Neurodegenerative Diseases Could Be Treated By Activating A Single Protein
The key to treating some neurodegenerative disorders might be a single protein. Researchers from Gladstone revealed that regulating a protein level using a single protein could lead to possible treatments of neurodegenerative diseases like Alzheimer's, Huntington's, Parkinson's, and and amyotrophic lateral sclerosis (ALS).
Neurodegenerative disorders are characterized by progressive loss of structure and function or death of neurons in the brain. Such diseases are currently incurable, however, these disorders are rooted from misbehaving proteins in the brain and are linked to protein misfolding, a process where proteins accumulate neurons and kills the cells.
In a study published in the Proceedings of the National Academy of Sciences, researchers tested the possibility of restoring the levels of proteins which triggers neurodegenerative disorders by using a single key protein -- the Nrf2 protein.
The researchers used two models Parkinson's disease (humans and rat neurons) with mutations in the proteins LRRK2 and α-synuclein and activated the Nrf2 to test its effect. They took thousands of images within a week to observe its developments. Results reveal that Nrf2 removed the LRRK2 and α-synuclein in the cells.
"Overexpressing Nrf2 in cellular models of Parkinson's disease resulted in a huge effect. In fact, it protects cells against the disease better than anything else we've found." Gaia Skibinski, PhD, a staff research scientist at Gladstone.
The researchers added that they have also tested Nrf2 with models of other neurodegenerative diseases like Huntington's disease and ALS and it is the safest treatment they found. "I am very enthusiastic about this strategy for treating neurodegenerative diseases," senior Gladstone researcher, Finkbeiner said.
He added that they aim at getting a better understanding of the role of Nrf2 in regulating protein levels based on the magnitude and the breadth of its the effect. Nrf2 is involved in numerous cellular processes so it is difficult to target by using only medications. But, the researchers are now focusing on investigating its downstream effects, in hope of discovering a pathway to interact with Nrf2 that would lead to the discovery of treatments for neurodegenerative diseases.
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