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

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by the loss of Survival Motor Neuron-1 (SMN1). In all SMA patients, a nearly identical copy gene called SMN2 is present, which produces low levels of functional protein owing to an alternative splicing event. To prevent exon-skippin...

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Main Authors: Osman, Erkan Y., Miller, Madeline R., Robbins, Kate L., Lombardi, Abby M., Atkinson, Arleigh K., Brehm, Amanda J., Lorson, Christian L.
Formato: Artigo
Idioma:Inglês
Publicado: Oxford University Press 2014
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Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4140465/
https://ncbi.nlm.nih.gov/pubmed/24781211
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu198
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