Mechanism revealed that dooms patients with Lou Gehrig’s disease

Memphis, TN, April 10, 2019

Richard Kriwacki, Ph.D., and colleagues

Richard Kriwacki, Ph.D., and colleagues used a 1.1 GHz nuclear magnetic resonance system in this study to reveal in greater detail how toxic DPRs bound to intrinsically disordered regions of nucleophosmin.

Sometimes oil and water work best apart.

Consider the nucleolus. It is a compartment inside the nucleus where the cells’ protein factories are assembled. The nucleolus lacks a membrane. Instead, to maintain its shape and function, the nucleolus relies on the same process that causes oil to form droplets in water.

St. Jude researchers have discovered that the most common genetic cause of Lou Gehrig’s disease, also known as ALS, disrupts that process. The findings help settle the mystery of how the mutation leads to the death of cells in patients with the disorder.

“We have identified a protein in cells whose function is changed by the mutation,” said Richard Kriwacki, PhD, of Structural Biology. “The finding solves the mystery of how the mutation leads to cell death.

“The work may also lead to new diagnostic tests or new treatments for this deadly disease.”

A report on this work appeared in the journal Molecular Cell

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