A team of scientists has recently discovered a new biochemical process that occurs in the brain cells of patients with Alzheimer’s disease motor neuron disease (MND). Although still in its early stages, this research could in the future lead to the development of treatments capable of block the progress of the disease.
MND (which also afflicted astrophysicist Stephen Hawking) is a terrible disease characterized by rapid neuronal degeneration leading to loss of muscle control.
The new discovery revolves around the biochemical changes affecting the protein called TDP-43, present in all the cells of our body but especially in the brain; these cells – responsible for muscle movements – are precisely i motor neurons.
The researchers used the now well-known method of gene editing CRISPR to be able to observe for the first time the behavior of the TDP-43 protein inside live cells, in genetically modified mice affected by MND. This allowed them to study extensively the protein malfunction.
“We have found that diseased versions of TDP-43 are capable of harming healthy versions as well, which can create a cycle of protein dysfunction and degeneration over time.” said Dr. Adam Walker.
“We also found that the biochemical pathways responsible for neuronal death are activated early, long before the first symptoms appear. To change the course of the disease you need drugs that can prevent this dysfunction of the TDP-43 protein.“
The researchers’ work will now focus onbreaking this cycle of dysfunction – through the administration of drugs in mice – with the hope of obtaining positive results and applying them to humans in the future.
