Regenerative gene therapy


Thank you so much for visiting our website for Wolfram syndrome patients, their families, friends, and supporters.

I hope you and your family are enjoying the beautiful autumn season and nature. I love to do a nature walk especially in autumn, and I came up with the following idea during my nature walk. I’d like to share this idea with you as I know you are my friend and supporter.

To provide a cure for Wolfram syndrome, we need to protect our eye cells, brain cells, and insulin-producing cells. If they are damaged, we need to fix them and even regenerate them. I came up with the concept of “Regenerative Gene Therapy.” This is a combination of regenerative therapy and gene therapy. As you know, my team has discovered a molecule that is produced naturally in the body that can activate the proliferation of damaged beta cells and brain cells. Expression levels of this molecule, MANF, are usually low in the human body. We are developing a method to enhance the activity of MANF using a gene therapy. We are trying to produce a safe virus expressing MANF to transduce into patients’ cells with resultant creation of MANF within the cell. This treatment could then be applicable to Wolfram syndrome, as well as other forms of neurodegenerative disease.

As always, please feel free to contact me with any questions or concerns ( I would like to know what you think and how you feel. I always appreciate your support and encouragement. You are a wonderful person. We will decrease human suffering together.

With gratitude,
Fumi Urano