Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models

Por Osman, Erkan Y., Miller, Madeline R., Robbins, Kate L., Lombardi, Abby M., Atkinson, Arleigh K., Brehm, Amanda J., Lorson, Christian L.
Publicado em 2014

Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent

Por Shababi, Monir, Feng, Zhihua, Villalon, Eric, Sibigtroth, Christine M, Osman, Erkan Y, Miller, Madeline R, Williams-Simon, Patricka A, Lombardi, Abby, Sass, Thalia H, Atkinson, Arleigh K, Garcia, Michael L, Ko, Chien-Ping, Lorson, Christian L
Publicado no Mol Ther (2016)