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

GSE269943 Mus musculus Expression profiling by high throughput sequencing 4 samples Submitted 2025/12/12 Platform GPL13112
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 intramuscularly into a DMD mouse model.
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Direct links to NCBI, no account and no request form: the whole study as GSE269943_RAW.tar, processed values as the series matrix, the supplementary file directory, and per-sample supplementary files for any of the 4 samples. Raw sequencing reads are also available from ENA.

Also filed as BioProject PRJNA1124150 and SRA study SRP514071. Searching any of these in the dataset finder brings you back here.

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