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filingDate 2021-02-02-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_98715253161b5b3ee6de9ab548a0d1c9
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publicationDate 2021-06-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber JP-2021088563-A
titleOfInvention Deletion of dystrophin gene exon with modified nuclease
abstract PROBLEM TO BE SOLVED: To provide a medicine for use in the treatment of Duchenne muscular dystrophy. SOLUTION: (i) RNA or DNA encoding a first nuclease or a first nuclease that cleaves a first recognition sequence; and (ii) RNA encoding a second nuclease or a second nuclease that cleaves a second recognition sequence. Alternatively, comprising a combination of DNA; the drug is administered to contact the DNA of the muscle cell of the mammalian subject with the first and second nucleases; the first recognition sequence is the first exon of the dystrophin gene of the muscle cell. The second recognition sequence is in the intron 5'upstream; the second recognition sequence is in the 3'downstream intron of the first exon of the dystrophin gene of muscle cells; the first and second recognition sequences are the first and second recognition sequences. When cleaved by a second nuclease, it is selected to have a complementary overhang; the first exon and at least one second exon are removed from the dystrophin gene in muscle cells. [Selection diagram] None
priorityDate 2014-03-12-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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