OPMD on track

From a scientific journal published two days ago.

https://www.sciencedirect.com/science/article/pii/S2162253119300332

"'Gene therapy provides a treatment option for OPMD. A recent study of intramuscular adeno-associated virus (AAV)-mediated gene therapy in an OPMD mouse model showed that silencing the mutated expPABPN1 using small hairpin RNAs (shRNAs) while overexpressing a human codon-optimized normal PABPN1 resistant to the degradation by the shRNAs substantially reduced the amount of insoluble aggregates, decreased muscle fibrosis, reverted muscle strength to the level of healthy muscles, and normalized the muscle transcriptome.20 Based on this evidence, a pre-clinical study is ongoing in sheep, and a new clinical trial of gene therapy for OPMD patients will be initiated in 2019 by Axovant using the same AAV-mediated gene therapy approach"'