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Leveraging a lung organoid model to study early cystic fibrosis lung disease

Award Number: R03TR005349
ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 05/01/2026
PERIOD OF PERFORMANCE END DATE: 04/30/2028

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Leveraging a lung organoid model to study early cystic fibrosis lung disease - PROJECT SUMMARY/ABSTRACT Cystic fibrosis (CF) is a multisystemic, autosomal recessive disorder with the majority of morbidity and mortality extending from lung disease. Given the benefits that older children and adults with CF have derived from cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, it is anticipated that early childhood – or even in utero – treatment with CFTR modulator therapies may significantly delay or even prevent the development of CF lung disease. The role of CFTR protein in fetal lung development, and thus the potential impact of early CFTR modulator therapies, has yet to be fully elucidated. The proposed research project aims to better understand the pathogenesis of CF lung disease and the impacts of deficient CFTR protein on fetal lung development while overcoming a general roadblock in the study of many pediatric lung diseases, the scarcity of available human material. Previous immunohistochemistry studies in the fetal CF lung described a three-week delay in the developmentally regulated pattern of CFTR protein expression as well as an early, intrinsic, pro- inflammatory state. Leveraging a three-dimensional, in vitro and in vivo, lung organoid model that undergoes branching morphogenesis and alveologenesis – thus recapitulating early fetal lung development – we will characterize CFTR gene transcript expression and CFTR protein expression and function and articulate the transcriptome and proteome of normal and CF lung organoids. We hypothesize that CFTR protein deficiency in the fetal CF lung results in pro-inflammatory transcriptomic and proteomic profiles in respiratory epithelial cell populations and that this can be reasonably demonstrated using a lung organoid model. To test our hypotheses and validate the findings in the lung organoids, we will also articulate the cellular multi-omes in fetal normal and CF lungs, characterizing any disease-related cellular, transcriptomic, epigenomic, and proteomic changes. The proposed research project will provide critical insights into the pathogenesis of CF lung disease and establish a new, well characterized, in vitro and in vivo, model system of the CF lung that can be leveraged in future research endeavors (e.g. viral disease modeling, therapeutic testing). The proposed research project has the potential to identify the earliest pathogenic changes in the CF lung, determine the pulmonary impact of the application of in utero CFTR modulator therapies, and determine the need for alternative, novel treatment modalities to more readily change the early trajectory of CF lung disease.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $168,000 )
2026	2026	ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI	1 GUSTAVE L LEVY PL	NEW YORK	NY	10029	NEW YORK	USA	National Center for Advancing Translational Sciences	000	1	5/5/2026	NEW	$168,000
														Subtotal = $168,000

Grand Total All Awards = $168,000

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Leveraging a lung organoid model to study early cystic fibrosis lung disease

Award Number: R03TR005349

ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 05/01/2026

PERIOD OF PERFORMANCE END DATE: 04/30/2028

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