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Single-dose gene therapy may stop deadly brain disorders in their tracks

Published in Brain/Neurology, Tools.

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 from working properly. These build-ups are associated with devastating neurodegenerative diseases like motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease, and frontotemporal dementia (FTD).

MND is a rapidly progressing disease that affects the brain and spinal cord’s ability to communicate with the muscles, causing weakness that worsens over time. FTD is a group of disorders characterized by loss of neurons in the frontal and temporal lobes of the brain, which causes a deterioration in behavior, personality and/or difficulty producing or comprehending language. Both conditions are incurable and eventually lead to death.

After uncovering, for the first time, the mechanism by which pathological TDP-43 accumulates in MND and FTD, researchers at Macquarie University, Sydney, created a genetic therapy to clear the blockages and prevent them from re-forming.