WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration
Wood M., Zhang J., Bowman K., Butler D., Rinaldi C., McClorey G., Frank-Kamenetsky M., Iwamoto N., Kothari N., Lu G., Mathieu S., Meena M., Menon S., Shimizu M., Standley S., Yang H., Zhong A., Francis C., Vargeese C.