Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Diagnosis of partial ornithine transcarbamylase deficiency Female heterozygote with abnormal allopurinol challenge or underlying defect in either N15 urea or N15 glutamine OR Male with childhood/adulthood onset OR Family history of 2 affected children * Stable for at least 1 month prior to study * Plasma ammonium levels less than 50 micromoles --Prior/Concurrent Therapy-- * Concurrent alternate pathway therapy to control hyperammonemia allowed --Patient Characteristics-- * Hepatic: No history of liver disease * Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No high level of neutralizing antibodies to the adenovirus