Abstract

To monitor safety and measure changes in ureagenesis resulting from gene therapy in patients with ornithine transcarbamylase (OTC) deficiency. OTC deficiency is an inborn error of urea synthesis inherited as a X-linked disorder. Current treatment relies on alternate pathway therapy or liver transplantation. A Phase I study of adenoviral mediated gene therapy is being carried out at the University of Pennsylvania on adult heterozygote, with this disorder. We are a collaborating institution in this study and will perform in the GCRC 1) heavy isotope (15N)/allopurinol studies to measure efficiency of the experimental treatment in correcting deficits in ureasynthesis; and 2) to obtain blood for liver, renal, hemotologic and immune function to study safety in objects with OTC deficiency.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000079-36
Application #
6276476
Study Section
Project Start
1997-12-01
Project End
1999-11-30
Budget Start
1997-10-01
Budget End
1998-09-30
Support Year
36
Fiscal Year
1998
Total Cost
Indirect Cost

  Institution

Name
University of California San Francisco
Department
Type
DUNS #
073133571
City
San Francisco
State
CA
Country
United States
Zip Code
94143

  Publications

Kurtz, Theodore W; DiCarlo, Stephen E; Pravenec, Michal et al. (2018) Functional foods for augmenting nitric oxide activity and reducing the risk for salt-induced hypertension and cardiovascular disease in Japan. J Cardiol 72:42-49
Anderegg, Nanina; Johnson, Leigh F; Zaniewski, Elizabeth et al. (2017) All-cause mortality in HIV-positive adults starting combination antiretroviral therapy: correcting for loss to follow-up. AIDS 31 Suppl 1:S31-S40
Kurtz, Theodore W; DiCarlo, Stephen E; Morris Jr, R Curtis (2016) Logical Issues With the Pressure Natriuresis Theory of Chronic Hypertension. Am J Hypertens 29:1325-1331
Cruz, Giovanna I; Shao, Xiaorong; Quach, Hong et al. (2016) A Child's HLA-DRB1 genotype increases maternal risk of systemic lupus erythematosus. J Autoimmun 74:201-207
Morris Jr, R Curtis; Schmidlin, Olga; Sebastian, Anthony et al. (2016) Vasodysfunction That Involves Renal Vasodysfunction, Not Abnormally Increased Renal Retention of Sodium, Accounts for the Initiation of Salt-Induced Hypertension. Circulation 133:881-93
Kurtz, Theodore W; DiCarlo, Stephen E; Pravenec, Michal et al. (2016) An alternative hypothesis to the widely held view that renal excretion of sodium accounts for resistance to salt-induced hypertension. Kidney Int 90:965-973
Brambati, Simona Maria; Amici, Serena; Racine, Caroline A et al. (2015) Longitudinal gray matter contraction in three variants of primary progressive aphasia: A tenser-based morphometry study. Neuroimage Clin 8:345-55
Abraham, Alison G; Althoff, Keri N; Jing, Yuezhou et al. (2015) End-stage renal disease among HIV-infected adults in North America. Clin Infect Dis 60:941-9
Wang, Julie B; Pierce, John P; Ayala, Guadalupe X et al. (2015) Baseline Depressive Symptoms, Completion of Study Assessments, and Behavior Change in a Long-Term Dietary Intervention Among Breast Cancer Survivors. Ann Behav Med 49:819-27
Brehm, John M; Ramratnam, Sima K; Tse, Sze Man et al. (2015) Stress and Bronchodilator Response in Children with Asthma. Am J Respir Crit Care Med 192:47-56

Showing the most recent 10 out of 634 publications