Novel therapeutic target for Huntington's disease identified

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Washington, Dec 25 (ANI): Scientists from University of California, Irvine have identified a potential therapeutic target for Huntington's disease.

In Huntington's disease, a mutated protein in the body becomes toxic to brain cells. Researches have shown that a small region adjacent to the mutated segment plays a major role in the toxicity.

The new study shows that very slight changes to this region can eliminate signs of Huntington's disease in mice

"These studies shed light on the structure and biochemistry of the mutant huntingtin protein and on potentially modifiable factors that affect its toxicity," said Dr Margaret Sutherland, a program director at NIH's National Institute of Neurological Disorders and Stroke (NINDS).

"They reveal sites within the huntingtin protein and within broader disease pathways that could serve as targets for drug therapy," she added.

According to the researchers, individuals with the disease carry mutations that affect the huntingtin protein.

Lead researchers Leslie Thompson and Joan Steffan, Ph.D. investigated how a process called phosphorylation affects huntingtin.

Phosphorylation is the attachment of chemical tags, known as phosphates, onto the amino acids in a protein.

The study shows that phosphorylation of just two amino acids, located at one end of huntingtin, targets the protein for destruction and protects against the toxic effects of the mutant protein.

The researchers found that modification of a huntingtin fragment reduced its tendency to form clumps.

Together, data from the mouse and test tube experiments provide strong support for the idea that phosphorylation acts as a molecular switch to alter clumping of the mutant protein.

They suggest that drugs that enhance or mimic the effects of phosphorylation may help to detoxify the mutant huntingtin protein.

The study is published in the Journal of Cell Biology. (ANI)

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