Abstract

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.

Public Health Relevance

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL051811-18
Application #
8292959
Study Section
Heart, Lung, and Blood Initial Review Group (HLBP)
Project Start
Project End
Budget Start
2011-05-01
Budget End
2012-04-30
Support Year
18
Fiscal Year
2011
Total Cost
$189,885
Indirect Cost

  Institution

Name
Johns Hopkins University
Department
Type
DUNS #
001910777
City
Baltimore
State
MD
Country
United States
Zip Code
21218

  Publications

Schneider, Craig S; Xu, Qingguo; Boylan, Nicholas J et al. (2017) Nanoparticles that do not adhere to mucus provide uniform and long-lasting drug delivery to airways following inhalation. Sci Adv 3:e1601556
Kates, Max; Date, Abhijit; Yoshida, Takahiro et al. (2017) Preclinical Evaluation of Intravesical Cisplatin Nanoparticles for Non-Muscle-Invasive Bladder Cancer. Clin Cancer Res 23:6592-6601
Guggino, William B; Benson, Janet; Seagrave, JeanClare et al. (2017) A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System. Hum Gene Ther Clin Dev 28:145-156
Schuster, Benjamin S; Allan, Daniel B; Kays, Joshua C et al. (2017) Photoactivatable fluorescent probes reveal heterogeneous nanoparticle permeation through biological gels at multiple scales. J Control Release 260:124-133
Duncan, Gregg A; Jung, James; Joseph, Andrea et al. (2016) Microstructural alterations of sputum in cystic fibrosis lung disease. JCI Insight 1:e88198
Yu, Tao; Chisholm, Jane; Choi, Woo Jin et al. (2016) Mucus-Penetrating Nanosuspensions for Enhanced Delivery of Poorly Soluble Drugs to Mucosal Surfaces. Adv Healthc Mater 5:2745-2750
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
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
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

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