project researchers will complete studies on efficacy, safety, dosing, and manufacturing that are critical for translating their novel cell-based therapy for MPS I. Successful completion of this project will advance this therapeutic approach into a clinical trial to improve outcomes for patients with MPS I.