The Vector Core's primary function is to supply recombinant Adeno-associated (rAAV) viral vector stocks that are used by all subprojects of this program. In addition to supplying research grade rAAV, the Core will develop a modified scalable baculovirus production system for rAAV1 that will be used for the anticipated clinical trial described in Project 1. Finally, the Core will supply GLP grade rAAV1 for preclinical toxicology studies and primate studies for Project 1.

Public Health Relevance

The Vector Core is essential to all of the projects in this Program Project grants because it will supply high quality research grade rAAV vectors. In addition, it will develop a high volume baculovirus production method for the clinical trial anticipated from Project 1 as well as produce the preclinical GLP material for toxicology testing for Project 1.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL051811-20
Application #
8479401
Study Section
Heart, Lung, and Blood Initial Review Group (HLBP)
Project Start
Project End
Budget Start
2013-05-01
Budget End
2014-04-30
Support Year
20
Fiscal Year
2013
Total Cost
$176,025
Indirect Cost
$46,929
Name
Johns Hopkins University
Department
Type
DUNS #
001910777
City
Baltimore
State
MD
Country
United States
Zip Code
21218
Duncan, Gregg A; Jung, James; Joseph, Andrea et al. (2016) Microstructural alterations of sputum in cystic fibrosis lung disease. JCI Insight 1:e88198
Schuster, Benjamin S; Ensign, Laura M; Allan, Daniel B et al. (2015) Particle tracking in drug and gene delivery research: State-of-the-art applications and methods. Adv Drug Deliv Rev 91:70-91
Mastorakos, Panagiotis; da Silva, Adriana L; Chisholm, Jane et al. (2015) Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy. Proc Natl Acad Sci U S A 112:8720-5
da Silva, Adriana L; Martini, Sabrina V; Abreu, Soraia C et al. (2014) DNA nanoparticle-mediated thymulin gene therapy prevents airway remodeling in experimental allergic asthma. J Control Release 180:125-33
Smith, Laura J; Ul-Hasan, Taihra; Carvaines, Sarah K et al. (2014) Gene transfer properties and structural modeling of human stem cell-derived AAV. Mol Ther 22:1625-34
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
Suk, Jung Soo; Kim, Anthony J; Trehan, Kanika et al. (2014) Lung gene therapy with highly compacted DNA nanoparticles that overcome the mucus barrier. J Control Release 178:8-17
Schuster, Benjamin S; Kim, Anthony J; Kays, Joshua C et al. (2014) Overcoming the cystic fibrosis sputum barrier to leading adeno-associated virus gene therapy vectors. Mol Ther 22:1484-93
Birket, Susan E; Chu, Kengyeh K; Liu, Linbo et al. (2014) A functional anatomic defect of the cystic fibrosis airway. Am J Respir Crit Care Med 190:421-32
Drouin, Lauren M; Agbandje-McKenna, Mavis (2013) Adeno-associated virus structural biology as a tool in vector development. Future Virol 8:1183-1199

Showing the most recent 10 out of 116 publications