Scientists can now target the cells at the center of ALS

The major scientific discovery opens the door to new treatments for the devastating disease. 

Originally published by Liz Dueweke / Allen Institute/ on  June 16, 2025

ALS is a cruel disease. It robs the body of its ability to control itself—the ability to move, the ability to communicate. While there are currently no effective treatments to reverse its debilitating symptoms, Allen Institute researchers have opened a window of hope. 

For the first time ever, scientists have developed a precise genetic toolkit that can target the exact nerve cells destroyed by the disease and potentially deliver therapies where they are needed most—a discovery that could dramatically speed up the quest for a cure. The findings were recently published in the journal Cell Reports. 

Image of a spinal cord section taken from the lumbar region. An enhancer AAV expressing a bright fluorescent protein in motor neurons was delivered to the animal and spinal cord sections were stained to reveal viral label in choline acetyltransferase or ChAT positive neurons (yellow = merge of two channels; green virus and red ChAT). Right inset image shows motor neuron cell bodies and axons emanating from the spinal cord tissue.

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