And that basic science could someday lead to new therapy for the debilitating disease, which cripples people by removing the brain's ability to communicate with their muscles, eventually leading to paralysis and early death.
"It's a story of fundamental research about what happens normally in the body's cells and what changes in the context of ALS," said Jade-Emmanuelle Deshaies, a research associate in neurosciences at the UdeM Hospital Research Centre (CRCHUM) and lead author of the joint Canada-Israel study, published online today in the journal Brain.
Studies - Rise - Treatments - People - ALS
"While studies such as this do not immediately give rise to new treatments for people living with ALS, they do deepen our understanding of the disease. ALS is very complicated; many cellular functions get mis-regulated. This type of work provides important information for future drug targets and the development of biomarkers aimed at detecting the disease more rapidly and following its progression."
The research began eight years ago when Deshaies and her supervisor, associate professor of neurosciences Christine Vande Velde, started investigating what happens to various molecules when TDP-43, a protein that binds the 'messengers' in the cell known collectively as RNA and that is central to ALS pathology, is removed from the nucleus.
Focus - Types - RNA - Proteins - Players
"Our specific focus was on other types of RNA binding proteins that could be interesting players in the disease," Deshaies recalled. "One of these, hnRNP A1, caught our eye. In particular because there was a second form that is rarely mentioned in the literature."
But first, a bit of basic science.
Biology - Genes - RNA - RNA - Proteins
In molecular biology, genes encode RNA and the RNA then gets translated into proteins, the workhorses of cells. There are many different versions of RNA, each encoding many different versions of a protein. TDP-43, for one, binds RNA and can change how it is spliced -- in a sequence of ABCD, for example, or of...
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