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Dr. Urano May 2018 Wolfram Update
Dear Friends,
First of all, I would like to express my gratitude to you for coming to see me today. Thank you so much for following my mission & vision and being the kindest person. I think about patients with Wolfram syndrome and their families and friends every morning. That’s one of the first things I do every day at 4:30 am. I would like to support, help, and save them. I would like to know their challenges and help them overcome these challenges. Three things are always on my mind: 1. Improve Clinical Care, 2. Raise Awareness, and 3. Provide a Cure. We are clearly making progress in #1 and #2. How about #3? Is it possible? If so, how long?
Here is my answer. “We are making progress. We are this close.”
This close = my head size. I have all the strategies and ideas for developing cutting-edge treatments for Wolfram syndrome in my head. My challenge is to realize these ideas. There are technical roadblocks for developing gene therapy. There are regulatory issues to bring new drugs from bench (lab) to bedside (patients). There are financial constraints. These are not so easy to overcome, but these are much smaller challenges than those our patients have been experiencing. I would like to articulate my strategies again. There are three steps. Step 1: Drug Therapy for halting progression. Step 2: Regenerative therapy for protecting and regrow remaining eye and brain cells. Step 3: Gene therapy for replacing pathogenic genes. To achieve these goals and accelerate our progress, I have started creating three new animal models (mice and rats) carrying human Wolfram gene mutations. They are humanized Wolfram mice and rats.
I plan to use these animals to test gene therapy and regenerative therapy.
In addition, I have been developing “genetic testing” for screening Wolfram syndrome and Wolfram-related diseases. I believe that Wolfram syndrome is an underdiagnosed disease. Using a single tube of blood, I would like to provide an accurate diagnosis. An accurate diagnosis serves as a basis for targeted therapy. An accurate diagnosis provides a sense of relief.
As always, please feel free to contact me with any questions or concerns (urano@wustl.edu). I would like to know what you think and how you feel. Thank you again for your support. Our potential is limitless. We have superpower to overcome this challenge.
With passion, hope, and gratitude,
Fumi Urano
Dear Friends,
First of all, I would like to express my gratitude to you for coming to see me today. Thank you so much for following my mission & vision and being the kindest person. I think about patients with Wolfram syndrome and their families and friends every morning. That’s one of the first things I do every day at 4:30 am. I would like to support, help, and save them. I would like to know their challenges and help them overcome these challenges. Three things are always on my mind: 1. Improve Clinical Care, 2. Raise Awareness, and 3. Provide a Cure. We are clearly making progress in #1 and #2. How about #3? Is it possible? If so, how long?
Here is my answer. “We are making progress. We are this close.”
This close = my head size. I have all the strategies and ideas for developing cutting-edge treatments for Wolfram syndrome in my head. My challenge is to realize these ideas. There are technical roadblocks for developing gene therapy. There are regulatory issues to bring new drugs from bench (lab) to bedside (patients). There are financial constraints. These are not so easy to overcome, but these are much smaller challenges than those our patients have been experiencing. I would like to articulate my strategies again. There are three steps. Step 1: Drug Therapy for halting progression. Step 2: Regenerative therapy for protecting and regrow remaining eye and brain cells. Step 3: Gene therapy for replacing pathogenic genes. To achieve these goals and accelerate our progress, I have started creating three new animal models (mice and rats) carrying human Wolfram gene mutations. They are humanized Wolfram mice and rats.
I plan to use these animals to test gene therapy and regenerative therapy.
In addition, I have been developing “genetic testing” for screening Wolfram syndrome and Wolfram-related diseases. I believe that Wolfram syndrome is an underdiagnosed disease. Using a single tube of blood, I would like to provide an accurate diagnosis. An accurate diagnosis serves as a basis for targeted therapy. An accurate diagnosis provides a sense of relief.
As always, please feel free to contact me with any questions or concerns (urano@wustl.edu). I would like to know what you think and how you feel. Thank you again for your support. Our potential is limitless. We have superpower to overcome this challenge.
With passion, hope, and gratitude,
Fumi Urano
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