Elucidating CHD in Down Syndrome with Cardiac Organoids and 3D Genome Architecture

NIH RePORTER · NIH · R01 · $385,799 · view on reporter.nih.gov ↗

Abstract

PROJECT SUMMARY Down syndrome (DS) or Trisomy 21 (T21) is a prevalent chromosomal defect worldwide, often associated with multiple cardiovascular and hematological anomalies. Congenital heart disease (CHD), affecting up to 50% of DS patients, is a leading cause of morbidity and mortality in infants. CHD is primarily caused by dysregulated transcriptional pathways. Although the 3D spatial organization of the genome has emerged as a crucial mechanism for regulating chromatin accessibility, its role in cell-to-cell variability and gene regulation within CHD remains poorly understood. In Aim 1, we will utilize 3D-vascularized cardiac organoids (3D-vCOs) and multi- omics approaches to elucidate the mechanisms of DS-associated cardiovascular complications. We will employ single-cell RNA sequencing (scRNA-seq) and single-cell ATAC sequencing (scATAC-seq) to analyze the vascular network and cardiomyocyte (CM) characteristics, thereby establishing phenotypes of DS-CHDs. Our study will specifically focus on analyzing the vascular network and cardiomyocyte (CM) characteristics to establish phenotypes of Down syndrome-associated congenital heart defects (DS-CHDs). In Aim 2, we will perform Dip-C (Diploid Chromosome Conformation Capture) at specific timepoints during 3D-vCO culture (Day 3, Day 6, and Day 16) using DS patients iPSC lines. We will analyze the inter-chromosomal interactions (ICIs) and 3D architecture of the genome in 3D-vCOs, namely focusing on differences among the CMs, CFs, and ECs between DS-CHD versus DS-nonCHD patients. We anticipate gaining new mechanistic insights into the pathogenesis of DS-CHDs and facilitating the development of novel therapeutic strategies aimed at mitigating and reversing cellular abnormalities in the diseased heart.

Key facts

NIH application ID
10852469
Project number
3R01HL141371-06S2
Recipient
STANFORD UNIVERSITY
Principal Investigator
Lei Stanley Qi
Activity code
R01
Funding institute
NIH
Fiscal year
2023
Award amount
$385,799
Award type
3
Project period
2018-05-01 → 2026-05-31