Tetracyclines That Promote SMN2 Exon 7 Splicing as Therapeutics for Spinal Muscular Atrophy

There is at present no cure or effective therapy for spinal muscular atrophy (SMA), a neurodegenerative disease that is the leading genetic cause of infant mortality. SMA usually results from loss of the SMN1 (survival of motor neuron 1) gene, which leads to selective motor neuron degeneration. SMN2...

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Main Authors: Hastings, Michelle L., Berniac, Joel, Liu, Ying Hsiu, Abato, Paul, Jodelka, Francine M., Barthel, Lea, Kumar, Sujatha, Dudley, Caroline, Nelson, Mark, Larson, Kelley, Edmonds, Jason, Bowser, Todd, Draper, Michael, Higgins, Paul, Krainer, Adrian R.
格式: Artigo
語言:Inglês
出版: 2009
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2818805/
https://ncbi.nlm.nih.gov/pubmed/20161659
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3000208
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