Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_ac409458365ff9d5dc07bf78b39ea951 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N7-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P43-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4708 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-54 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-465 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-04 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-22 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-46 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P21-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P43-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
filingDate |
2021-02-02-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_98715253161b5b3ee6de9ab548a0d1c9 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_9a3a32b9a5f1a56ff3eaab4472636822 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_88dc4ba2ae4d722b755c65ae7da5bae3 |
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> |
type |
http://data.epo.org/linked-data/def/patent/Publication |