GEO series
Evaluation of cellular distribution of intravenously injected LNP in the skeletal muscle
GSE296617
Mus musculus
Expression profiling by high throughput sequencing
10 samples
2025/12/12
GPL34290
Summary
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 into a DMD mouse model.
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