Mechanism of uptake of Morpholino oligos in dystrophic (DMD) muscle

This work elucidates the mechanism of uptake of Morpholino oligos in dystrophic (DMD) muscle, including roles for muscle satellite cells and macrophages.

Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.

Novak JS, Hogarth MW, Boehler JF, Nearing M, Vila MC, Heredia R, Fiorillo AA, Zhang A, Hathout Y, Hoffman EP, Jaiswal JK, Nagaraju K, Cirak S, Partridge TA.

Nat Commun. 2017 Oct 16;8(1):941. doi: 10.1038/s41467-017-00924-7.

https://www.nature.com/articles/s41467-017-00924-7

Mice, C57Bl/10ScSn-mdx/J or C57Bl/10ScSnJ (Bl10 or wild-type) injection

Discussion on other sites:
https://childrensnational.org/news-and-events/childrens-newsroom/2017/ch...
https://www.drugtargetreview.com/news/26773/macrophages-play-critical-ro...
https://musculardystrophynews.com/2017/10/17/study-shows-that-immune-cel...