Karen L. Kelly

Up-regulation of cholesterol synthesis pathways and limited neurodegeneration in a knock-in Sod1 mutant mouse model of ALS.

This study shows that Sod1G85R knock-in mice exhibit up-regulated cholesterol synthesis pathways and limited neurodegeneration, contrasting with severe TgSOD1G93A mice, suggesting lipid pathway dysregulation in ALS.

Engineering Nme2Cas9 Adenine Base Editors with Improved Activity and Targeting Scope

This paper engineers Nme2Cas9 base editors to improve activity and targeting scope, enabling correction of MECP2 mutations with minimal off-target effects.

Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo

This study demonstrates the use of protecting oligos to enhance crRNA stability and potency for in vivo genome editing via AAV co-delivery in mice.

Adenine Base Editing in vivo with a Single Adeno-Associated Virus Vector

This study develops a compact adenine base editor (Nme2-ABE) that can be delivered via a single AAV vector to efficiently edit mouse genomic loci and revert disease mutations in a tyrosinemia model.