At an American Society of Gene & Cell Therapy meeting, Sonia Vallabh presented preclinical data on AAV‑mediated epigenetic editing designed to silence the prion protein gene as a potential treatment for prion diseases. Vallabh argued the approach could remove the pathogenic protein driving rapidly progressive neurodegeneration and described both technical results and personal motivation. The strategy uses targeted epigenetic modifiers delivered by adeno‑associated virus to reduce or eliminate expression of the normal prion protein, thereby preventing the template‑driven misfolding cascade. Presenters highlighted efficacy signals in model systems and discussed safety concerns around durable gene modulation in the CNS. If translatable, epigenetic silencing could alter the prognosis for prion disorders, a group of fatal diseases with no current disease‑modifying options. The work also exemplifies growing interest in locus‑specific epigenetic therapies for neurodegeneration, though clinical translation will require robust CNS delivery and long‑term safety data.
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