JP2016528897A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2016528897A5 JP2016528897A5 JP2016534875A JP2016534875A JP2016528897A5 JP 2016528897 A5 JP2016528897 A5 JP 2016528897A5 JP 2016534875 A JP2016534875 A JP 2016534875A JP 2016534875 A JP2016534875 A JP 2016534875A JP 2016528897 A5 JP2016528897 A5 JP 2016528897A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- item
- nucleotides
- complementary
- composition
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 229920000272 Oligonucleotide Polymers 0.000 claims description 172
- 125000003729 nucleotide group Chemical group 0.000 claims description 144
- 239000002773 nucleotide Substances 0.000 claims description 134
- 230000000295 complement Effects 0.000 claims description 100
- 239000000203 mixture Substances 0.000 claims description 37
- 108020004999 Messenger RNA Proteins 0.000 claims description 23
- 229920002106 messenger RNA Polymers 0.000 claims description 23
- 230000001402 polyadenylating Effects 0.000 claims description 16
- 239000005547 deoxyribonucleotide Substances 0.000 claims description 12
- 229920000795 Polyadenylation Polymers 0.000 claims description 8
- 229920001320 Leader sequence (mRNA) Polymers 0.000 claims description 7
- 108020004412 RNA 3' Polyadenylation Signals Proteins 0.000 claims description 6
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 6
- 229920000401 Three prime untranslated region Polymers 0.000 claims description 5
- 239000003937 drug carrier Substances 0.000 claims description 4
- 229920001914 Ribonucleotide Polymers 0.000 claims description 3
- 238000000338 in vitro Methods 0.000 claims description 3
- 239000008194 pharmaceutical composition Substances 0.000 claims description 3
- 239000002336 ribonucleotide Substances 0.000 claims description 3
- 125000002652 ribonucleotide group Chemical group 0.000 claims description 3
- 108020003589 5' Untranslated Regions Proteins 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 claims 1
- 229920000160 (ribonucleotides)n+m Polymers 0.000 description 112
- 230000000087 stabilizing Effects 0.000 description 39
- 102100010392 GCH1 Human genes 0.000 description 15
- 101700086161 GCH1 Proteins 0.000 description 15
- CZPWVGJYEJSRLH-UHFFFAOYSA-N 289-95-2 Chemical compound C1=CN=CN=C1 CZPWVGJYEJSRLH-UHFFFAOYSA-N 0.000 description 8
- UYTPUPDQBNUYGX-UHFFFAOYSA-N Guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 description 8
- 230000035897 transcription Effects 0.000 description 7
- OGHAROSJZRTIOK-KQYNXXCUSA-O 7-Methylguanosine Chemical compound C1=2N=C(N)NC(=O)C=2[N+](C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OGHAROSJZRTIOK-KQYNXXCUSA-O 0.000 description 6
- 101700022675 HBG Proteins 0.000 description 6
- 102100011763 KCNMB3 Human genes 0.000 description 6
- 108060004186 KCNMB3 Proteins 0.000 description 6
- 239000000969 carrier Substances 0.000 description 6
- 230000014509 gene expression Effects 0.000 description 6
- 102000004169 proteins and genes Human genes 0.000 description 5
- 108090000623 proteins and genes Proteins 0.000 description 5
- IQFYYKKMVGJFEH-XLPZGREQSA-N DEOXYTHYMIDINE Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 description 4
- 229950003499 FIBRIN Drugs 0.000 description 4
- BWGVNKXGVNDBDI-UHFFFAOYSA-N Fibrin Chemical compound CNC(=O)CNC(=O)CN BWGVNKXGVNDBDI-UHFFFAOYSA-N 0.000 description 4
