Scientists Discover Protein ‘Zip Codes’

Canadian scientists say they are very close to defining small molecule drugs that might lead to a therapy for Huntington’s disease.

The McMaster University researchers say they expect their study to result in the ability to redirect the huntingtin protein from accumulating in the wrong place within brain cells.

There is currently no way to stop or reverse Huntington’s disease, which is a progressive, and eventually fatal, genetic neurological disease.

But McMaster Associate Professor Ray Truant’s lab has discovered molecular zip codes — or protein sequences in the huntingtin protein — that dictate where it goes within a brain cell.

We have shown that the mutant huntingtin protein is mis-localized in brain cells in Huntington’s disease, because it is being improperly signaled, or instructed where to go in the cell, said Truant. In particular, huntingtin is accumulating at the heart of the cell, the nucleus, where it shouldn’t be. This is causing the brain cells to not function properly, and eventually die.

Truant and his colleagues have received a $260,000 grant from the U.S.-based High Q Foundation to pursue the research.