autologous CD34+ cells genetically modified with IDUA lentiviral vector

**Autologous CD34+ cells genetically modified with IDUA lentiviral vector** is an investigational cell and gene therapy comprised of a patient's own CD34+ hematopoietic stem and progenitor cells that are collected, ex vivo genetically modified using a lentiviral vector encoding the human IDUA (alpha-L-iduronidase) gene, and then reinfused into the patient. The aim is to restore IDUA enzyme activity in patients with mucopolysaccharidosis type I (MPS I; Hurler syndrome), a lysosomal storage disorder caused by IDUA deficiency, thereby reducing accumulation of glycosaminoglycans (GAG) and ameliorating disease pathology. The mechanism is based on stable engraftment of the modified cells, which then supply functional IDUA enzyme to deficient tissues. Lentiviral vectors provide stable integration of the therapeutic gene, aiming for long-term expression. Early studies demonstrated durable IDUA expression and significant reduction of GAG in relevant animal models. It is under development as a potential alternative to enzyme replacement therapy and allogeneic transplantation[2][4].