Rare Disease Report

Developing a Gene Therapy for Hurler Syndrome

FEBRUARY 28, 2017
Li Ou, Ph.D.

Li Ou, Ph.D., of the University of Minnesota talks about the studies he is involved with to develop a gene therapy for MPS I (Hurler Syndrome).
Hurler syndrome is a rare, hereditary, lysosomal disease that arises from a deficiency or absence of the enzyme iduronidase (IDUA), which is needed to break down complex sugars. Hurler syndrome affects the brain and spinal cord in children, resulting in debilitating signs and symptoms that include developmental delay, progressive mental decline, loss of physical function, impaired language development (due to hearing loss and an enlarged tongue), corneal and retinal damage, carpal tunnel syndrome, and restricted joint movement. Hurler syndrome is the most severe form of MPS I. Less severe forms include Hurler-Scheie and Scheie syndromes.

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