- 102000009123 Fibrin Human genes 0.000 description 4
- 108010073385 Fibrin Proteins 0.000 description 4
- 108020005198 Long Noncoding RNA Proteins 0.000 description 4
- 229920003003 Telomeric non-coding RNA Polymers 0.000 description 4
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical class C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 description 4
- 239000002719 pyrimidine nucleotide Substances 0.000 description 4
- 150000003230 pyrimidines Chemical class 0.000 description 4
- 102100017550 ABCA1 Human genes 0.000 description 3
- 102100011470 ABCA4 Human genes 0.000 description 3
- 101700012451 ABCA4 Proteins 0.000 description 3
- 102100006243 ABCB11 Human genes 0.000 description 3
- 102100014002 ABCB4 Human genes 0.000 description 3
- 101700085131 ABCB4 Proteins 0.000 description 3
- 102100002711 ABCG5 Human genes 0.000 description 3
- 102100016981 ABCG8 Human genes 0.000 description 3
- 101710003225 ADIPOQ Proteins 0.000 description 3
- 102100000322 ADIPOQ Human genes 0.000 description 3
- 102100001249 ALB Human genes 0.000 description 3
- 101710027066 ALB Proteins 0.000 description 3
- 101700002867 ALB4 Proteins 0.000 description 3
- 102100007787 APOA1 Human genes 0.000 description 3
- 101700077954 APOA1 Proteins 0.000 description 3
- 102100007877 APOE Human genes 0.000 description 3
- 101700025839 APOE Proteins 0.000 description 3
- 108010064466 ATP Binding Cassette Transporter 1 Proteins 0.000 description 3
- 101710039338 ATP2A2 Proteins 0.000 description 3
- 102100005631 ATP2A2 Human genes 0.000 description 3
- 102000036638 BRCA1 Human genes 0.000 description 3
- 108010042977 BRCA1 Protein Proteins 0.000 description 3
- 108010040168 Bcl-2-Like Protein 11 Proteins 0.000 description 3
- 102000001765 Bcl-2-Like Protein 11 Human genes 0.000 description 3
- 102000004219 Brain-Derived Neurotrophic Factor Human genes 0.000 description 3
- 108090000715 Brain-Derived Neurotrophic Factor Proteins 0.000 description 3
- 102100007290 CD274 Human genes 0.000 description 3
- 101710012053 CD274 Proteins 0.000 description 3
- 102100000344 CEP290 Human genes 0.000 description 3
- 101710042896 CEP290 Proteins 0.000 description 3
- 108010037462 Cyclooxygenase 2 Proteins 0.000 description 3
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 description 3
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 description 3
- 101700077246 EHMT1 Proteins 0.000 description 3
- 101700084039 EPO Proteins 0.000 description 3
- 101710042422 EPX Proteins 0.000 description 3
- 101700061907 FBF1 Proteins 0.000 description 3
- 102100014925 FLI1 Human genes 0.000 description 3
- 101700057417 FLI1 Proteins 0.000 description 3
- 108060002977 FMR1 Proteins 0.000 description 3
- 102100014905 FMR1 Human genes 0.000 description 3
- 101700005927 FNDC5 Proteins 0.000 description 3
- 102100008753 FNDC5 Human genes 0.000 description 3
- 102100011855 FOXP3 Human genes 0.000 description 3
- 101700064140 FOXP3 Proteins 0.000 description 3
- 102100008497 FXN Human genes 0.000 description 3
- 101710020205 FXN Proteins 0.000 description 3
- 101710042131 GCG Proteins 0.000 description 3
- 101710004768 GER5 Proteins 0.000 description 3
- 101710004110 GLP Proteins 0.000 description 3
- 102100018691 GRN Human genes 0.000 description 3
- 101700007987 GRN Proteins 0.000 description 3
- 101710007394 HAMP Proteins 0.000 description 3
