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ALS Alert Newsletter

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Jun 7

Packard Center awards new research grant to study proteogenomic identification of TDP-43-related splicing dysfunction in ALS/FTD.

Note: This grant was made possible through a generous gift from the Bruce Edwards Foundation.

The Packard Center is pleased to announce a grant to Michael Ward, MD, PhD at the National Institutes of Health to study TDP-43-related splicing dysfunction in ALS/FTD. Ward’s recent work focuses on developing new biomarkers for ALS.

As a field, researchers recognize that loss of a crucial RNA binding protein, TDP-43, contributes to neuronal dysfunction and death. When TDP-43 is lost, hundreds of RNA transcripts become mis-spliced. Using a combination of iPSC neurons, CRISPR-inhibition, and ‘omics, Ward’s lab has developed a new platform to identify these mis-spliced transcripts and their companion protein products. Funding from the Robert Packard Center will enable Ward to determine if these splicing changes also occur in patient biospecimens, the first step in developing new biomarkers of TDP-43 function that could be useful readouts in clinical trials.

This project was funded through a grant from The Bruce Edwards Foundation.