I actually looked it up as I have always been interested in all things genetic. They are using a viral vector as the cellular delivery system.
Basically infecting the entire body with a functional gene to counteract the failings of the incomplete damaged gene.
I don’t think the nanoparticles have the right vectoring characteristics for such a systemic whole body treatment.
Gene therapy is a completely different science than vax development.
Yes, it is a virus vector. It should only be expressed in muscle cells. It’s the gene, dystrophin, that is mutated in this disease.
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DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. In the absence of dystrophin, muscle cells deteriorate. Fordadistrogene movaparvovec is an investigational recombinant adeno-associated virus serotype 9 (AAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The AAV9 capsid was chosen as the delivery mechanism because of its potential to target muscle tissue.
After further reading they didn’t use the lipid nanoparticles with this. Seems that is used primarily for RNA delivery not DNA delivery. The DNA is much more stable and probably wouldn’t require the protective lipid coat to maintain its integrity like RNA would.