I have three things always on my mind.
1. A cure for Wolfram syndrome
2. Provide better clinical care for Wolfram syndrome
3. Raise awareness of Wolfram syndrome
To me, these three are the same, and I am doing my best to contribute to each component. For #1, I have three ideas.
#1. Drugs (chemical compounds)
#2. Biologics
#3. Cell-based therapy combined with gene therapy
I have recently realized that #2 and #3 can be potentially combined. We can potentially create “SUPER CELLS” that are resistant to cell stress and transplant them to our patients. I was quite encouraged by a clinical trial for ALS patients based on this concept. SUPER CELLS can also produce hormones that make our cells more resistant to cell stress. MANF is a emerging target in this regard because this hormone makes our cells more resistant to cell stress, more specifically ER stress.
Thank you so much for reading this blog. I always appreciate your support.
Kindly,
Fumi Urano