Personalized Cystic Fibrosis Therapy and Research Center

NIH RePORTER · NIH · P30 · $1,158,088 · view on reporter.nih.gov ↗

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
10017681
Project number
5P30DK117467-03
Recipient
CINCINNATI CHILDRENS HOSP MED CTR
Principal Investigator
John Paul Clancy
Activity code
P30
Funding institute
NIH
Fiscal year
2020
Award amount
$1,158,088
Award type
5
Project period
2018-08-01 → 2023-06-30