Methods of screening for compounds for treating spinal muscular atrophy using SMN mRNA translation regulation
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United States of America Patent
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Oct 9, 2012
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Jun 23, 2008
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Jun 22, 2007
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Abstract
The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of spinal muscular atrophy (SMA), in which said compounds up-regulate the post-transcriptional expression of SMN1 or SMN2.
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