Dr. Urano's Blog - Three things always on my mind

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

http://wolframsyndrome.dom.wustl.edu/three-th...n-my-mind/