- 102100016195 HAMP Human genes 0.000 description 3
- 102100002631 HBA1 Human genes 0.000 description 3
- 101700000918 HBA2 Proteins 0.000 description 3
- 102100019126 HBB Human genes 0.000 description 3
- 101700071556 HBB Proteins 0.000 description 3
- 102100013132 HBD Human genes 0.000 description 3
- 101700070089 HBD Proteins 0.000 description 3
- 102100007204 HBE1 Human genes 0.000 description 3
- 101700003868 HBE1 Proteins 0.000 description 3
- 102100004900 HBG1 Human genes 0.000 description 3
- 101700073128 HBG1 Proteins 0.000 description 3
- 102100004899 HBG2 Human genes 0.000 description 3
- 101700075536 HBG2 Proteins 0.000 description 3
- 102100016790 HPRT1 Human genes 0.000 description 3
- 101710011940 HPRT1 Proteins 0.000 description 3
- 101700022962 HSP12 Proteins 0.000 description 3
- 102100008614 IDO1 Human genes 0.000 description 3
- 101710031171 IDO1 Proteins 0.000 description 3
- 102100014231 IGF1 Human genes 0.000 description 3
- 101700074337 IGF1 Proteins 0.000 description 3
- 102000003814 Interleukin-10 Human genes 0.000 description 3
- 108090000174 Interleukin-10 Proteins 0.000 description 3
- 102000004889 Interleukin-6 Human genes 0.000 description 3
- 108090001005 Interleukin-6 Proteins 0.000 description 3
- 102100005788 KCNMA1 Human genes 0.000 description 3
- 101710012779 KCNMA1 Proteins 0.000 description 3
- 102100005818 KCNMB1 Human genes 0.000 description 3
- 108060004184 KCNMB1 Proteins 0.000 description 3
- 108060004185 KCNMB2 Proteins 0.000 description 3
- 102100011740 KCNMB4 Human genes 0.000 description 3
- 108060004187 KCNMB4 Proteins 0.000 description 3
- 102100006533 KLF1 Human genes 0.000 description 3
- 101700018946 KLF1 Proteins 0.000 description 3
- 101700048573 KLF4 Proteins 0.000 description 3
- 102100006525 KLF4 Human genes 0.000 description 3
- 102100012475 LDLR Human genes 0.000 description 3
- 108060004326 LDLR Proteins 0.000 description 3
- 102100012761 LRP12 Human genes 0.000 description 3
- 102100014726 MECP2 Human genes 0.000 description 3
- 101700029603 MECP2 Proteins 0.000 description 3
- 102100002038 MSX2 Human genes 0.000 description 3
- 101700072011 MSX2 Proteins 0.000 description 3
- 102100010185 MYBPC3 Human genes 0.000 description 3
- 101710035669 MYBPC3 Proteins 0.000 description 3
- 108010093662 Member 11 Subfamily B ATP Binding Cassette Transporter Proteins 0.000 description 3
- 108010090837 Member 5 Subfamily G ATP Binding Cassette Transporter Proteins 0.000 description 3
- 108010090822 Member 8 Subfamily G ATP Binding Cassette Transporter Proteins 0.000 description 3
- 108020004388 MicroRNAs Proteins 0.000 description 3
- -1 NANOG Proteins 0.000 description 3
- 101700071069 NDP Proteins 0.000 description 3
- 229920001850 Nucleic acid sequence Polymers 0.000 description 3
- 101710042151 Os01g0681900 Proteins 0.000 description 3
- 101710039481 Os03g0179100 Proteins 0.000 description 3
- 102100008799 PTEN Human genes 0.000 description 3
- 108010011536 PTEN Phosphohydrolase Proteins 0.000 description 3
- 102100015381 PTGS2 Human genes 0.000 description 3
- 102100000895 RB1 Human genes 0.000 description 3
- 101710003919 RB1 Proteins 0.000 description 3
- 101710017811 RB1 Proteins 0.000 description 3
- 101710007880 RPS14 Proteins 0.000 description 3
- 102100018347 RPS14 Human genes 0.000 description 3
