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 John Hopkins University during the past budget period of the grant. This core has considerable expertise in assaying for normal and defective function of CFTR by general assays such as: 1. 36Cl-efflux and more specific assays such as patch clamping; 2. evaluating gene transfer, mRNA expression, vector shedding and spread in animal and patients samples 3. assays for protein expression Work from this core during the previous budget period was instrumental in the identification of adenoviral and adeno-associated virus systems as potential therapeutic agents and also phenylbutyrate as a potential therapeutic. The recent development of gene transfer technologies as potential therapies and new pharmacologic therapies such as phenylbutyrate have increased considerably the demand for the services our Expression Core. The Expression Core is both a research and development core and provides support for the projects within this Research Development Center.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL051811-07
Application #
6318399
Study Section
Project Start
2000-04-01
Project End
2001-03-31
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
7
Fiscal Year
2000
Total Cost
$270,973
Indirect Cost

  Institution

Name
Johns Hopkins University
Department
Type
DUNS #
045911138
City
Baltimore
State
MD
Country
United States
Zip Code
21218

  Publications

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
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
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
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
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
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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