I'd like to propose a two-step strategy to achieve a cure for Wolfram syndrome. The first step is to stop the progression using a small molecule (i.e., a drug) and the second step is to replace the damaged tissues using a stem cell technology. In parallel, we try to restore the functions of remaining pancreatic beta cells, eye cells, and brain cells using a small molecule or biologics such as MANF. A new technology called a "genome editing" can be combined with a "gene therapy." All of these are ongoing in my team.
I believe that these are all achievable goals. John Crowley, a father of two children with Pompe disease, and William Canfield, MD, PhD, a Pompe disease researcher, worked together, established a biotech, and discovered a treatment for the disease. Mr. Crowley could save his children's lives and is now looking for a cure. If they can do it, we can do it.
https://en.wikipedia.org/wiki/John_Crowley_(biotech_executive)
https://en.wikipedia.org/wiki/William_Canfield
