Precision Medicine Center for Cystic Fibrosis

University Of Iowa, Iowa City IA

Investigators

Linked publications & trials

Abstract

PROJECT SUMMARY/ABSTRACT (PRECISION MEDICINE CENTER FOR CF – OVERALL) For the past 25 years, the P30 Center for Gene Therapy of CF has concentrated on advancing CF treatments through enhanced understanding of disease mechanisms, identifying key targets for gene and cell manipulation, and developing technologies for CFTR gene delivery and editing in vivo. As of 2024, the Center will transition leadership and become the Precision Medicine Center for Cystic Fibrosis, reflecting updated patient needs and investigator expertise. The landscape of CF has evolved significantly with the introduction of highly effective modulator therapy (HEMT), which has resulted in new challenges such as increased prevalence of multisystem effects in aging CF patients. Complications like CF-related diabetes (CFRD), pancreatitis, kidney dysfunction, liver issues, obesity, and shifts in dynamics of the gut microbiome are now critically influencing quality of life. As individuals with CF age, the variability and organ-specific nature of CF-associated diseases are influenced by the involvement of diverse CFTR gene mutations across patients, interactions with modifier genes, and environmental factors. The Center uses a bedside-to-bench-to-bedside approach in CF management and research, integrating clinical observations, experimental research, and clinical applications. This iterative process ensures that insights from work with patients drive the development of new therapies, bridging clinical practice with scientific discoveries. The highly collaborative environment at the University of Iowa fosters rapid CF research aligned with the NIDDK mission, focusing on innovative therapies. Key research areas that build on the center’s strengths include CF pancreatic disease, CFRD, CF gallbladder disease, and CF intestinal disease. The Center has effectively supported CF research through several mechanisms: 1) Its Pilot and Feasibility Program, which has sponsored 74 pilots over the previous 25 years of funding, has brought numerous new Members and expertise into the Center, and has facilitated the maturation of talented junior Associate Members into independent tenure-track faculty. 2) The establishment or expansion of several core facilities (Viral and Non-Viral Vector Core; Cell, Tissues, and Models Core; Comparative Pathology and Animal Models Core; and Clinical Phenotyping Core) dedicated to CF research has provided Center investigators with specialized vectors, CF model systems (human, pig, ferret, and mouse), and approaches suitable for testing important hypotheses about CF pathogenesis and developing effective therapies. These resources have also enabled the Center to serve as a resource for the distribution of viral vectors, cells, tissues, and CF pig resources to numerous outside institutions. 3) The Center’s Enrichment Programs have promoted interdisciplinary interactions and training. 4) Formal internal and external mechanisms for review of the Center, the Cores, and the Pilot and Feasibility projects has ensured a high level of excellence and the most appropriate utilization of the Center’s resources. In summary, the Center has significantly enhanced CF research at UI and serves as a valuable resource for institutions worldwide engaged in CF research.