Novel Druggable Target for Wolfram Syndrome
- Wolfram syndrome = rare genetic disorder
- Loss of function of gene WFS1
- Homozygous mutation (1 in 770,000 in US)
- blindness, deafness, mood disorders
- Heterozygous patients
- 1% of US, 8-fold higher mood disorders
- No available treatment
- palliative care only
- Target structures + hits known
- Screenable/Structure-based drug design
- Animal models available for in vivo validation