# UNC Cystic Fibrosis Research and Translation Core Center

> **NIH NIH P30** · UNIV OF NORTH CAROLINA CHAPEL HILL · 2024 · $1,166,250

## Abstract

The UNC CFRTCC has set as its long-term goal the systemic “cure” of the cystic fibrosis (CF) phenotype,
including in the GI tract, pancreas, and lung. There is a broad need on the UNC-CH campus for a Core Research
Center to synergize and accelerate the pace of therapeutics development for CF. Our CF research base is broad
and encompasses groups with expertise in CFTR biogenesis/ion transport, genetics, gene therapy, GI and
airway epithelial cell biology, mucus/mucin biochemistry and biophysics, microbiology/immunology, and clinical
research. This group of investigators with CF therapeutic interests includes presently > 100 faculty. To accelerate
therapeutics development on the UNC-CH campus, there are needs for accurate and efficient in vitro and in vivo
testing of therapeutic agents, access to diverse and sufficient supplies of epithelial cells (GI, airway), access to
new mucus/mucin technologies relevant to CF pathogenesis, and a clinical research network that provides
patient specimens, clinical testing, and advisory services necessary for the therapeutics development process.
In response to these needs, we have proposed a CF RTCC with four service cores that are designed to provide
access to their reagents/technologies for UNC, national, and international investigators. The CF RTCC will
service a broad spectrum of therapies, including new chemical entities (NCEs), gene transfer vectors, and gene
modification approaches. The four service cores include: (1) the Molecular/Functional Measurement Core
(Martina Gentzsch, PI),which will offer state of the art in vitro measures of CFTR function in GI and airway
systems, complemented by novel mutant CFTR, mutant mucin, and βENaC mice, complemented by new CF
rabbit models, for in vivo studies of CF molecular pathogenesis/therapeutics and GI/airway epithelial disease;
(2) the Cell Models Core (Scott Randell, PI),which builds on its world-class experience in providing high-quality
primary, P1, P2, conditionally reprogrammed cells (CRC), and immortalized cells, and robust GI stem cell
techniques for provision of organoid and novel 2D cultures; (3) the Mucus Biochemistry and Biophysics Core
(Brian Button, PI), which will provide access to unparalleled new measures of the key biochemical and
biophysical mucus abnormalities that relate to CF pathogenesis and therapeutics and novel biomarker
development; and (4) The Clinical Translation Core (Scott Donaldson, PI), which will provide the requisite GI
biopsies for epithelial pathogenesis and genotype-specific therapeutic studies, generate sputum repositories,
bring a spectrum of novel imaging technologies (including F19 based MRI ventilation/washout kinetics measures)
into the CF field, and provide advice to the UNC CF and outside communities to with respect to therapeutics
development. These Cores will be supported by an Administrative Core (R. Boucher, PI), which will oversee
budgetary, communication, and program enhancement functions. The UNC CF RTCC is desi...

## Key facts

- **NIH application ID:** 10917152
- **Project number:** 5P30DK065988-20
- **Recipient organization:** UNIV OF NORTH CAROLINA CHAPEL HILL
- **Principal Investigator:** Richard Charles Boucher
- **Activity code:** P30 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $1,166,250
- **Award type:** 5
- **Project period:** 2003-09-30 → 2025-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10917152

## Citation

> US National Institutes of Health, RePORTER application 10917152, UNC Cystic Fibrosis Research and Translation Core Center (5P30DK065988-20). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10917152. Licensed CC0.

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