Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

You may be eligible if

• PROTOCOL ENTRY CRITERIA:
• -Disease Characteristics--
• Mucopolysaccharidosis II (mild Hunter syndrome) as defined by the following:
• Characteristic coarse facial features, hepatosplenomegaly, and radiographic evidence of dysostosis multiplex
• Elevated urinary excretion of glycosaminoglycans in 3 urine specimens
• Deficient iduronate-2-sulfatase enzyme activity as measured in plasma and leukocytes
• Mutation consistent with mild Hunter syndrome must have either: A single base substitution of the coding sequence not previously associated with severe Hunter syndrome phenotype OR An exon-skipping mutation that would allow for occasional production of (minimal amounts of) normal protein
• -Patient Characteristics--
• Cardiovascular: No severe cardiac disease
• Pulmonary: No severe respiratory disease
• Other:
• Must have IQ score of 80 or higher
• Effective contraception required of all fertile patients