"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."