This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Cystic fibrosis (CF) is an autosomal recessive disorder with an incidence of approximately 1 in 3300 live births . It is due to defects in the CFTR gene, which is located on chromosome 7 . The CFTR can function as a cAMP-activated chloride channel and can also regulate the activity of other chloride channels . Through a mechanism that is not fully understood, CFTR defects strongly predispose affected individuals to chronic endobronchial infections with organisms such as Pseudomonas aeruginosa and Staphylococcus aureus . The CFTR defect may also exacerbate the inflammatory response to microbiological colonization of the lung. Adeno-associated virus (AAV) vectors are among a small number of recombinant virus vector systems which have been shown to have utility as in vivo gene transfer agents and thus are potentially of great importance for human gene therapy. Subjects will be involved in study procedures for a total of up to 30 weeks, which includes 14 weeks of active participation, followed by two structured telephone interviews during the final 16 weeks of participation. Screening of subjects will be up to two weeks prior to the first dose on Day 0. Subjects will receive two doses of study drug or placebo after randomization at 30-day intervals (Day 0, 30) and will be followed up for efficacy, tolerability and safety evaluations on the following study visit days: Day 14, 30, 45, 60, 75, and 90. During this time, the subject will make a total of eight visits to the PCRU. Adverse events occurring after Day 90 will be collected using a structured telephone interview on Days 150 and 210. Subjects with ongoing adverse events will be followed until the event has resolved or the investigator determines the event has stabilized.
| Altekruse, Sean F; Shiels, Meredith S; Modur, Sharada P et al. (2018) Cancer burden attributable to cigarette smoking among HIV-infected people in North America. AIDS 32:513-521 |
| Salemi, Parissa; Skalamera Olson, Julie M; Dickson, Lauren E et al. (2018) Ossifications in Albright Hereditary Osteodystrophy: Role of Genotype, Inheritance, Sex, Age, Hormonal Status, and BMI. J Clin Endocrinol Metab 103:158-168 |
| Robert Braši?, James; Mari, Zoltan; Lerner, Alicja et al. (2018) Remission of Gilles de la Tourette Syndrome after Heat-Induced Dehydration. Int J Phys Med Rehabil 6: |
| Altman, Matthew C; Whalen, Elizabeth; Togias, Alkis et al. (2018) Allergen-induced activation of natural killer cells represents an early-life immune response in the development of allergic asthma. J Allergy Clin Immunol 142:1856-1866 |
| AIDS-defining Cancer Project Working Group of IeDEA, COHERE in EuroCoord (2018) Non-Hodgkin lymphoma risk in adults living with HIV across five continents. AIDS 32:2777-2786 |
| Juraschek, Stephen P; Miller 3rd, Edgar R; Appel, Lawrence J (2018) Orthostatic Hypotension and Symptoms in the AASK Trial. Am J Hypertens 31:665-671 |
| Elion, Richard A; Althoff, Keri N; Zhang, Jinbing et al. (2018) Recent Abacavir Use Increases Risk of Type 1 and Type 2 Myocardial Infarctions Among Adults With HIV. J Acquir Immune Defic Syndr 78:62-72 |
| Al-Sofiani, Mohammed E; Yanek, Lisa R; Faraday, Nauder et al. (2018) Diabetes and Platelet Response to Low-Dose Aspirin. J Clin Endocrinol Metab 103:4599-4608 |
| Grover, Surbhi; Desir, Fidel; Jing, Yuezhou et al. (2018) Reduced Cancer Survival Among Adults With HIV and AIDS-Defining Illnesses Despite No Difference in Cancer Stage at Diagnosis. J Acquir Immune Defic Syndr 79:421-429 |
| Grams, Morgan E; Sang, Yingying; Ballew, Shoshana H et al. (2018) Predicting timing of clinical outcomes in patients with chronic kidney disease and severely decreased glomerular filtration rate. Kidney Int 93:1442-1451 |
Showing the most recent 10 out of 1014 publications
