Award
NIH Reporter #1R61NS140718-01A1
Evaluation of Gene Replacement Therapy in In Vivo and Patient-Derived In Vitro Models of Vanishing White Matter Disease
Recipient
Not Specified
Award Amount
$381,127.00
Ceiling
$381,127.00
Awarded
March 09, 2026
Identifier
1R61NS140718-01A1
This award supports the development and testing of gene replacement therapy for Vanishing White Matter Disease using in vivo mouse models and patient-derived in vitro human organoids, with a focus on astrocytic pathogenesis.
Description
The project involves developing and testing AAV-mediated gene therapy for Vanishing White Matter Disease (VWM). It includes preclinical testing in mouse models and validation in human organoids, aiming to develop a translatable therapy targeting astrocytic pathogenesis.