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

Saturday, June 7, 2014

MANF, Wolfram, and Diabetes #5: Protecting remaining tissues

We have been 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). 

All of us have this molecule in our bodies, and our strategy is to enhance the effect of MANF. There are at least three ways to do it.

1. Inject recombinant MANF to patients.

We can crate MANF in the tissue culture dish using biotechnology. We then purify this "artificial" MANF and use it for the treatment.

2. Stabilize endogenous MANF

There is continuous process of loss and replacement of MANF in our bodies. MANF is degraded after certain amount of time in our cells. If we find a way to stabilize MANF, we can enhance the effect of MANF.

3. Activate a receptor for MANF

Many secreted proteins in our bodies have their "receptors." These receptors mediate the effect of their partner secreted proteins. For example, we have "insulin" receptors to mediate the effect of insulin. We are trying to identify a receptor for MANF, so that we can develop a drug that can activate this receptor.

I will continue this topic.