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filingDate 2017-12-29-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2018-07-05-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2018122610-A1
titleOfInvention Exon skipping by peptide nucleic acid derivatives
abstract A peptide nucleic acid derivative of Formula (I) is provided to tightly bind to a splice site withiun a pre-mRNA in a sequence specific manner. Given with excellent cell membrane permeability and strong affinity for RNA, the said peptide nucleic acid derivative induces exon skipping in cells treated with the peptide nucleic acid at sub-femtomolar concentration as "naked" oligonucleotide. The said compound shows therapeutic activity in subjects upon systemic administration even at 1 μg/Kg or less, and therefore is useful to treat a disease or symptom at affordable treatment cost.
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http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO13077
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP00564
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP04248
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID3355084
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02040
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7T044
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02039
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA4WIW2
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO13163
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ64467
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02049
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP19646
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ9Z518
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO13078
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID5460005
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID419527287
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID36054
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID6013
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02048
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ28932
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ865X2
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02052
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7M3C1
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP19314
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO13164
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID419485131
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP25809
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02051
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226433313
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP33180
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02028
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID6436605
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID100136352
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226433312
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID280979
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02026
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID85344
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP19315
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID14462484
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO08782
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID105061856
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP68080
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02030
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7LZM1
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID43580
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02029
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ6M0E6
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP68082
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02032
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA3CYG8
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7LZM3
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP68081
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02031
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7LZM2
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02035
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID744399
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7LZM5
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226405626
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO09232
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID105097953
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCCAI43080
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02033
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID374193
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7LZM4
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID6727838
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID1174
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP86873
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02070
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226396964
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02066
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP19885
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02073
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID29560
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ6P8J7
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP35048
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02072
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA6P7L6
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP35034
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02076
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID5133
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO14556
http://rdf.ncbi.nlm.nih.gov/pubchem/taxonomy/TAXID432487
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02075
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID23501187
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226399646
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID172198
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ10733
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID161166
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02085
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ03459
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02084
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ3ZBP1
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID6650919
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02054
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226399605
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ29416
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ8I1F6
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ07885
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP45377
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID945112
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02053
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP13557
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID43826
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP26460
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02058
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID134437690
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP02055
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID419011
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP19799
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID419528439

Total number of triples: 1081.