Sonia Vallabh, MIT and Havard Senior Group Leader, and her team were researching a type of prion disease known as fatal familial insomnia. Vallabh began her research on this prion disease after finding out she carries the disease-causing genes and that there are no current treatments, preventatives, or therapies for the fatal disease. Knowing that she would develop fatal familial insomnia in the years to come, Vallabh knew that she was racing time to develop her own treatment.
Less than two years after starting, Vallabh, Weissman, and other contributors developed gene-silencing tools that are now referred to as, "CHARMs", that can turn off the specific necessary genes to prevent prion disease proteins from being created. The CHARMs can turn off these specific genes via epigenetic editing where chemical tags are added to DNA to silence specific gene sequences. The benefits of CHARM are that the genes are kept intact so no DNA is destroyed and epigenetic editing will keep the genes suppressed with only one treatment instead of needing to constantly take pills.
The team is now working on taking these "molecular tools" and making them more versatile in their ability to provide therapy and fight against other neurodegenerative diseases. Although human trials have not taken place yet, the concept and technology have been developed and are revolutionary to the battle against neurodegenerative diseases. It is very likely that soon human trials will begin using CHARMs as a therapy and preventative measure against prion diseases. The speed at which this technology has been developed is impressive; however, passing human trials has still yet to come, which could display potential side effects that could render CHARMs ineffective.
https://news.mit.edu/2024/charmed-collaboration-creates-therapy-candidate-fatal-prion-diseases-0627
https://rarediseases.info.nih.gov/diseases/6429/fatal-familial-insomnia
