Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model

por Angelbello, Alicia J., Rzuczek, Suzanne G., Mckee, Kendra K., Chen, Jonathan L., Olafson, Hailey, Cameron, Michael D., Moss, Walter N., Wang, Eric T., Disney, Matthew D.
Publicado 2019

A blood–brain penetrant RNA-targeted small molecule triggers elimination of r(G(4)C(2))(exp) in c9ALS/FTD via the nuclear RNA exosome

por Bush, Jessica A., Meyer, Samantha M., Fuerst, Rita, Tong, Yuquan, Li, Yue, Benhamou, Raphael I., Aikawa, Haruo, Zanon, Patrick R. A., Gibaut, Quentin M. R., Angelbello, Alicia J., Gendron, Tania F., Zhang, Yong-Jie, Petrucelli, Leonard, Heick Jensen, Torben, Childs-Disney, Jessica L., Disney, Matthew D.
Publicado 2022