According to a story from brightsurf.com, a team from the Washington University School of Medicine, based in St. Louis, has succesfully developed a gene therapy that appears to prevent damage to the peripheral nervous system in mice. Peripheral neuropathy, or peripheral nerve damage, is a major component of many serious neurdegenerative diseases, such as Parkinson’s disease and amyotrophic lateral sclerosis.
The Process of Neurodegeneration
This gene therapy could potentially play a major role in slowing or even preventing the progression of diseases of this type. The portion of the neuron called the axon is responsible for sending electrical messages that control sensation and movements. Any sort of damage to these axons ultimately triggers a process that causes them to destroy themselves.
Peripheral neuropathy is also a common after effect of cancer treatment with chemotherapy. At this juncture, many neurodegenerative diseases lack therapies that can stop the process of nerve fiber breakdown. In a test of the gene therapy in mice, it was capable of stopping the mice from experiencing neurodegeneration. These findings are undeniably significant and the next step will be to test this gene therapy in human patients to see if these encouraging results can be effectively replicated.
SARM1: The Point of Intervention
The genetic process that triggers the destruction of axons, regardless of the cause, involves a protein called SARM1. When nerves are undamaged, this protein is not expressed. Earlier research confirmed that the SARM1 protein was responsible for triggering the self-destruct process in damaged axons.
In effect, this gene therapy works in the opposite manner of most gene therapies that you hear about. Typically, gene therapy is used to treat a genetic disorder by using a viral vector delivery system to implant a corrected, non-mutated version of the affected gene. In this case, a mutated version of the SARM1 gene, which does not trigger axon destruction, is implanted to replace the non-mutated version. This mutated form can also block the activity of regular SARM1 proteins.
The big takeaway though is the fact that this therapy could theoretically be useful in a large number of neurodegenerative diseases. If human trials go favorably, the situations for patients with devastating diseases like amyotrophic lateral sclerosis or Parkinson’s could be transformed for the better.
You can view the abstract for this study here at the Journal of Experimental Medicine.
