The purpose of this study is to measure changes in ureagenesis after gene therapy in patients with OTC deficiency, an inborn error of urea synthesis. Current treatment is unsatisfactory. A Phase I study of adenoviral mediated gene therapy is underway. We are a collaborating insititution and wish to 1) perform heavy isotope/allopurinol studies to measure efficacy of the experimental treatment; and 2) to obtain blood for liver, renal, hematologic, and immune function to study safety.

Project Start
1998-12-01
Project End
1999-11-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
33
Fiscal Year
1999
Total Cost
Indirect Cost
Name
Oregon Health and Science University
Department
Type
DUNS #
009584210
City
Portland
State
OR
Country
United States
Zip Code
97239
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