Identification of potent, muscle-targeting investigational stereo-pure oligonucleotides for exon 53 DMD therapy
Durbin A., Shivalila C., Kothari N., Rinaldi C., McClorey G., Antonijevic I., Byrne M., II DJ., Guo L., Iwamoto N., Liu F., Longo K., Lu G., Nadella P., Purcell-Estabrook E., Standley S., Zhong Z., Zhang J., Wood M., Vargeese C.