Paterson Institute for Cancer Research other research news

Researchers reported in the Proceedings of the National Academy of Sciences the successful transfer of the enzyme alpha-L-iduronidase (IDUA) into bone marrow cells of a patient suffering from Hurler syndrome, a fatal neurological and musculoskeletal disease.

Using a retroviral vector, full length cDNA for IDUA was transferred into CD34+ cells, resulting in transfection of 25-56 percent of cells exposed to the virus. Functional enzymes were produced at levels 10-35 percent of those found in healthy individuals. ...