is a non-profit corporation whose board includes the states biggest land managers and landowners, comprising: US Forest Service, Minnesota Department of Natural Resources, University of Minnesota, Minnesota Power, county land departments, forest industry, and private landowners.
The team used iPS technology to transform skin cells taken from two RP patients--each with a different MFRP mutation--into retinal cells, creating patient-specific models for studying the disease and testing potential therapies.
By analyzing these cells, the researchers found that the primary effect of MFRP mutations is to disrupt the regulation of actin, the protein that makes up the cytoskeleton, the scaffolding that gives the cell its structural integrity.
The researchers also found that MFRP works in tandem with another gene, CTRP5, and that a balance between the two genes is required for normal actin regulation.
The team then used adeno-associated viruses (AAVs) to introduce normal copies of MFRP into the iPS-derived retinal cells, successfully restoring the cells' function.