Qi Long Lu

TL;DR: It is demonstrated for the first time that regular 1-year administration of peptide-conjugated phosphorodiamidate morpholino can be safely applied to achieve significant therapeutic effects in an animal model and is associated with greatly reduced serum creatine kinase levels, near-normal histology, and functional improvement of skeletal muscle.

TL;DR: It is shown that oral administration of ribitol increases dystropglycan glycosylation and ameliorates symptoms of muscular dystrophy in FKRP-deficient mouse models.

TL;DR: The results indicate that PMO could be used safely as effective drugs for long-term systemic treatment of DMD and suggest that low levels of dystrophin induction may be able to provide detectable benefit to cardiac muscle with limited myopathy.

TL;DR: FKRP gene therapy ameliorated dystrophic pathology and cardiomyopathy such as muscle degeneration, fibrosis, and myofiber membrane leakage, resulting in restoration of muscle and heart contractile functions.