Researching Mutants

The first potential treatment drugs for Glaucoma have been tested by an American Research Facility in Atlanta. In a study published at the end of last year the findings are the result of a collaborative effort between Emory University, Georgia Institute of Technology Atlanta, and South Florida University. The Associate Professor at the Georgia Tech School of Chemistry and Biochemistry led the research to try and identify molecules that may assist in blocking the build up of toxic proteins within the eye that aggravate the onset of glaucoma.As high pressure within the eye is what causes nerve damage leading to loss of sight the research is vital to producing a viable treatment for a disease that affects millions of people globally.

The researchers designed a straight forward high through-put assay then screened collective compounds. What they pulled from the data was two molecules that appeared to bind mutant myocilin. Mutant myocilin is toxic to the cells in the eye that regulate pressure. They form into clumps in front of the eye that blocks the natural drainage of fluid out of the eye increasing overall pressure.

By combining myocilin with a fluorescent compound, more light can be detected from it when the protein is unwound. If it combines with a mutant it then  becomes stable and as such tightly bound emitting less fluorescent light. The measurement of the fluorescence  helped show which molecules were being bound. Once identified , they were added to cultured human cells that were already making the poisonous mutants. treatment of these cells with the newly identified molecules blocked the aggregation and caused the mutated version of myocilin to be released from the cells, reducing toxicity.

To forward the research, they are now mapping out the structure of myocilin to discover it's biological role within the eye.