Key facts
- Base editing extended lifespan by about 50 percent in mice infected with human prion protein and reduced brain PrP by up to 60 percent; led by Vallabh, Minikel and David Liu; AAV delivery built on Ben Deverman lab vector engineering; Liu approached the team in 2018.
- Vallabh (Broad) and Weissman (MIT and Whitehead) built CHARM by replacing Cas9 with a zinc finger protein; AAV-delivered CHARM turned off the prion protein gene across mouse brains and was modified to turn itself off. Paper: Neumann EN, Bertozzi TM, ... Deverman BE, Minikel EV, Vallabh SM, Weissman JS (Science 2024).
- His lab's brain-wide gene delivery work, published in Science on 17 May 2024, was described as bringing CHARM closer to clinical trials.
- He is a co-author of the CHARM paper in Science (28 June 2024) with Weissman, Vallabh and Minikel.
- He is listed among the authors of the 2025 Nature Medicine base editing paper led by David Liu with Vallabh and Minikel.
- CHARM delivery uses AAV carrying a compact zinc finger epigenetic editor to silence the prion gene across the mouse brain.
- Engineered vectors are the main translation gap because a capsid that works in human brain at tolerable doses is still needed.
Timeline
- 2024Delivery vector paper published.
- 2024CHARM paper published.
- 2025Base editing paper online.
Institutions
Projects and trials
Works with
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Last checked 16 October 2026