Award
NIH Reporter #5R01HL165247-04
Targeted conditioning to maximize prenatal HSC engraftment for SCD
Recipient
Wake Forest University Health Sciences
Award Amount
$597,782.00
Ceiling
$597,782.00
Awarded
August 14, 2026
Identifier
5R01HL165247-04
The NIH awarded a grant to Wake Forest University Health Sciences to develop targeted conditioning strategies to improve prenatal hematopoietic stem cell engraftment for sickle cell disease, using fetal sheep models to overcome barriers to successful intrauterine transplantation.
Description
This project aims to identify obstacles to intrauterine transplantation (IUTX) success and test novel strategies to enable successful application of IUTX to diseases such as sickle cell disease (SCD). The study will utilize fetal sheep as a model to define barriers to engraftment, assess the impact of donor HSC phenotype, and evaluate the therapeutic efficacy of IUTX for SCD. The goal is to achieve 20-25% HSC engraftment to make IUTX a viable therapy for hemoglobinopathies.