http://rdf.ncbi.nlm.nih.gov/pubchem/patent/JP-2019059769-A

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filingDate 2018-12-12-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_46e80b0e165c65d5b0ea9b1c8d6afe27
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publicationDate 2019-04-18-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber JP-2019059769-A
titleOfInvention Induced exon inclusion in spinal muscular atrophy
abstract The object is to provide induced exon inclusion in spinal muscular atrophy. The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment of spinal muscular atrophy (SMA). More specifically, the present invention relates to the induction of exon 7 inclusion to restore the level of survival motor neuron (SMN) protein encoded by the survival motor neuron (SMN) gene. Also within the scope of the invention is a method of treating spinal muscular atrophy (SMA) in a patient. Such a method comprises administering to the patient an antisense oligonucleotide comprising a nucleotide sequence of sufficient length and complementarity to specifically hybridize to a region within the SMN2 gene, and containing exon 7 within the cell. Including the step of enhancing the levels of SMN2 mRNA to exon deleted SMN2 mRNA. 【Selection chart】 None
priorityDate 2011-11-30-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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