ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

This study tests an experimental gene therapy using modified B cells (immune cells) to treat Mucopolysaccharidosis type I (MPS I), a rare genetic disease where the body cannot break down certain sugars, leading to organ damage. You may be eligible if you: - Have a diagnosis of MPS I Hurler-Scheie or Scheie syndrome - Are 10 years of age or older - Have adequate kidney function (creatinine clearance >60 ml/min) - Have adequate heart function (ejection fraction ≥40%) - Are willing to travel to the study site and stay nearby for at least 5 days after cell infusion You may NOT be eligible if you: - Have a known familial cancer syndrome - Have a history of B cell-related cancer or autoimmune disorders - Have had a prior stem cell transplant (HSCT) - Require systemic immune suppression or continuous supplemental oxygen Talk to your doctor to see if this trial is right for you.