Scientists have identified a method of treatment of Huntington's disease by restoring cellular quality control process that researchers Duke Health Science Center identified as the major factor of degenerative disease.
Huntington's disease leads to a gradual erosion of nerve cells and loss of motor function, causing ill health and death. It affects about one in 10 000 people in the United States and so far incurable.
Huntington's disease develops when protein molecules are folded into the desired structural shape. Proteins accumulate and become toxic to nerve cells that control movement and mental activity. The study, published Feb. 13, the researchers looked at what causes failure of cellular processes.
When proteins fold properly, cells use a special mechanism to deal with it, - said study author J. Thiele and George Barth Geller Professor of the department of pharmacology and cancer biology. - These mechanisms return proteins back to normal three-dimensional form. In patients suffering from Huntington's disease, this is not observed. "
Thiele and colleagues conducted experiments using genetic techniques, biochemistry, screening, mouse models and stem cell ill patients. They found biochemical explanation of how quality control process is disturbed.
The researchers studied special proteins called chaperones. They contribute to the installation of other proteins. People with Huntington's disease chaperones very little, but the reason is still unknown.
Scientists have found that the mechanism for the production of chaperones called HSF1, when disease destroyed due to the presence of high levels of CK2 modifier. As a result, neurons die due to the inability to produce a sufficient level of chaperone assistants.
We have demonstrated that we could restore the protein chaperone by chemical inhibition of CK2 in a cellular model of Huntington's disease or a genetically lower levels of kinase CK2 in a mouse model - say the study authors. - We have increased the number of healthy neurons and prevented the loss of muscle mass that occurs in the disease. "
When greater numbers of functioning neurons in Huntington's disease symptoms are reduced. According to Thiele, you can create drugs that could delay or prevent the cellular processes that cause Huntington's disease and similar diseases. To study these chemicals require additional preclinical studies.