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A cure for Wolfram syndrome could lead to a cure for diabetes and blindness.

Friday, June 6, 2014

MANF, Wolfram, and Diabetes #4: Protecting remaining tissues

We are trying to target MANF to protect remaining pancreatic beta cells and brain cells in patients with Wolfram syndrome (beta cells and brain cells) and type 1 diabetes (beta cells). 

Although we hold an intellectual property related to MANF in the context of endoplasmic reticulum (ER) stress, this molecule was originally identified by Dr. John Commissiong in 2003. His research team discovered a novel neurotrophic factor in a type of brain cells. 

Dr. Commissiong has established a biotech company and has been trying to use MANF for the treatment of Parkinson disease. Recently, my team and others revealed that MANF is induced by ER stress and confers protection against ER stress-mediated cell death. Because beta cell death in Wolfram and type 1 diabetes is mediated by ER stress, MANF is an attractive drug target for these disorders. I will articulate our strategy in my next blog.