Scientists have discovered a new way to stop neuronal cell death in the most common motor neuron disease and frontotemporal dementia. This could change how these neurodegenerative conditions are treated. Moreover, because it can be administered orally, it may even be packaged into a nasal spray.
Groundbreaking research by the University of Sheffield’s Institute for Translational Neuroscience (SITraN) focuses on using peptides with cell-permeable modules to block pathways of mutated repetitive RNA molecules . Once these rogue molecules translocate from the cell’s nucleus to the outer cytoplasm, they are used to generate toxic repeat proteins that ultimately kill neurons, leading to motor neuron disease (MND) and frontotemporal dementia (FTD). ) contributes to the degeneration of
Guillaume Hautbergue, professor of translational RNA biology at the University of Sheffield, who led the study, said:
Previously, researchers had found that movement of the problematic RNA copied from the C90RF72 gene (the most frequent cause of MND and FTD) was due to excessive stickiness of the SRSF1 cellular transporter. Their new peptide, composed of small assembled chains of amino acids, penetrates cells and he attaches to the SRSF1 transporter, blocking its trafficking.
In clinical studies with fruit flies, scientists have even observed that there is an improvement in neuronal function when peptides seal this cellular pathway.
“This means that the peptides can effectively block the progression of neurodegenerative conditions and also help restore the function of affected neurons,” said Hautbergue.
The researchers also say the peptide can be administered to MND and FTD patients orally or via a nasal spray developed to enter the brain.
Both FTD and MND, the most common causes of early-onset dementia, are debilitating and fatal conditions with few treatments to halt their rapid degeneration. This work opens the door to human trials and the development of non-invasive, targeted and effective medical interventions in the not too distant future.
“MND and FTD are devastating diseases for which there is currently no cure,” said Hautbergue. “This is a promising alternative to traditional small molecule drugs, which are often limited by poor blood-brain barrier permeability.”
This research Science Translational Medicine.
Source: University of Sheffield
