Long-term In Vitro Correction of a-L-iduronidase Deficiency (Hurler Syndrome) in Human Bone Marrow

Posted on by immusoft

Allogeneic bone marrow transplantation is the most effective treatment for Hurler syndrome but, since this therapy is not available to all patients, we have considered an alternative approach based on transfer and expression of the normal gene in autologous bone marrow. A retroviral vector carrying the full-length cDNA for a-L-iduronidase has been constructed and used to transduce bone marrow from patients with this disorder…