RESEARCH INST NATIONWIDE CHILDREN'S HOSP
Evaluation of Gene Replacement Therapy in In Vivo and Patient-Derived In Vitro Models of Vanishing White Matter Disease
Evaluation of Gene Replacement Therapy in In Vivo and Patient-Derived In Vitro Models of Vanishing White Matter Disease is a funding opportunity from RESEARCH INST NATIONWIDE CHILDREN'S HOSP, up to 381127, closing February 28, 2027.
Feb 28, 2027
(212d left)Overview
Project Summary/Abstract Leukodystrophy with vanishing white matter (VWM) is a severe, progressive neurodegenerative disease that most commonly afflicts infants and children. There are no disease modifying treatments. VWM is caused by autosomal recessive mutations in the five subunit genes of the Eukaryotic Initiation…