Single-dose gene therapy may stop deadly brain disorders in their tracks
Researchers have developed a single-dose genetic therapy that can clear protein blockages that cause motor neurone disease, also called amyotrophic lateral sclerosis, and frontotemporal dementia, two incurable neurodegenerative diseases that eventually lead to death.In healthy neurons, TAR DNA-binding protein 43 (TDP-43) is naturally produced and important for their healthy function. However, TDP-43 can be modified after synthesis, leading to its accumulation and aggregation in the wrong part of the cells, preventing them…