One-time intervention. Gene therapy and fatal brain diseases
<p>Healthy neurons naturally produce “TAR DNA binding protein 43,” called TDP-43 for short. This protein is important for healthy neuronal function. However, TDP-43 can be altered after synthesis, causing it to accumulate and concentrate in the “wrong” part of cells, preventing them from functioning normally.</p>
<p>These accumulations are associated with devastating neurodegenerative diseases such as motor neuron disease (MND), also known as amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease, and frontotemporal dementia (FTD).</p>
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