Study shows potential for Lipid Nanoparticles to successfully deliver dystrophin gene to muscles

One of the biggest challenges in gene therapy for DMD is the delivery of the dystrophin protein to the affected muscles.

Adeno-associated viruses  are used to deliver gene therapy. However, due to their small size and the very large size of the dystrophin gene, only a shortened version of the dystrophin gene (mycrodystrophin) can be delivered through them.

In 2021, Duchenne UK funded a preclinical study that has been successful in producing and developing Lipid Nanoparticles (LNPs) linked to a myomerger protein that could carry larger versions of dystrophin. The research team are committed to continue their investigations of LNPs linked to myomergers for potential future success in treating Duchenne muscular dystrophy.

Published on 27 February 2024

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