- 102100018787 RPS19 Human genes 0.000 description 3
- 101710007829 RPS19 Proteins 0.000 description 3
- 101710008324 RPS24 Proteins 0.000 description 3
- 101710008144 RSM19 Proteins 0.000 description 3
- 102100001086 SCARB1 Human genes 0.000 description 3
- 101710031645 SCARB1 Proteins 0.000 description 3
- 102100007444 SERPINF1 Human genes 0.000 description 3
- 101710039390 SERPINF1 Proteins 0.000 description 3
- 102100007214 SIRT1 Human genes 0.000 description 3
- 101700050776 SIRT1 Proteins 0.000 description 3
- 101710045142 SIRT6 Proteins 0.000 description 3
- 102100017984 SIRT6 Human genes 0.000 description 3
- 102100000643 SMAD7 Human genes 0.000 description 3
- 101700026522 SMAD7 Proteins 0.000 description 3
- 101710028394 SMN2 Proteins 0.000 description 3
- 102100002720 SNRPN Human genes 0.000 description 3
- 101710024156 SNRPN Proteins 0.000 description 3
- 108060007902 ST7 Proteins 0.000 description 3
- 102100019667 STAT3 Human genes 0.000 description 3
- 108010017324 STAT3 Transcription Factor Proteins 0.000 description 3
- 101700082977 STMN1 Proteins 0.000 description 3
- 108020003224 Small Nucleolar RNA Proteins 0.000 description 3
- 102100002607 TIMP1 Human genes 0.000 description 3
- 101700040544 TIMP1 Proteins 0.000 description 3
- 108020004417 Untranslated RNA Proteins 0.000 description 3
- 108010075653 Utrophin Proteins 0.000 description 3
- 102000011856 Utrophin Human genes 0.000 description 3
- 102100005236 ZGLP1 Human genes 0.000 description 3
- 101700078733 ZGLP1 Proteins 0.000 description 3
- 150000001720 carbohydrates Chemical class 0.000 description 3
- 235000014633 carbohydrates Nutrition 0.000 description 3
- 150000001768 cations Chemical class 0.000 description 3
- 229920001239 microRNA Polymers 0.000 description 3
- 239000002679 microRNA Substances 0.000 description 3
- 229920001894 non-coding RNA Polymers 0.000 description 3
- PYNVSZMFFVWFQA-NOMGDLSISA-N 2-amino-9-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(1-hydroxyethyl)oxolan-2-yl]-3H-purin-6-one Chemical compound O[C@@H]1[C@H](O)[C@@H](C(O)C)O[C@H]1N1C(NC(N)=NC2=O)=C2N=C1 PYNVSZMFFVWFQA-NOMGDLSISA-N 0.000 description 2
- 229960000643 Adenine Drugs 0.000 description 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Natural products NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 description 2
- 229940104302 Cytosine Drugs 0.000 description 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N Cytosine Chemical group NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 2
- 241000083551 Ena Species 0.000 description 2
- 229940104230 Thymidine Drugs 0.000 description 2
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 description 2
- 229940045145 Uridine Drugs 0.000 description 2
- DRTQHJPVMGBUCF-UCVXFZOQSA-N Uridine Natural products O[C@H]1[C@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UCVXFZOQSA-N 0.000 description 2
- 239000007864 aqueous solution Substances 0.000 description 2
- 150000002632 lipids Chemical class 0.000 description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 description 2
- 239000002105 nanoparticle Substances 0.000 description 2
- 229920001606 poly(lactic acid-co-glycolic acid) Polymers 0.000 description 2
