Abstract

The Clinical Core is an essential element of the Gene Therapy Center and will be the focus of all clinical activities involving patients. Patients currently enrolled in the Johns Hopkins Hospital Cystic Fibrosis Center will be invited to participate in the cystic fibrosis gene therapy protocol. Patients taking part in the gene therapy protocol will be hospitalized in the General Clinical Research Center (GCRC) and all outpatient follow-up clinical evaluations will be carried out in the GCRC. All laboratory, radiographic and imaging studies will be coordinated through the Clinical Core. Specimens for laboratory evaluations including respiratory tract secretions, blood, and bronchoalveolar lavage fluid will be obtained and processed in the GCRC. Pulmonary function testing will be carried out in the GCRC by Center staff and all nasal brushings, transepithelial potential difference measurements and bronchoscopies will be performed in the GCRC by Clinical Core staff. The services of the GCRC staff, which include a senior research nurse and pediatric nutritionist, will be available to patients enrolled in the gene therapy protocol. In addition, harvesting of bone marrow for preparation of cell lines will take place through the Clinical Core in the GCRC.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL051811-04
Application #
5214128
Study Section
Project Start
Project End
Budget Start
Budget End
Support Year
4
Fiscal Year
1996
Total Cost
Indirect Cost

  Publications

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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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