METHODS FOR TREATING SPINAL MUSCULAR ATROPHY
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United States of America Patent
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Issued Date -
May 15, 2014
app pub date -
Dec 12, 2013
filing date -
May 27, 2008
priority date (Note) -
Abandoned
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Abstract
The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
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