Gene and Pharmacological Therapies for Cystic Fibrosis

描述（由申请人提供）：

DESCRIPTION (provided by applicant): It is the overall goal of this Center to utilize the strengths of our current CF Research Group in basic and clinical research and patient care to develop gene and pharmacologic therapies for patients with CF. The specific aims are to develop adeno-associated viral vectors as gene therapy agents and to explore new pharmacologic therapies based upon altering expression and trafficking of mutant CFTR. Project by Guggino will develop a new AAV gene therapy vector based on the AAV5 serotype as a new therapy for CF. The aims will be to: assess the efficiency and distribution of vector DNA transfer and to determine the risk of immunologic reactions, alterations in pulmonary function, or spread in distant organs in monkeys. This will be followed by a clinical trial in adult CF patients. Project by Flotte: Adeno-associated virus vectors for CF gene therapy. The primary hypotheses to be tested is that: Aberrant down-regulation of certain anti-protease, anti-inflammatory, and glycosylation-related genes contribute to CF lung disease, and augmentation of these substances will ameliorate the CF lung disease phenotype. Project by Muzyczka will focus on the Biology of AAV. Finally, there are three cores, an Expression, a Vector Core, and an Administration Core.