Wednesday, August 20, 2025 8/20/2025

The role of RNA binding proteins in heart development and congenital heart defects

Award Number: R01HL157780
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 04/12/2023
PERIOD OF PERFORMANCE END DATE: 04/30/2026

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The role of RNA binding proteins in heart development and congenital heart defects - ABSTRACT Hypoplastic left heart syndrome (HLHS) is a congenital heart disease with a high mortality rate. Infants with this syndrome are born with circulation problems due to hypoplasia of the left part of the heart that includes aorta, left ventricle, mitral valve and aortic valve. Infants may need to undergo a series of open-heart surgeries to restore systemic circulation. Even after the surgeries, these patients can develop heart failure and other complications due to the overload on the right ventricle that serves as the single ventricle. Very little is known about the etiology and pathogenic mechanisms of HLHS. Recent human genetic studies identified de novo loss of function mutations in the RNA binding protein RBFOX2 that are linked to the HLHS phenotype. It is currently unknown how RBFOX2 contributes to HLHS developmental defects. This is mainly because of our lack of knowledge regarding the role of RBFOX2 in cardiovascular development and its target RNAs in the embryonic heart. Using our Rbfox2 conditional knockout mouse model, which recapitulates several phenotypic, cellular, and molecular features of HLHS, we will address these urgent and clinically relevant questions utilizing genetic, molecular/cellular and state-of-the art custom designed sequencing and computational approaches. Our main goal is to determine the mechanisms by which RBFOX2-regulated RNA networks contribute to cardiovascular development and investigate their contributions to HLHS. Our work will determine the underlying mechanisms of HLHS developmental defects caused by loss of RBFOX2. Ultimately, our findings will advance development of innovative HLHS therapeutic strategies by restoring aberrant RBFOX2-regulated gene expression in HLHS patients using modified antisense oligonucleotides in the future.


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: 2025 ( Subtotal = $728,985 )
2025	2025	RECTOR & VISITORS OF THE UNIVERSITY OF VIRGINIA	1001 EMMET ST N	CHARLOTTESVILLE	VA	22903	CHARLOTTESVILLE CITY	USA	Cardiovascular Diseases Research	000	4	5/27/2025	NON-COMPETING CONTINUATION	$728,985
														Subtotal = $728,985

Issue Date FY: 2024 ( Subtotal = $719,153 )
2024	2024	RECTOR & VISITORS OF THE UNIVERSITY OF VIRGINIA	1001 EMMET ST N	CHARLOTTESVILLE	VA	22903	CHARLOTTESVILLE CITY	USA	Cardiovascular Diseases Research	001	3	6/20/2024	NON-COMPETING CONTINUATION	$58,706
2024	2024	RECTOR & VISITORS OF THE UNIVERSITY OF VIRGINIA	1001 EMMET ST N	CHARLOTTESVILLE	VA	22903	CHARLOTTESVILLE CITY	USA	Cardiovascular Diseases Research	000	3	5/3/2024	NON-COMPETING CONTINUATION	$660,447
2024	2023	RECTOR & VISITORS OF THE UNIVERSITY OF VIRGINIA	1001 EMMET ST N	CHARLOTTESVILLE	VA	22903	CHARLOTTESVILLE CITY	USA	Cardiovascular Diseases Research	003	2	7/17/2024	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$426,810
2024	2022	UNIVERSITY OF TEXAS MEDICAL BRANCH AT GALVESTON	301 UNIVERSITY BLVD	GALVESTON	TX	77555	GALVESTON	USA	Cardiovascular Diseases Research	002	1	7/17/2024	NEW	-$426,810
														Subtotal = $719,153

Issue Date FY: 2023 ( Subtotal = $733,830 )
2023	2023	RECTOR & VISITORS OF THE UNIVERSITY OF VIRGINIA	1001 N EMMET ST	CHARLOTTESVILLE	VA	22903	CHARLOTTESVILLE CITY	USA	Cardiovascular Diseases Research	001	2	5/25/2023	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$733,830
2023	2022	UNIVERSITY OF TEXAS MEDICAL BRANCH AT GALVESTON	301 UNIVERSITY BLVD	GALVESTON	TX	77555	GALVESTON	USA	Cardiovascular Diseases Research	000	1	5/25/2023	NEW	$0
														Subtotal = $733,830

Issue Date FY: 2022 ( Subtotal = $759,511 )
2022	2022	UNIVERSITY OF TEXAS MEDICAL BRANCH AT GALVESTON	301 UNIVERSITY BLVD	GALVESTON	TX	77555	GALVESTON	USA	Cardiovascular Diseases Research	000	1	5/12/2022	NEW	$759,511
														Subtotal = $759,511

Grand Total All Awards = $2,941,479

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The role of RNA binding proteins in heart development and congenital heart defects

Award Number: R01HL157780

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 04/12/2023

PERIOD OF PERFORMANCE END DATE: 04/30/2026

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