Pew Scholar Identifies Potential Targets for Treating ALS

ALS, the disease that was the focus of the Ice Bucket Challenge campaign on social media last year, has confounded scientists for decades. People afflicted with amyotrophic lateral sclerosis, the neurodegenerative illness also called Lou Gehrig’s disease, gradually lose their ability to walk, speak, and eventually breathe. Treatment options are limited, and there is no known cure.

Scientists focused on ALS may finally be closing in on some important answers, and Pew biomedical scholar Aaron Gitler, Ph.D., is one of them. Gitler and his team at Stanford University are investigating an inherited form of ALS that is often attributed to a specific genetic mutation. The dysfunctional gene triggers the production of additional ribonucleic acid (RNA) molecules. RNA provides instructions for protein production and protein molecules, called dipeptide repeat proteins, or DPRs. Scientists hypothesize that excess RNA or DPRs, or a combination of the two, contribute to the development of ALS.

Gitler set out to uncover DPRs’ functionality and understand how they might cause ALS. His team ran a series of experiments looking for genes that either increase or decrease the toxic effects of DPRs, and then categorized them by their cellular roles. Many of the genes Gitler identified make proteins that are involved in nuclear transport—the movement of proteins in and out of the cell’s nucleus. Though it is not clear exactly how, DPRs appear to be disrupting the ability of molecules to move in and out of the cell’s central compartment.

Gitler’s study suggests that drugs that help boost nuclear transport may be a therapeutic strategy to combat DPR’s effects. His work also lays the groundwork for future ALS research, with nuclear transport as the central focus, as scientists continue to try and understand the disease.

Read Aaron Gitler’s full paper, published last month in Nature Neuroscience, here. Click to learn more about the Pew Scholars Program in the Biomedical Sciences.

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