- NKCXQMYPWXSLIZ-PSRDDEIFSA-N (2S)-2-[[(2S)-1-[(2S)-5-amino-2-[[2-[[(2S)-6-amino-2-[[2-[[(2S)-2-[[(2S)-4-amino-2-[[(2S)-2-[[(2S,3R)-2-[[(2S)-2-[[(2S,3R)-2-amino-3-hydroxybutanoyl]amino]-3-(1H-indol-3-yl)propanoyl]amino]-3-hydroxybutanoyl]amino]propanoyl]amino]-4-oxobutanoyl]amino]-3-m Chemical compound O=C([C@H](CCC(N)=O)NC(=O)CNC(=O)[C@H](CCCCN)NC(=O)CNC(=O)[C@@H](NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](C)NC(=O)[C@@H](NC(=O)[C@H](CC=1C2=CC=CC=C2NC=1)NC(=O)[C@@H](N)[C@@H](C)O)[C@@H](C)O)C(C)C)N1CCC[C@H]1C(=O)N[C@@H](CO)C(O)=O NKCXQMYPWXSLIZ-PSRDDEIFSA-N 0.000 description 1
- RUKRVHYQIIURNV-RLKNHCSUSA-N Cc1cn([C@@H]2O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)C2F)c(=O)[nH]c1=O Chemical compound Cc1cn([C@@H]2O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)C2F)c(=O)[nH]c1=O RUKRVHYQIIURNV-RLKNHCSUSA-N 0.000 description 1
- 108091007152 Extracellular RNA Proteins 0.000 description 1
- 229920003004 Extracellular RNA Polymers 0.000 description 1
- 229940012952 Fibrinogen Drugs 0.000 description 1
- 108010049003 Fibrinogen Proteins 0.000 description 1
- 102000008946 Fibrinogen Human genes 0.000 description 1
- 229940019698 Fibrinogen containing hemostatics Drugs 0.000 description 1
- 108020004688 Small Nuclear RNA Proteins 0.000 description 1
- 108090000190 Thrombin Proteins 0.000 description 1
- 229920001949 Transfer RNA Polymers 0.000 description 1
- 108020004566 Transfer RNA Proteins 0.000 description 1
- BQJWHJWAEOHVCK-ZOQUXTDFSA-N [[(2R,3R,4R,5R)-5-(2,4-dioxopyrimidin-1-yl)-3-hydroxy-4-methoxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound CO[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C(=O)NC(=O)C=C1 BQJWHJWAEOHVCK-ZOQUXTDFSA-N 0.000 description 1
- HCXHLIFQJYSIBK-XVFCMESISA-N [[(2R,3R,4R,5R)-5-(2,4-dioxopyrimidin-1-yl)-4-fluoro-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound F[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C(=O)NC(=O)C=C1 HCXHLIFQJYSIBK-XVFCMESISA-N 0.000 description 1
- GMUJBFZJAFFHIW-KQYNXXCUSA-N [[(2R,3R,4R,5R)-5-(2-amino-6-oxo-3H-purin-9-yl)-3-hydroxy-4-methoxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound CO[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C(NC(N)=NC2=O)=C2N=C1 GMUJBFZJAFFHIW-KQYNXXCUSA-N 0.000 description 1
- KYEUAYQSPABKRL-DXTOWSMRSA-N [[(2R,3R,4R,5R)-5-(2-amino-6-oxo-3H-purin-9-yl)-4-fluoro-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)[C@H]1F KYEUAYQSPABKRL-DXTOWSMRSA-N 0.000 description 1
- YCZSHICNESTART-ZOQUXTDFSA-N [[(2R,3R,4R,5R)-5-(4-amino-2-oxopyrimidin-1-yl)-3-hydroxy-4-methoxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound CO[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C(=O)N=C(N)C=C1 YCZSHICNESTART-ZOQUXTDFSA-N 0.000 description 1
- YKEIUAOIVAXJRI-XVFCMESISA-N [[(2R,3R,4R,5R)-5-(4-amino-2-oxopyrimidin-1-yl)-4-fluoro-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](F)[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O1 YKEIUAOIVAXJRI-XVFCMESISA-N 0.000 description 1
- OARVGPYQJRLYFE-IOSLPCCCSA-N [[(2R,3R,4R,5R)-5-(6-aminopurin-9-yl)-3-hydroxy-4-methoxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound CO[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C2=NC=NC(N)=C2N=C1 OARVGPYQJRLYFE-IOSLPCCCSA-N 0.000 description 1
- CCXCDDWASROAJA-QYYRPYCUSA-N [[(2R,3R,4R,5R)-5-(6-aminopurin-9-yl)-4-fluoro-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)[C@H]1F CCXCDDWASROAJA-QYYRPYCUSA-N 0.000 description 1
