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filingDate 2015-02-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b74b035af6c225d270ae18a68ca85e89
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publicationDate 2016-12-08-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2016355811-A1
titleOfInvention Antisense Oligonucleotides for Treatment of Spinal Muscular Atrophy
abstract Various aspects of the present invention are directed to compounds targeted to various regions of the survival motor neuron 2 (SMN2) gene. Such compounds may be used to increase incorporation of exon 7 in processed transcripts of SMN2. Such compounds may therefore be useful in increasing the amount of full-length SMN protein produced by the SMN2 gene. As such, these compounds may provide a therapeutic approach for treatment of spinal muscular atrophy (SMA).
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