# Personalized Cystic Fibrosis Therapy and Research Center

> **NIH NIH P30** · CINCINNATI CHILDRENS HOSP MED CTR · 2021 · $1,144,093

## Abstract

SUMMARY/ABSTRACT
Cystic Fibrosis results from the absence or dysfunction of the Cystic Fibrosis Transmembrane Conductance
Regulator (CFTR) chloride channel. Defects in CFTR are strongly associated with genetic variations in the
CFTR gene that are unique to individual CF patients or to subgroups within the CF patient population. Thus, a
personalized medicine approach helps customize care to individual CF patients based on the unique molecular
and phenotypic characteristics associated with their CFTR gene. This will drive successful therapeutic
outcomes and minimize potential adverse effects of chronic therapies. Given that our CF Center at CCHMC
already has an excellent electronic medical record system (EPIC) and a site-specific IRB approved genotype
database available for clinical and research use, we intend to leverage these capabilities to develop
Personalized Cystic Fibrosis Therapy and Research Center (PCFC) for individual patients to (i) determine
his/her susceptibility to a particular form of CF mutation, and ii) take steps to mitigate the early onset of the
disease. Finally, the future of CF research offers the potential for a variety of CFTR-targeted therapies (i e.,
CFTR modulators) to treat the underlying cause of CF, with multiple modulators currently in different stages of
development.

## Key facts

- **NIH application ID:** 10249239
- **Project number:** 5P30DK117467-04
- **Recipient organization:** CINCINNATI CHILDRENS HOSP MED CTR
- **Principal Investigator:** John Paul Clancy
- **Activity code:** P30 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,144,093
- **Award type:** 5
- **Project period:** 2018-08-01 → 2023-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10249239

## Citation

> US National Institutes of Health, RePORTER application 10249239, Personalized Cystic Fibrosis Therapy and Research Center (5P30DK117467-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10249239. Licensed CC0.

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