Evaluation of cellular distribution of LNP-CRISPR in the skeletal muscle

Muscle satellite cells are essential for the regenerative ability of skeletal muscles and are an attractive therapeutic target for gene transduction in Duchenne muscular dystrophy (DMD). However, there is currently no efficient delivery technology to transduce satellite cells in vivo. This study showed that a lipid nanoparticle (LNP)-mediated delivery of CRISPR-Cas9 mRNA and gRNA (LNP-CRISPR) induces exon skipping in satellite cells more efficiently when injected intramuscularly into a DMD mouse model. Overall design: Single-cell mRNA sequencing of cells isolated from skeletal muscles in DMD mouse model.