University Launches First Gene Therapy Clinical Trial Minnesota's first gene therapy trial began on Friday, November 15 when physician and Institute of Human Genetics researcher Chester Whitley, Ph.D., M.D. infused genetically-corrected lymphocytes into a patient with Hunter syndrome. Individuals missing IDS are unable to breakdown mucopolysaccharides in a normal fashion so they are stored in various tissues. As a result, tissues become thickened giving rise to heart and respiratory failure.
Joints become stiff and arthritic often leaving patients wheel-chair bound.
The most severely affected children suffer progressive mental retardation and die before age 15. Rare individuals have a more mild course, typically escaping brain damage, and surviving into adulthood. One such more mildly-affected adult is the first patient to undergo this innovative gene transfer treatment. David Stroncek and Jeff McCullough from the patient's blood by apheresis, a routine outpatient procedure in which lymphocytes are isolated from the blood. This therapeutic vector was constructed by Dr. Whitley's group from genes assembled from a virus, a bacterium and also contain the normal human IDS gene.