The new drug candidate, developed by Broad Institute and University of Massachusetts Chan Medical School scientists, is a small interfering RNA (siRNA) that binds and snips RNA molecules encoding the prion protein, reducing the amount of the disease-causing protein in the brain. Previous research in animals has shown that lowering prion protein levels can delay onset and slow down the disease.
I just watched a video the other day about this. Apparently we are not aware what the prion protein does in the brain… So it would not be wise to destroy all of them. So they made it such that the population of prion proteins reduced to a certain percentage.
It’s also cool that they came up with a solution to bypass a lot of flushing mechanisms in the blood-brain barrier and the CSF (Cerebro-Spinal-Fluid) when injecting the RNA.
Fascinating
I just watched a video the other day about this. Apparently we are not aware what the prion protein does in the brain… So it would not be wise to destroy all of them. So they made it such that the population of prion proteins reduced to a certain percentage.
It’s also cool that they came up with a solution to bypass a lot of flushing mechanisms in the blood-brain barrier and the CSF (Cerebro-Spinal-Fluid) when injecting the RNA.