A cure for prions?
2025-02-05 18:21:33.448972+01 by Dan Lyke 0 comments
Editor’s summary
Prion diseases are devastating neurodegenerative disorders that are invariably fatal, but removal of the prion protein from neurons can protect against disease progression. Neumann et al. developed a compact epigenetic silencer called CHARM that could efficiently shut off the prion gene throughout the mouse brain when delivered systemically by a viral vector without changing the underlying DNA sequence (see the Perspective by Whittaker and Musunuru). The epigenetic editor can also be programmed to turn itself off after silencing its target, thus limiting potential adverse effects from long-term expression. CHARM represents a therapeutic modality that could be applied to a range of other diseases caused by the toxic buildup of unwanted proteins. —Di Jiang
DOI: 10.1126/science.ado7082
As rahaeli @rahaeli.bsky.social said:
Holy SHIT. Okay, this needs an "in mice" caveat, but the tactic they took means it's less of a caveat than usual and also HOLY FUCKING SHIT