Vallabh and her husband, Eric Minikel, are racing against time to find treatments for fatal familial insomnia, a neurodegenerative prion disease that Vallabh will likely develop. Collaborating with Jonathan Weissman, their CHARM technology epigenetically edits genes to prevent disease at its source, marking a new approach to drug development. The team successfully engineered CHARM to target the prion protein gene and efficiently deliver it to the brain, paving the way for clinical trials. With promising results in mice, CHARM could revolutionize gene therapy for patients with prion diseases and beyond, offering hope for effective treatment in the near future.
https://news.mit.edu/2024/charmed-collaboration-creates-therapy-candidate-fatal-prion-diseases-0627