In a new application of gene-editing technology, researchers at the Stanford University School of Medicine have gleaned insights into the genetic underpinnings of amyotrophic lateral sclerosis, a neurodegenerative disease that’s notoriously tricky to parse.
In a new application of gene-editing technology, researchers at the Stanford University School of Medicine have gleaned insights into the genetic underpinnings of amyotrophic lateral sclerosis, a neurodegenerative disease that’s notoriously tricky to parse.
The team’s findings are a step toward demystifying how the disease progresses and could even help lay the groundwork for new therapeutic targets.
ALS, also known as Lou Gehrig’s disease, erodes muscle function and impairs the brain’s ability to communicate with the body, making simple voluntary muscle movements — such as brushing your teeth, talking or even breathing — exceedingly difficult and, eventually, impossible. ALS falls into a category of neurodegenerative diseases that all share a common “signature” — abnormal protein clumps that build up in the brain.
In ALS, these protein clumps, or aggregates, are thought to be fatally toxic to neurons, ultimately leading to the devastating physical symptoms of the disease. But the process of the cells’ demise is still largely a black box.
Read more at: Stanford University
Photo: Aaron Gitler and his colleagues used genome editing to help identify the genes that play a part in amyotrophic lateral sclerosis. (CREDIT: Paul Sakuma)
