Award
National Heart, Lung, and Blood Institute 5R01HL166462-04
Using human liver tissue equivalents to optimize AAV-mediated GT and better define age-related clinical risks
Recipient
Wake Forest University Health Sciences
Award Amount
$383,674.00
Ceiling
$383,674.00
Awarded
February 26, 2026
Identifier
5R01HL166462-04
This NIH-funded project uses human liver tissue equivalents to improve the safety and effectiveness of AAV-based gene therapy for hemophilia A, with a focus on age-related clinical risks and pediatric applications.
Description
The project aims to utilize a human liver tissue equivalent (hLTE) platform to address questions related to AAV-mediated gene therapy for hemophilia A, focusing on optimizing safety and efficacy, especially in pediatric populations. It addresses risks such as pre-existing AAV antibodies, hepatotoxicity, and potential genome integration leading to hepatocellular carcinoma.