Study Shows Gene Therapy Shields Brain from TDP-43 Damage
Summary: Researchers report that an experimental systemic gene therapy can protect the central nervous system from the cognitive decline and structural damage driven by TDP-43–related proteinopathy. The treatment uses a neuron-targeted approach that delivers the SynCav1 gene throughout the brain and spinal cord via a modified, nonpathogenic viral vector to increase levels of caveolin-1, a … Read more