TOWARDS A REVOLUTIONARY THERAPY FOR BLINDNESS AND

TOWARDS A REVOLUTIONARY THERAPY FOR BLINDNESS AND DIABETES IN WOLFRAM SYNDROME
I am excited by a new collaboration with Amarantus Bioscience to develop A REVOLUTIONARY THERAPY FOR WOLFRAM SYNDROME, the most difficult form of blindness and diabetes. We will use MANF protein derived from human brain to achieve this goal. Amarantus is a leading biotech company developing MANF-based therapies for neurodegeneration and blindness, and my team has expertise in Wolfram syndrome. This will be a wonderful collaboration. Please see the press release in the Wall Street Journal.
http://online.wsj.com/article/PR-CO-20140620-905760.html

Wolfram syndrome is caused by stress in the endoplasmic reticulum (ER), a cell component involved in many vital functions of eyes, brain, and pancreas. Despite the underlying importance of ER stress in Wolfram syndrome, no currently available drugs can control ER stress. MANF has been shown to block ER stress. So we will test if MANF protein can block ER stress in eye cells created from induced pluripotent stem cells derived from Wolfram syndrome patients. How did we purify stem cells from Wolfram syndrome patients? By a new method developed by a Japanese physician-scientist, we created stem cells from patients’ skin cells! We have chosen eye cells to test the efficacy of MANF first because BLINDNESS IS THE MOST DEVASTATING QUALITY-OF-LIFE ISSUE faced by patients with Wolfram syndrome. If successful, MANF-based therapies for blindness and diabetes will be established because ER stress is an emerging target for both diseases.