Finding cures for children's genetic diseases


Example proteomics project: Dynamin

The question: is it possible to control nerve communication to ultimately treat various brain and nerve disorders?

Many years ago, the Cell Signalling Unit discovered that the protein dynamin plays a central role in a process called endocytosis, which is crucial to nerve communication (or nerve cell signalling).

The group initially discovered dynamin as a phosphoprotein (a protein with a phosphate molecule attached to it) that is rapidly de-phosphorylated upon stimulation of nerve terminals. Using various techniques, including proteomics, the group deciphered the molecular mechanisms of dynamin’s phosphorylation cycle, and they have now identified all of the specific sites of phosphorylation in dynamin.

Their work revealed that each site plays an essential role in endocytosis, and that they act cooperatively for maximal effect. They were also able to discover dynamin’s protein partner for endocytosis in neurons. These findings have led directly to the development of a new class of drugs to potentially treat epilepsy and to greater understanding of nerve communication that may one day lead to treatments for other nerve disorders.