The TDP-43 protein is considered a key factor in neurodegeneration, so scientists from the University of California, San Diego sought to develop a treatment that would target it. The new therapy delivers the SynCav1 gene to neurons, which increases production of the neuroprotective protein caveolin-1, which is involved in maintaining signaling pathways for normal nerve cell function.
Unlike other approaches that focus solely on removing toxic proteins, the new strategy can increase neurons' resistance to damage.
"Many treatments for neurodegenerative diseases focus on removing toxic proteins, but neurons simultaneously lose their ability to cope with stress. Strengthening the resilience of neurons themselves could be an effective therapeutic strategy even in the presence of existing accumulation of toxic proteins," said study author Brian Head.
In preclinical experiments on mouse models, the therapy successfully penetrated the protective blood-brain barrier and increased caveolin-1 levels in neurons in the brain and spinal cord. In these animals, the treatment preserved memory and learning function and reduced TDP-43 levels. Furthermore, the scientists noted benefits for mitochondrial function and overall neuronal structure.
"We discovered that TDP-43 not only accumulates in abnormal subcellular structures but also disrupts communication processes essential to neurons," the scientists commented. In other words, the results offer new insights into the mechanisms of neurodegeneration.
The treatment has potential for treating various neurodegenerative diseases. Further research will focus on its long-term effects on cognitive function.
Scientists have recently discovered a new cause of neurodegeneration.
