Basic and Clinical Studies of Cystic Fibrosis

自1997年以来，匹兹堡大学就有一个囊性纤维化研究中心，尽管它的结构和支持机制已经并将继续发生变化。目前，该中心收集了超过960万美元的外部赠款和合同，以支持cf相关的研究。它由7个部门的39名研究人员组成，他们的研究集中在三个主要领域。CF的细胞和分子生物学领域，针对模型系统和CFTR的研究

A Cystic Fibrosis Research Center has existed at the University of Pittsburgh since 1997, although its structure and support mechanisms have, and continue to, change. The current center gathers over $9.6M in external grants and contracts in support of CF-related research. It consists of 39 investigators in 7 departments, whose research is focused in three major areas. The area of Cell and Molecular Biology of CF, directed at studies of CFTR in model systems and human airway cells, is led by Drs. Raymond Frizzell and Joseph Pilewski, and is supported largely through NIH R01 and Cystic Fibrosis Foundation (CFF) grants, as well as pharmaceutical industry contracts. This group studies mechanisms of CFTR biogenesis, trafficking and regulation, the role of CFTR in airway cell and pancreatic physiology, airway stem cells, and the epithelial sodium channel (ENaC), its regulation and its relation to the activity of CFTR. Therapeutic approaches evolving from several of these basic studies are being pursued as well. A second research area, Lung Infection and Inflammation, headed by Dr. Jay Kolls, focuses on-the pulmonary inflammatory response to bacterial infection in human airway cell and animal models, defining the underlying mechanisms of these responses and how they can be modified therapeutically. This work is also supported primarily by NIH and CFF grants, and it represents a new and rapidly growing area within the Center. The third and also expanding area of focus is Clinical Research in CF, headed by Drs. Joseph Pilewski and David Orenstein. This group is pursuing several clinical studies that have emerged from the basic science initiatives of the Center, as well as projects within the Therapeutic Development Network (TON) of the CFF; it is supported primarily by CFF grants at present. The proposed CF Research and Translation Core Center will be directed by Dr. Raymond Frizzell, who also directs the CFF-sponsored Research Development Program, a current NIH SCOR entitled 'CFTR in Airway Cell Function', is co-investigator on a T32-supported training program in epithelial cell biology, and participates in two other T32 training programs. Drs. Jay Kolls and Joseph Pilewski will serve as Associate Directors of the Center. The Center will be comprised of three cores other than the Administrative: Human Airway Cell Physiology (Raymond Frizzell and Joseph Pilewski, co-directors), Clinical Studies/Outcomes (Jay Kolls and Joseph Pilewski, co-directors), and Imaging (Simon Watkins, director). In addition, the Core Center will operate a Pilot and Feasibility Program to encourage new ideas and investigators in CF research. Of past P/F projects within the NTH SCOR application, 100% have received NIH R01 grant support and all continue to be involved in CF research. This Center emphasizes the translation of basic knowledge into applied therapeutics. The projected funding period should witness the clinical testing of several novel strategies originating at the Center in CF patients.