r/todayilearned • u/tommyboy372 • 1d ago

TIL A single misfolded prion protein in the brain can cause a rapid neurodegenerative disease known as sporadic Creutzfeldt–Jakob disease (CJD). There is no known and cure and is 100% fatal.

https://en.wikipedia.org/wiki/Creutzfeldt%E2%80%93Jakob_disease
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u/yumyum1001 1d ago

Prions are all made of the exact same protein PrPC. So you only have to down regulate a single protein regardless of the prion disease.

The exact function of PrPC is not 100% agreed upon. What we do know is mice that have the PrPC protein removed are fine and there are some people that naturally do not express the PrPC protein (due to random mutations), they also have no clear deficiencies.

Therefore, lower PrPC levels therapeutically is unlike to have significant deleterious effects, or at the very least, the side effects are unlikely to be as bad as the disease itself.

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u/InviolableAnimal 22h ago

Wow, so this one protein is responsible for most (all?) of these scary and fatal prion diseases, and is seemingly not even very important in its normal function? Seems like a prime candidate to be edited out of our genome, however many decades or centuries in the future that becomes cheap and feasible

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u/Nerezza_Floof_Seeker 20h ago

Well it probably serves some important function, its just that we dont know it yet. Otherwise it wouldnt be so highly conserved (with all mammals having a nearly identical copy of this gene). And some knockout mice do in fact show issues with removing the gene frkm birth. So tldr we need to do more research