Abstract

This study will evaluate the safety and efficacy of gene therapy in cystic fibrosis (CF) utilizing a replication deficient recombinant adenovirus (Av1Cf2). Av1Cf2 will be used to deliver the human cystic fibrosis transmembrane conductance regulator (CFTR) cDNA to the upper respiratory tract of cystic fibrosis patients. Fifteen subjects will be studied in groups of three. Each group will receive an escalating dose of Av1Cf2 starting at 1x10(6) pfu and increasing by half-log increments after acute toxicity studies verify the safety of the regimen. Safety, gene expression, and antibody response will be evaluated.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR008084-03
Application #
5225642
Study Section
Project Start
Project End
Budget Start
Budget End
Support Year
3
Fiscal Year
1996
Total Cost
Indirect Cost

  Publications

Natarajan, Girija; Shankaran, Seetha; Laptook, Abbot R et al. (2018) Association between sedation-analgesia and neurodevelopment outcomes in neonatal hypoxic-ischemic encephalopathy. J Perinatol 38:1060-1067
Askie, Lisa M; Darlow, Brian A; Finer, Neil et al. (2018) Association Between Oxygen Saturation Targeting and Death or Disability in Extremely Preterm Infants in the Neonatal Oxygenation Prospective Meta-analysis Collaboration. JAMA 319:2190-2201
DiFrancesco, Mark W; Shamsuzzaman, Abu; McConnell, Keith B et al. (2018) Age-related changes in baroreflex sensitivity and cardiac autonomic tone in children mirrored by regional brain gray matter volume trajectories. Pediatr Res 83:498-505
Autmizguine, Julie; Tan, Sylvia; Cohen-Wolkowiez, Michael et al. (2018) Antifungal Susceptibility and Clinical Outcome in Neonatal Candidiasis. Pediatr Infect Dis J 37:923-929
Jilling, Tamas; Ambalavanan, Namasivayam; Cotten, C Michael et al. (2018) Surgical necrotizing enterocolitis in extremely premature neonates is associated with genetic variations in an intergenic region of chromosome 8. Pediatr Res 83:943-953
Hogan, Jonathan J; Palmer, Matthew D; Loren, Alison W et al. (2017) Quiz Page May 2017: CKD and Nephrotic Syndrome After Allogeneic Hematopoietic Cell Transplantation. Am J Kidney Dis 69:A10-A13
Di Fiore, Juliann M; Martin, Richard J; Li, Hong et al. (2017) Patterns of Oxygenation, Mortality, and Growth Status in the Surfactant Positive Pressure and Oxygen Trial Cohort. J Pediatr 186:49-56.e1
Hahn, Andrew D; Higano, Nara S; Walkup, Laura L et al. (2017) Pulmonary MRI of neonates in the intensive care unit using 3D ultrashort echo time and a small footprint MRI system. J Magn Reson Imaging 45:463-471
Kingery, Kathleen M; Narad, Megan E; Taylor, H Gerry et al. (2017) Do Children Who Sustain Traumatic Brain Injury in Early Childhood Need and Receive Academic Services 7 Years After Injury? J Dev Behav Pediatr 38:728-735
Glauser, Tracy A; Holland, Katherine; O'Brien, Valerie P et al. (2017) Pharmacogenetics of antiepileptic drug efficacy in childhood absence epilepsy. Ann Neurol 81:444-453

Showing the most recent 10 out of 502 publications