Feb 19, 2024

New genetic therapy shows promise for motor neuron disease and frontotemporal dementia

Posted by in categories: biotech/medical, genetics, neuroscience

Macquarie University neuroscientists have developed a single-dose genetic medicine that has been proven to halt the progression of both motor neuron disease (MND) and frontotemporal dementia (FTD) in mice—and may even offer the potential to reverse some of the effects of the fatal diseases.

It may also hold opportunities for treating more common forms of dementia, such as Alzheimer’s disease, which is the second most common cause of death in Australia after heart disease.

The new treatment, dubbed CTx1000, targets pathological build-ups of the protein TDP-43 in cells in the brain and spinal cord.

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