According to a story from the National Institutes of Health, the innovative new CRISPR gene editing technology is being used in order to identify specific genes that could play a role in controlling the severity of neurological conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig disease. The gene that has determined as causative for these conditions is C9orf72. The study discovered several genes that could worsen the progression of nerve cell death.
C9orf72 has a role in a significant percentage of inherited cases of frontotemporal dementia and amyotrophic lateral sclerosis, being implicated in 25 percent and 40 percent of genetically linked cases respectively. Mutations of this gene produce extra sequences of DNA that, in turn, can release toxic proteins and RNA that kill nerve cells and result in the symptoms experienced in the two conditions.
Using CRISPR, scientists were able to able each gene and tested human leukemia cells by exposing them to the toxic proteins, known as DPRs. The genes that either allowed the cells to survive longer or killed them more rapidly were noted carefully. The researchers also noted a possible role of genes responsible for moving molecules into and outside of cells and genes that controlled the assembly of proteins moving through the endoplasmic reticulum.
One of the genes that was implicated in the endoplasmic reticulum was called TMX2. Activity of the TMX2 gene appeared to have a negative effect on outcomes. When the TMX2 gene was deactivated, the body produced proteins that are associated with survivability against the toxic DPR proteins. This was tested using neurons created from patients’ skin cells.
At this juncture, CRISPR is the most advanced gene editing module in the world, and the study could not have been conducted without it. In the past, a study like this would have taken months just to find the potential genes for the researchers to test, and the testing could not have been done on the human genome. CRISPR allowed the discovery of relevant genes to take only a couple of weeks.
Clearly, CRISPR has incredible potential to help with similar neurological conditions by identifying the genes that play a role. Therefore, developing effective treatments can begin at a much more rapid pace.
