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Home » Efficient editing in skeletal muscle of dystrophic mice with SORT LNPs encapsulating Cas9 mRNA
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Neurology/psychiatric

Efficient editing in skeletal muscle of dystrophic mice with SORT LNPs encapsulating Cas9 mRNA

March 25, 2026
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Gene editing holds promise for treating neuromuscular disorders such as limb-girdle muscular dystrophy, but its clinical translation remains challenging due to a lack of complementary delivery tools for the extensive network of skeletal muscles in the human body. A team at University of Massachusetts Chan Medical School compared editing outcomes mediated by either Cas9 mRNA and RNP delivery to skeletal muscle via local injection in the context of the previously described selective organ targeting (SORT) lipid nanoparticles (LNPs) platform.
BioWorld Science Genetic/congenital Neurology/psychiatric Gene therapy

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