Nathan Appling

The paper establishes a method for in utero genome editing in fetal mouse cortex at midgestation, demonstrating efficient editing with reduced off-target effects.

The paper solves cryo-EM structures of AAV capsids and their complexes with AAVR to design variants with reduced liver targeting and altered brain cell tropism.

The paper identifies transcriptional crosstalk between enhancers and promoters delivered by AAVs, showing concatemer formation enables targeted delivery of large genetic cargo for cell-type-specific gene editing.

The paper introduces a programmable self-assembling protein system to model intracellular protein aggregation in neurodegenerative diseases and reveals disease-specific aggregate-organelle interactions.