- ZUZUHROPOMGOKE-ZOQUXTDFSA-N [[(2R,3S,4R,5R)-3,4-dihydroxy-5-[4-(methylamino)-2-oxopyrimidin-1-yl]oxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound O=C1N=C(NC)C=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O1 ZUZUHROPOMGOKE-ZOQUXTDFSA-N 0.000 description 1
- RJZLOYMABJJGTA-XVFCMESISA-N [[(2R,3S,4R,5R)-4-amino-5-(2,4-dioxopyrimidin-1-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound N[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C(=O)NC(=O)C=C1 RJZLOYMABJJGTA-XVFCMESISA-N 0.000 description 1
- KLLLBAMLXCTIJV-DXTOWSMRSA-N [[(2R,3S,4R,5R)-4-amino-5-(2-amino-6-oxo-3H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound N[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C(N=C(N)NC2=O)=C2N=C1 KLLLBAMLXCTIJV-DXTOWSMRSA-N 0.000 description 1
- WNVZQYHBHSLUHJ-XVFCMESISA-N [[(2R,3S,4R,5R)-4-amino-5-(4-amino-2-oxopyrimidin-1-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound N[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C(=O)N=C(N)C=C1 WNVZQYHBHSLUHJ-XVFCMESISA-N 0.000 description 1
- BPRNAIIYYOQQKN-QYYRPYCUSA-N [[(2R,3S,4R,5R)-4-amino-5-(6-aminopurin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound N[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C2=NC=NC(N)=C2N=C1 BPRNAIIYYOQQKN-QYYRPYCUSA-N 0.000 description 1
- KIMZRVBFSWBUIP-DXTOWSMRSA-N [[(2R,3S,4R,5R)-5-(2-amino-6-oxo-3H-purin-9-yl)-4-azido-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)[C@H]1N=[N+]=[N-] KIMZRVBFSWBUIP-DXTOWSMRSA-N 0.000 description 1
- YEVIXVIVDZWHKS-QYYRPYCUSA-N [[(2R,3S,4R,5R)-5-(6-aminopurin-9-yl)-4-azido-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)[C@H]1N=[N+]=[N-] YEVIXVIVDZWHKS-QYYRPYCUSA-N 0.000 description 1
- VEWJOCYCKIZKKV-BLOUUBGSSA-N [[(2R,5S)-5-(2,4-dioxo-1H-pyrimidin-5-yl)-3,4-dihydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound OC1C(O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1C1=CNC(=O)NC1=O VEWJOCYCKIZKKV-BLOUUBGSSA-N 0.000 description 1
- HWSNFUNJICGTGY-RNMRRNDGSA-J [[[(2R,3S,5R)-5-(4-amino-2-oxopyrimidin-1-yl)-4-azido-3-hydroxyoxolan-2-yl]methoxy-oxidophosphoryl]oxy-oxidophosphoryl] phosphate Chemical compound O=C1N=C(N)C=CN1[C@H]1C(N=[N+]=[N-])[C@H](O)[C@@H](COP([O-])(=O)OP([O-])(=O)OP([O-])([O-])=O)O1 HWSNFUNJICGTGY-RNMRRNDGSA-J 0.000 description 1
- ODAFAEFSKRENSW-IOSLPCCCSA-N [hydroxy-[[(2R,3R,4R,5R)-3-hydroxy-4-methoxy-5-(6-oxo-3H-purin-9-yl)oxolan-2-yl]methoxy]phosphoryl] phosphono hydrogen phosphate Chemical compound CO[C@@H]1[C@H](O)[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O[C@H]1N1C2=NC=NC(O)=C2N=C1 ODAFAEFSKRENSW-IOSLPCCCSA-N 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 201000010099 disease Diseases 0.000 description 1
- 239000000499 gel Substances 0.000 description 1
- 239000003292 glue Substances 0.000 description 1
- 239000000017 hydrogel Substances 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 239000004005 microsphere Substances 0.000 description 1
- 239000003607 modifier Substances 0.000 description 1
- 150000002772 monosaccharides Chemical class 0.000 description 1
- 239000000546 pharmaceutic aid Substances 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
- 239000000565 sealant Substances 0.000 description 1
- 229920001255 small nuclear ribonucleic acid Polymers 0.000 description 1
- 238000011105 stabilization Methods 0.000 description 1
- 125000002345 steroid group Chemical group 0.000 description 1
