Abstract

The purpose of the Pilot/Feasibility Core is to provide pilot funding for novel approaches to gene transfer related to the cardiopulmonary Program Project. The Core will fund two pilot projects at any one time and a total of four projects over the five year period of the proposal. A description of the administrative Procedures that will be used in the selection of the projects, their oversight, and the scienctific content of the two Pilot Projects chosen for funding in year one are described.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Specialized Center (P50)
Project #
5P50HL059412-04
Application #
6353089
Study Section
Project Start
2000-09-15
Project End
2001-08-31
Budget Start
1997-10-01
Budget End
1998-09-30
Support Year
4
Fiscal Year
2000
Total Cost
$269,466
Indirect Cost

  Institution

Name
University of Florida
Department
Type
DUNS #
073130411
City
Gainesville
State
FL
Country
United States
Zip Code
32611

  Publications

Smith, Barbara K; Martin, A Daniel; Lawson, Lee Ann et al. (2017) Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity. Exp Neurol 287:216-224
Salganik, Maxim; Aydemir, Fikret; Nam, Hyun-Joo et al. (2014) Adeno-associated virus capsid proteins may play a role in transcription and second-strand synthesis of recombinant genomes. J Virol 88:1071-9
Elder, Melissa E; Nayak, Sushrusha; Collins, Shelley W et al. (2013) B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease. J Pediatr 163:847-54.e1
ElMallah, Mai K; Falk, Darin J; Lane, Michael A et al. (2012) Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9. Hum Gene Ther Methods 23:148-56
Salganik, Maxim; Venkatakrishnan, Balasubramanian; Bennett, Antonette et al. (2012) Evidence for pH-dependent protease activity in the adeno-associated virus capsid. J Virol 86:11877-85
Qiu, Kai; Falk, Darin J; Reier, Paul J et al. (2012) Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice. Mol Ther 20:21-7
Wu, Ke; Li, Shoudong; Bodhinathan, Karthik et al. (2012) Enhanced expression of Pctk1, Tcf12 and Ccnd1 in hippocampus of rats: Impact on cognitive function, synaptic plasticity and pathology. Neurobiol Learn Mem 97:69-80
Petrs-Silva, Hilda; Dinculescu, Astra; Li, Qiuhong et al. (2011) Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol Ther 19:293-301
DeRuisseau, Lara R; Fuller, David D; Qiu, Kai et al. (2009) Neural deficits contribute to respiratory insufficiency in Pompe disease. Proc Natl Acad Sci U S A 106:9419-24
Clément, Nathalie; Knop, David R; Byrne, Barry J (2009) Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum Gene Ther 20:796-806

Showing the most recent 10 out of 19 publications