Exciting Breakthrough in Dementia Research at Macquarie University

The image depicts a scientist working with digital genetic data. In the foreground, a graphical representation of a DNA double helix is superimposed over the scene, indicating the genetic focus of the work. The scientist, wearing a lab coat, is engaged with a tablet, likely analyzing data or controlling laboratory equipment. The overall blue tone of the image conveys a high-tech, clinical atmosphere.

Neuroscientists at Macquarie University in Australia have made a groundbreaking discovery in the fight against neurodegenerative diseases. They have developed a single-dose genetic medicine called CTx1000, which has shown promising results in halting the progression of both ALS (amyotrophic lateral sclerosis) and frontotemporal dementia (FTD) in mice. This innovative treatment targets the pathological build-ups of the protein TDP-43, which are associated with these diseases, and may even offer the potential to reverse some of their effects.

The research team, led by Professor Lars Ittner, is hopeful that CTx1000 will begin human clinical trials within the next two years. The treatment’s ability to specifically target pathological TDP-43 while allowing healthy proteins to function normally makes it an incredibly safe option. This breakthrough could not only provide a much-needed treatment for ALS and FTD but also holds promise for more common forms of dementia, such as Alzheimer’s disease. With the formation of Celosia Therapeutics, a spin-out company from Macquarie University, efforts are underway to bring this groundbreaking work from the lab to patients.

Read “New Genetic Therapy Could Be a Gamechanger for MND and Frontotemporal Dementia.” Lighthouse, Macquarie University, February 2024

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