Toward personalized regenerative gene therapy for Wolfram syndrome

Regenerative Gene Therapy

Dear Friends,

I hope you and your family are safe and well during this COVID-19 pandemic. Three things are always on my mind: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. As I mentioned on January 1st, I am determined to make 2020 the game-changing year for us despite this challenging time. Today, I would like to share the good news with you.

We have been testing if gene editing by CRISPR-Cas9, in combination with patient-derived induced pluripotent stem cells (iPSCs), can be utilized for the treatment of Wolfram Syndrome. I am glad to inform you that gene editing worked in Wolfram patient iPSC-derived beta cells. We were able to use these cells to cure one of the problems, making normal beta cells by correcting the WFS1 gene mutation. We could cure diabetes in cells and mice. This is a proof of concept demonstrating that correcting gene defects that cause or contribute to medical problems— in this case, in the Wolfram syndrome gene — we can cure the problems. This is a major discovery in the gene therapy field, and it has been just published in a high-profile medical research journal, Science Translational Medicine.

It is possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram Syndrome patients experience, such as visual impairment and neurodegeneration. So we are currently working on eye and brain cells derived from iPSCs of patients with Wolfram Syndrome to replicate this success for other problems. Genetic testing will guide us to identify gene mutations that should be corrected, which will lead to personalized regenerative gene therapy for our patients. Please feel free to contact me if you have any questions related to genetic testing.

I would like to thank my patients, colleagues at Washington University, and supporters in the world. As always, please feel free to contact me with any questions ( I would like to know what you think and how you feel. Thank you again for your continued support and encouragement, especially in this very trying time, not only for our country but for the world. We will work as one team and change history together.

“That’s one small step for us today, leading to one giant leap toward a cure for Wolfram Syndrome.”

Fumi Urano
Sunday, April 25, 2020