The demonstration of gene transfer, mRNA and protein expression and biological efficacy is critical to the successful development of gene transfer vectors and pharmacologic therapies. An Expression Core has been in operation at Johns Hopkins University since the beginning of the grant. This Core has considerable expertise in assaying for normal and defective function of CFTR by methods such as: 1. Functional assays such as patch clamping; 2. Evaluating gene transfer, mRNA expression, vector shedding and spread in animal and patients samples 3. Assaying for protein expression Work from this Core during the previous budget period was instrumental in the continued development of adeno-associated virus as an extremely promising therapeutic agent. The recent development of gene transfer technologies as potential therapies have increased considerably the demand for the services of our Expression Core. Along these lines, the Core has developed considerable expertise in primate experiments in Rhesus monkeys and more recently in chimpanzees. The Expression Core functions as both a research and development Core, and in addition, provides support for the projects within this Gene Therapy Center.

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National Heart, Lung, and Blood Institute (NHLBI)
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Heart, Lung, and Blood Initial Review Group (HLBP)
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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

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