Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis." In this program, Lentigen will collaborate with Dr. R. Scott McIvor Professor, Department of Genetics, Cell Biology and Development, and Dr. Walter Low, Professor, Departments of Neurosurgery and Physiology at the University of Minnesota, Minneapolis, MN.
Hunter syndrome (Mucopolysaccharidosis type II, MPS II) is a rare X-linked recessive, inherited lysosomal storage disorder with an onset at 2-4 years of age. It has been estimated to affect approximately 1 in 155,000 live male births. It is caused by the absence of iduronate-2-sulfatase, resulting in systemic accumulation of glycosaminoglycans. Affected individuals suffer from skeletal abnormalities, organomegaly, life-threatening obstructive airway disease, and, in the severely enzyme deficient form, neurologic degeneration and death by age 15. The University of Minnesota is a world-leading center for the treatment of lysosomal storage diseases by hematopoietic stem cell transplantation.