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Breakthrough Gene Therapy Tool Illuminates Path to Treating Neurological Disorders

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The Researcher Daily
May 22, 2025
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Table S1. Predicted accessibility of all putative pan-SMN enhancers, transcription factor motifs within the 1137m enhancer sequence, and variance in completeness and specificity measures

Scientists have unveiled a groundbreaking genetic tool that promises to transform research into spinal cord injuries and neurodegenerative diseases like ALS. Developed by researchers at the Allen Institute, this innovative technology uses engineered viruses to precisely target and manipulate the nerve cells responsible for movement.

The new approach func…

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