Jonathan S. Weissman

Key facts

• Led the Whitehead group that built CHARM: a zinc finger protein replaces Cas9 and recruits the cell's own DNMT3A to methylate and silence PRNP.
• Co-corresponding author of the Science paper (28 June 2024) with Sonia Vallabh; co-first authors Edwin Neumann and Tessa Bertozzi; collaborators include Ben Deverman and Eric Minikel.
• A single intravenous AAV dose eliminated more than 80 percent of brain PrP in mice, with a self-silencing feature added for safety.
• The brain-wide AAV capsid enabling delivery came from Deverman's lab (Science, 17 May 2024).
• The paper describes CHARM as enzyme-free and compatible with several DNA-binding tools, with up to three targeting elements per AAV.

Timeline

1. 2024Deverman lab delivery vector published.
2. 2024CHARM published in Science.
3. 2024NIH summarises the CHARM paper.