A synthetic protein against neurodegenerative diseases

The first synthetic version of the protein at the origin of serious neurodegenerative diseases such as Alzheimer's will allow us to fight these pathologies, shedding light on their key mechanism and leading to new weapons for both diagnosis and therapy : it was obtained in the laboratory by a group of researchers from Northwestern University and the University of California at Santa Barbara, who published the results obtained in the journal Pnas of the American National Academy of Sciences.

The synthetic tau protein developed by researchers coordinated by Songi Han is a reduced version of the original one , but it behaves exactly the same way , folding incorrectly , aggregating into tangles and transmitting the defect to the surrounding proteins, in an endless chain reaction. "We have created a mini version that is easier to control," says Han, "but it performs the same functions as the full-size protein." The accumulation of misfolded tau proteins is precisely at the basis of the progression of this class of neurodegenerative diseases, also called 'tauopathies'.

The results show that the mutation called P301L plays a fundamental role: it facilitates, in fact, a type of incorrect folding often observed in patients, but not only. It seems that this mutation directly influences the behavior of the water molecules that surround the proteins , and this is what allows them to clump together in tangles and filaments.

“Water is a fluid molecule,” Han says, “but it still has its own structure: the mutation could lead to a more structured arrangement of the surrounding water molecules, which influences the way the protein interacts with the others, binding them together. If we could understand how to block this activity,” the researcher concludes, “we could discover new therapeutic agents.”