Award
National Heart, Lung, and Blood Institute 5R01HL168659-04
Genetic and molecular basis of hematopoietic abnormalities in ZTTK syndrome
Recipient
University of Alabama at Birmingham
Award Amount
$508,794.00
Ceiling
$508,794.00
Awarded
February 11, 2026
Identifier
5R01HL168659-04
Research grant awarded to study hematopoietic abnormalities in ZTTK syndrome using mouse models to understand molecular mechanisms and develop therapeutic strategies.
Description
This project aims to examine hematopoietic abnormalities using mouse models, focusing on how SON haploinsufficiency affects hematopoietic stem and progenitor cells (HSPCs). Preliminary data suggest that SON haploinsufficiency alters chromatin modifier gene expression, skewing lineage bias and impairing HSPC function. The project investigates the functional output of HSCs and lineage-primed MPPs in vivo and dissects the molecular mechanisms by which SON haploinsufficiency leads to HSPC abnormalities.