- 150000003431 steroids Chemical class 0.000 description 1
- 238000003786 synthesis reaction Methods 0.000 description 1
- 230000002194 synthesizing Effects 0.000 description 1
- 229960004072 thrombin Drugs 0.000 description 1
- 239000001226 triphosphate Substances 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361866989P | 2013-08-16 | 2013-08-16 | |
| US61/866,989 | 2013-08-16 | ||
| US201361898461P | 2013-10-31 | 2013-10-31 | |
| US61/898,461 | 2013-10-31 | ||
| US201462010417P | 2014-06-10 | 2014-06-10 | |
| US62/010,417 | 2014-06-10 | ||
| PCT/US2014/051331 WO2015023975A1 (en) | 2013-08-16 | 2014-08-15 | Compositions and methods for modulating rna |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016528897A JP2016528897A (ja) | 2016-09-23 |
| JP2016528897A5 true JP2016528897A5 (US07794700-20100914-C00152.png) | 2017-09-28 |
Family
ID=52467112
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016534875A Pending JP2016528897A (ja) | 2013-08-16 | 2014-08-15 | Ｒｎａを調節するための組成物および方法 |
Country Status (13)
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012135805A2 (en) | 2011-03-31 | 2012-10-04 | modeRNA Therapeutics | Delivery and formulation of engineered nucleic acids |
| DK2718269T3 (en) | 2011-06-08 | 2018-04-09 | Translate Bio Inc | SPLITLY LIPIDS |
| WO2014144767A1 (en) | 2013-03-15 | 2014-09-18 | Moderna Therapeutics, Inc. | Ion exchange purification of mrna |
| WO2014152030A1 (en) | 2013-03-15 | 2014-09-25 | Moderna Therapeutics, Inc. | Removal of dna fragments in mrna production process |
| US11377470B2 (en) | 2013-03-15 | 2022-07-05 | Modernatx, Inc. | Ribonucleic acid purification |
| EP3971287A1 (en) | 2013-07-11 | 2022-03-23 | ModernaTX, Inc. | Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use |
| WO2015023941A1 (en) | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Oligonucleotides targeting euchromatin regions of genes |
| WO2015023939A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin |
| WO2015051169A2 (en) | 2013-10-02 | 2015-04-09 | Moderna Therapeutics, Inc. | Polynucleotide molecules and uses thereof |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3157573A4 (en) | 2014-06-19 | 2018-02-21 | Moderna Therapeutics, Inc. | Alternative nucleic acid molecules and uses thereof |
| AU2015289656A1 (en) | 2014-07-16 | 2017-02-16 | Modernatx, Inc. | Circular polynucleotides |
| CA2955250A1 (en) * | 2014-07-16 | 2016-01-21 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
| JP6867945B2 (ja) | 2014-10-03 | 2021-05-12 | コールド スプリング ハーバー ラボラトリー | 核内遺伝子出力の標的とされた増強 |
| US10822369B2 (en) | 2014-11-14 | 2020-11-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of proteins |
| US10758558B2 (en) | 2015-02-13 | 2020-09-01 | Translate Bio Ma, Inc. | Hybrid oligonucleotides and uses thereof |
| EP3256592A4 (en) * | 2015-02-13 | 2018-09-12 | Translate Bio Ma, Inc. | Compositions and methods for modulating rna |
| WO2017049286A1 (en) | 2015-09-17 | 2017-03-23 | Moderna Therapeutics, Inc. | Polynucleotides containing a morpholino linker |
| WO2017060731A1 (en) | 2015-10-09 | 2017-04-13 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| WO2017066789A1 (en) | 2015-10-16 | 2017-04-20 | Modernatx, Inc. | Mrna cap analogs with modified sugar |
| WO2017066791A1 (en) | 2015-10-16 | 2017-04-20 | Modernatx, Inc. | Sugar substituted mrna cap analogs |
