Protein deficiency in neurons of patients with neurodegenerative diseases could be targeted by new gene therapy approach

TDP-43 is an RNA-binding protein that normally resides in the nucleus of neurons but is abnormally located in the cytoplasm of neurons in most patients with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, and up to half of patients with Alzheimer’s disease.


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Source: Phys.org