Anya T. Joynt

This study corrects a CFTR splice site variant in airway cells using base editors and nanoparticles, showing increased CFTR expression and functional rescue in multiple cell types.

SORT LNPs enable efficient Cas9-mediated repair of a pathogenic allele in skeletal muscle in a mouse model of LGMDR7.

This study identifies mutations to improve prime editing efficiency in low-dNTP environments by enhancing MMLV-reverse transcriptase and reducing SAMHD1 activity.