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

Sunday, January 12, 2014

Treating vision impairment part 2 - my concept

We are actively working on induced pluripotent stem cells (iPSCs) to replace damaged tissues in patients with Wolfram syndrome and type 1 diabetes in the future. iPSCs are a type of stem cells derived from skin cells through forced expression of specific genes. In short, we can create any type of cells from skin cells of each patient through iPSCs.

We have created iPSCs using skin cells from patients with Wolfram syndrome here at Washington University. These cells can be differentiated into any types of cells including retinal ganglion cells and pancreatic beta cells that are severely damaged in Wolfram syndrome. We have already created neural progenitor cells from iPSCs. Our next step is to make retinal ganglion cells from iPSCs-derived neural progenitor cells. Our future goal is to correct genetic defects in iPSCs, make iPSC-derived retinal ganglion cells, and transplant these cells to patients.

This strategy will be used for treating age-related macular degeneration, a leading cause of vision loss among people age 50 and older, and the first clinical trial will start in March, 2014 in Japan. I sincerely hope that this trial will be successful. The information about this trial is summarized in Nature.