Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy
Osman, Erkan Y, Washington, Charles W, Kaifer, Kevin A, Mazzasette, Chiara, Patitucci, Teresa N, Florea, Kyra M, Simon, Madeline E, Ko, Chien-Ping, Ebert, Allison D, Lorson, Christian LLangue:
english
Journal:
Molecular Therapy
DOI:
10.1038/mt.2016.145
Date:
July, 2016
Fichier:
PDF, 5.06 MB
english, 2016