Assessment of three routes of delivery of the IDUA gene (gene implicated in MPS-I) in non-human primates comparing two serotypes, AAV9 and AAVrh.10 for the treatment of MPS-I.

PI: Dolan Sondhi, PhD

Institution: Cornell University

MPS Statistics

Institutions Awarded


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# of Awards


Total Amount


Award Period: 2013-2014

Award Total: $150,000


Award Period: 2014-2015

Award Total $75,000

Hurler disease (MPS I) is caused by a defect in the IDUA gene leading to a shortage of iduronidase protein. Without enough idurondiase, glycosaminoglyclans (GAG’s, heparan sulfate and dermatan sulfate) accumulate in cells throughout the body leading to neurodegeneration and organ failure among other symptoms. The only current viable therapy for Hurler disease is enzyme replacement therapy. This therapy is burdened by the need for constant administration of recombinant protein, and lack of impact on the central nervous system. This makes Hurler disease a prime candidate for gene therapy, where a correct copy of IDUA could be introduced into the patient leading to ongoing production of functional iduronidase protein and an abatement of symptoms.  In pre-clinical work funded by the Orphan disease center, our lab has demonstrated a restoration of iduronidase activity in the mouse model of Hurler disease after CNS administration of an adenoassociated virus serotype rh.10 expressing the IDUA gene.  This restoration of activity led to a marked reduction of GAGs, an improvement in behavioral phenotypes, and an improvement in survival.