We are looking for disease modifying loci for Wolfram syndrome to identify new drug targets. What are disease modifying loci? These are the genes that may play a role in delaying or accelerating the progression of the disease. In addition to WFS1 or WFS2, it seems like there are genes affecting the manifestations of the disease. If we can identify these genes, we may be able to target the products of these genes to delay the progression. So we are searching for these genes.
Thank you for reading this blog. I hope you will have a wonderful week. I am sending kind thoughts toward you. Warmly, Fumi Urano
I received several questions related to my blog of September 26, 2014.
http://wolframsyndrome.blogspot.com/2014/09/soluble-factors.html
How can we utilize MANF, a natural soluble factor produced in our body, to protect and proliferate remaining beta cells and neurons?
The key is to understand how MANF simulates the proliferation and enhances the function of remaining tissues. It seems like there are two independent mechanisms. There are two different mediators for MANF in our body. Thus, our goal is to develop two different types of small molecules to enhance the functions of endogenous MANF. Another option is to inject recombinant MANF. Because of several scientific reasons, we prefer small molecules at the moment.
Thank you for reading this blog. I hope you will have a wonderful and beautiful Sunday. Please feel free to contact me if you have any questions. I am here for you. Take care, Fumi Urano
I keep on figuring out how to delay the progression and provide a cure for patients with Wolfram syndrome. In parallel, I keep on thinking about how we can improve the quality of life of our patients. One of the possibilities is to create a planform where patients can share their experiences. There are multiple non-profit and for-profit organizations working on this. Patients-like-me is one of them.
http://www.patientslikeme.com/
I am willing to donate our data from our patients to this type of organization. Of course, the data should be de-identified. If it is difficult to do it because of all the regulations, I am willing to help people create such a platform. By connecting each other, we would feel empowered, I think.
Thank you for reading this blog. I hope you will have a wonderful weekend. Kindly, Fumi Urano
We are looking for soluble factors in our body that can enhance the regeneration of damaged tissues in Wolfram syndrome and Type 1 diabetes. We have discovered two molecules so far. One of them is MANF.
We are trying to produce a large amount of these factors in the dish (recombinant proteins) and give these factors to animal models and cell models. We are also looking for methods/small molecules (i.e., drugs) to enhance the functions of these factors. This strategy is also related to "regenerative therapy", but independent of stem cells.
Thank you for reading this. I hope you will have a wonderful Friday. Warmly, Fumi Urano
