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New Review Focuses on the Connection of Disturbances in RNA-Binding Proteins to ALS

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. In this series of articles, we are celebrating some of the key innovations helping us change the nature of ALS forever.

In the May 2018 issue of Frontiers in Neuroscience, current The ALS Association-funded researchers, Drs. J. Paul Taylor and Maria Purice of St. Jude Children’s Research Hospital in Memphis, published a comprehensive mini-review describing disease pathways that cause ALS, with a focus on mutations in RNA-binding proteins.

Many recent innovations in ALS animal and cells models that were instrumental in understanding the role of RNA-binding proteins in ALS are described.

Dr. Purice is a current Milton Safenowitz fellow who is currently studying how molecular pathways lead to both inherited (in which researchers have identified mutated ALS genes) and sporadic (non-inherited) forms of ALS, with a focus on RNA-binding protein, TDP-43.

In this review, Drs. Purice and Taylor do a deep dive into how disturbances in RNA-binding proteins, including TDP-43, cause ALS.

RNA-binding proteins are a large class of proteins that play a role in RNA metabolism. They are among the most abundant proteins in cells that reside both in the cell’s nucleus and surrounding cytoplasm.

Over the last decade, mutations in RNA-binding proteins have been linked to ALS and frontotemporal dementia (FTD), including TDP-43, FUS, hnRNPs A1 and A2B1, MATR3, and TIA1.

Learn more about RNA-binding proteins, with a special focus on the above proteins’ normal function, and what happens in cells when those proteins’ normal function is disrupted, leading to characteristics of ALS.

The ALS Association proudly supports both Dr. Taylor’s and Dr. Purice’s projects exploring RNA-binding proteins.

To learn more about Biohaven’s commitment to advances in ALS and other neurological diseases, click here.

Purice MD and Taylor JP (2018) Linking hnRNP Function to ALS and FTD Pathology. Front. Neurosci. 12:236. Doi: 10.3389/fnins.2018.00326
*This publication is open access.