P01HL172740
Project Grant
Overview
Grant Description
Pathogenesis and therapeutic approaches in primary ciliary dyskinesia - overall abstract/project summary.
Mutations in more than 50 different genes cause primary ciliary dyskinesia (PCD) by disrupting the activity of motile cilia that facilitate mucociliary transport.
Knowledge of PCD has come from studies identifying disease-causing mutations, characterizing structural cilia abnormalities, finding genotype-phenotype relationships, and studying the cell biology of cilia.
Despite these important findings, we still lack effective treatments and people with PCD have significant pulmonary impairment.
As with nearly every other disease, a better understanding of pathogenic mechanisms can lead us to effective treatments.
To overcome some of these limitations, we developed a PCD pig with DNAI1 disrupted.
PCD pigs develop the hallmark sinonasal and pulmonary features of human PCD including neonatal respiratory distress in newborns and mucus obstruction, inflammation, infection, and bronchiectasis in older pigs.
It is already providing exciting discoveries that provide some of the foundation for the central theme of this PPG.
The overarching goal of this program is to understand better the pathophysiology of PCD airway disease to improve treatments and preventions that will change the lives of people who suffer from this debilitating disease.
The projects are closely interrelated.
We will address 3 main questions:
What is the early pathogenesis of PCD?
What happens to mitochondrial-dependent metabolism when ciliary beating is impaired?
Will DNAI1 gene addition to ciliated epithelial cells repair the outer dynein arm, restore cilia function, and impact disease development and progression?
We hope to have an important positive impact on accelerating discovery of new disease mechanisms and therapeutic interventions for PCD.
Mutations in more than 50 different genes cause primary ciliary dyskinesia (PCD) by disrupting the activity of motile cilia that facilitate mucociliary transport.
Knowledge of PCD has come from studies identifying disease-causing mutations, characterizing structural cilia abnormalities, finding genotype-phenotype relationships, and studying the cell biology of cilia.
Despite these important findings, we still lack effective treatments and people with PCD have significant pulmonary impairment.
As with nearly every other disease, a better understanding of pathogenic mechanisms can lead us to effective treatments.
To overcome some of these limitations, we developed a PCD pig with DNAI1 disrupted.
PCD pigs develop the hallmark sinonasal and pulmonary features of human PCD including neonatal respiratory distress in newborns and mucus obstruction, inflammation, infection, and bronchiectasis in older pigs.
It is already providing exciting discoveries that provide some of the foundation for the central theme of this PPG.
The overarching goal of this program is to understand better the pathophysiology of PCD airway disease to improve treatments and preventions that will change the lives of people who suffer from this debilitating disease.
The projects are closely interrelated.
We will address 3 main questions:
What is the early pathogenesis of PCD?
What happens to mitochondrial-dependent metabolism when ciliary beating is impaired?
Will DNAI1 gene addition to ciliated epithelial cells repair the outer dynein arm, restore cilia function, and impact disease development and progression?
We hope to have an important positive impact on accelerating discovery of new disease mechanisms and therapeutic interventions for PCD.
Awardee
Funding Goals
NOT APPLICABLE
Grant Program (CFDA)
Awarding / Funding Agency
Place of Performance
Iowa City,
Iowa
52242
United States
Geographic Scope
Single Zip Code
Related Opportunity
Analysis Notes
Amendment Since initial award the total obligations have increased 97% from $2,363,088 to $4,665,316.
The University Of Iowa was awarded
PCD Pathogenesis & Therapies: A PCD Pig Model Study
Project Grant P01HL172740
worth $4,665,316
from National Heart Lung and Blood Institute in June 2025 with work to be completed primarily in Iowa City Iowa United States.
The grant
has a duration of 4 years 9 months and
was awarded through assistance program 93.837 Cardiovascular Diseases Research.
The Project Grant was awarded through grant opportunity NHLBI Program Project Applications (P01 Clinical Trials Optional).
Status
(Ongoing)
Last Modified 6/5/26
Period of Performance
6/15/25
Start Date
3/31/30
End Date
Funding Split
$4.7M
Federal Obligation
$0.0
Non-Federal Obligation
$4.7M
Total Obligated
Activity Timeline
Subgrant Awards
Disclosed subgrants for P01HL172740
Transaction History
Modifications to P01HL172740
Additional Detail
Award ID FAIN
P01HL172740
SAI Number
P01HL172740-1800464366
Award ID URI
SAI UNAVAILABLE
Awardee Classifications
Public/State Controlled Institution Of Higher Education
Awarding Office
75NH00 NIH National Heart, Lung, and Blood Institute
Funding Office
75NH00 NIH National Heart, Lung, and Blood Institute
Awardee UEI
Z1H9VJS8NG16
Awardee CAGE
2D354
Performance District
IA-01
Senators
Charles Grassley
Joni Ernst
Joni Ernst
Modified: 6/5/26