Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy

Loss of Survival Motor Neuron-1 (SMN1) causes Spinal Muscular Atrophy, a devastating neurodegenerative disease. SMN2 is a nearly identical copy gene; however SMN2 cannot prevent disease development in the absence of SMN1 since the majority of SMN2-derived transcripts are alternatively spliced, encod...

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Bibliographic Details
Published in:Mol Ther
Main Authors: 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 L
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2016
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5113110/
https://ncbi.nlm.nih.gov/pubmed/27401142
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.145
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