Abstract

This study will test the hypothesis that reconstitution of immune function in adenosine deaminase (ADA)-deficient severe combined immunodeficiency syndrome (SCID) will result from the autologous transplantation of CD34+ umbilical cord blood stem cells that have been transduced with a normal human ADA complementary DNA by retroviral-mediated gene transfer.
The specific aims of this protocol are to: 1)determine whether transduced CD34+ umbilical cord blood stem cells will engraft following autologous transplantation in an infant with ADA deficiency; 2) determine the extent and durability of engraftment in different hematopoietic cell populations; 3) determine the extent and durability of immune reconstitution following gene therapy.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR001271-19
Application #
6308615
Study Section
Project Start
1999-12-01
Project End
2000-11-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
19
Fiscal Year
2000
Total Cost
$25,799
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

Peyvandi, Shabnam; Kim, Hosung; Lau, Joanne et al. (2018) The association between cardiac physiology, acquired brain injury, and postnatal brain growth in critical congenital heart disease. J Thorac Cardiovasc Surg 155:291-300.e3
Peyvandi, Shabnam; Chau, Vann; Guo, Ting et al. (2018) Neonatal Brain Injury and Timing of Neurodevelopmental Assessment in Patients With Congenital Heart Disease. J Am Coll Cardiol 71:1986-1996
Jacobson, Denise L; Lindsey, Jane C; Coull, Brent A et al. (2018) The Association of Fat and Lean Tissue With Whole Body and Spine Bone Mineral Density Is Modified by HIV Status and Sex in Children and Youth. Pediatr Infect Dis J 37:71-77
Wahlstrom, Justin; Patel, Kiran; Eckhert, Erik et al. (2017) Transplacental maternal engraftment and posttransplantation graft-versus-host disease in children with severe combined immunodeficiency. J Allergy Clin Immunol 139:628-633.e10
Ward, Shan L; Turpin, Autumn; Spicer, Aaron C et al. (2017) Long-Term Pulmonary Function and Quality of Life in Children After Acute Respiratory Distress Syndrome: A Feasibility Investigation. Pediatr Crit Care Med 18:e48-e55
Ruan, Alexandra; Tobin, Nicole H; Mulligan, Kathleen et al. (2016) Brief Report: Macrophage Activation in HIV-Infected Adolescent Males Contributes to Differential Bone Loss by Sex: Adolescent Trials Network Study 021. J Acquir Immune Defic Syndr 72:372-5
Desikan, Rahul S; Barkovich, A James (2016) Malformations of cortical development. Ann Neurol 80:797-810
Peyvandi, Shabnam; De Santiago, Veronica; Chakkarapani, Elavazhagan et al. (2016) Association of Prenatal Diagnosis of Critical Congenital Heart Disease With Postnatal Brain Development and the Risk of Brain Injury. JAMA Pediatr 170:e154450
Harmatz, Paul R; Mengel, Karl Eugen; Giugliani, Roberto et al. (2015) Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome. Mol Genet Metab 114:186-94
Aweeka, F T; Hu, C; Huang, L et al. (2015) Alteration in cytochrome P450 3A4 activity as measured by a urine cortisol assay in HIV-1-infected pregnant women and relationship to antiretroviral pharmacokinetics. HIV Med 16:176-83

Showing the most recent 10 out of 502 publications