Abstract

This is a study to investigate the treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency (ADA-) with CD34+ selected autologous hematopoietic stem cells transduced with a human ADA gene in a newborn.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR001271-15
Application #
5223079
Study Section
Project Start
Project End
Budget Start
Budget End
Support Year
15
Fiscal Year
1996
Total Cost
Indirect Cost

  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