Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy
| Autor: | Osman, Erkan Y, Washington, Charles W, III, Kaifer, Kevin A, Mazzasette, Chiara, Patitucci, Teresa N, Florea, Kyra M, Simon, Madeline E, Ko, Chien-Ping, Ebert, Allison D, Lorson, Christian L |
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| Zdroj: | In Molecular Therapy September 2016 24(9):1592-1601 |
| Databáze: | ScienceDirect |
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