Eye Hope Foundation

Project of Professor Urano

"Study of the the efficacy of a new small molecule (JTV-519 and oxidized form of JTV-519) in 
mouse and rat models of Wolfram syndrome by Prof. Fumihiko Urano - Division of 
Endocrinology,Washington University, St Louis, USA. We will test the  efficacy of JTV-519 and 
the best JTV-519 derivative in cell-specific WFS1 gene knockout mice, a diabetes mouse 
model of Wolfram syndrome, and neuronal-specific WFS1 gene knockout mice, a 
neurodegeneration mouse model of Wolfram syndrome.
To study the efficacy of any new treatments, especially in eyes and brain, it would be 
important to develop a larger animal model of Wolfram syndrome. Recent advancement of 
CRISPR/Cas9 technology has enabled my research team to create a rat model of Wolfram 
syndrome. We propose to create whole-body WFS1 gene knockout rats using the 
CRISPR/Cas9 technology and assess cell functions, visual acuity, and balance in these rats."