Charles P. Emerson

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.

This study shows SMCHD1 activates cell cycle genes in human myoblasts, promoting proliferation independently of DUX4, and identifies LAP2 as a key target gene.

This study develops a humanized mouse model to investigate the role of innate immunity in FSHD muscle pathology and identifies immune cell accumulation and complement gene expression in FSHD muscle xenografts.