| CA3001014A1 (en) | 2015-10-16 | 2017-04-20 | Modernatx, Inc. | Mrna cap analogs and methods of mrna capping |
| ES2914225T3 (es) | 2015-10-16 | 2022-06-08 | Modernatx Inc | Análogos de cap de ARNm con enlace de fosfato modificado |
| WO2017066782A1 (en) | 2015-10-16 | 2017-04-20 | Modernatx, Inc. | Hydrophobic mrna cap analogs |
| AU2016344384A1 (en) | 2015-10-26 | 2018-05-17 | Translate Bio Ma, Inc. | Nanoparticle formulations for delivery of nucleic acid complexes |
| JP2019500899A (ja) | 2015-11-23 | 2019-01-17 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | ＣＲＩＳＰＲ／Ｃａｓ９の核送達を通じた細胞ＲＮＡの追跡と操作 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| US10689689B2 (en) * | 2015-12-28 | 2020-06-23 | Roche Molecular Systems, Inc. | Generic method for the stabilization of specific RNA |
| KR102475301B1 (ko) | 2016-04-08 | 2022-12-09 | 트랜슬레이트 바이오 인코포레이티드 | 다량체 코딩 핵산 및 그 용도 |
| WO2017188898A1 (en) | 2016-04-29 | 2017-11-02 | Nanyang Technological University | G-quadruplex-containing antisense oligonucleotides |
| CN107841510B (zh) * | 2016-09-20 | 2021-02-09 | 中国科学院青岛生物能源与过程研究所 | 一种原核细胞转录后水平控制不同基因表达比例的方法 |
| CN110191952A (zh) * | 2016-10-07 | 2019-08-30 | 瑟卡尔纳制药有限公司 | 治疗癌症的新方法 |
| WO2018089688A1 (en) | 2016-11-09 | 2018-05-17 | Jinjun Shi | Restoration of tumor suppression using mrna-based delivery system |
| MX2019005235A (es) * | 2016-11-09 | 2019-12-05 | Intrexon Corp | Constructos de expresion de frataxina. |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| CN106813960A (zh) * | 2016-12-26 | 2017-06-09 | 广州和实生物技术有限公司 | 一种血液游离rna保护剂及其制备方法与应用 |
| AU2018265022A1 (en) | 2017-05-10 | 2019-11-21 | The Regents Of The University Of California | Directed editing of cellular RNA via nuclear delivery of CRISPR/Cas9 |
| PL3673080T3 (pl) | 2017-08-25 | 2024-03-11 | Stoke Therapeutics, Inc. | Oligomery antysensowne do leczenia stanów i chorób |
| JP6960641B2 (ja) * | 2017-11-09 | 2021-11-05 | 国立大学法人 東京大学 | ｍＲＮＡの安定化方法 |
| BR112020011670A2 (pt) | 2017-12-15 | 2020-11-17 | Flagship Pioneering Innovations Vi, Llc. | composições compreendendo polirribonucleotídeos circulares e seus usos |
| CN108531496B (zh) * | 2018-04-04 | 2020-11-06 | 江南大学 | 一种提高外源基因mRNA数量的DNA及其应用 |
| CN111041001B (zh) * | 2018-10-15 | 2023-02-28 | 上海行深生物科技有限公司 | 治疗kras突变型肿瘤的安全型柯萨奇病毒及其药物组合物 |
| JP2022515211A (ja) | 2018-12-20 | 2022-02-17 | アールネイティブズ・インコーポレイテッド | 合成マイクロｒｎａミミック |
| US20220204978A1 (en) * | 2019-04-16 | 2022-06-30 | The Regents Of The University Of California | Protein translational control |
| CN111041025B (zh) | 2019-12-17 | 2021-06-18 | 深圳市瑞吉生物科技有限公司 | 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法 |
| CN112111524B (zh) * | 2020-01-10 | 2024-02-27 | 深圳瑞吉生物科技有限公司 | mRNA-GalNAc靶向分子的制备方法及其体内递送系统和应用 |
| KR20230022409A (ko) | 2020-05-11 | 2023-02-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머 |
| CN111744019B (zh) | 2020-07-01 | 2023-08-04 | 深圳瑞吉生物科技有限公司 | 基于甘露糖的mRNA靶向递送系统及其应用 |
Family Cites Families (73)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US21020A (en) * | 1858-07-27 | Improved combination of the needle and sun-dial to ascertain time | ||
| US546A (en) * | 1838-01-06 | Loom for weaving knotted counterpanes and other fabrics in which the | ||
| US13402A (en) * | 1855-08-07 | Forming screw-threads | ||
| US1099771A (en) * | 1913-05-26 | 1914-06-09 | Guy P Slater | Derrick. |
| US6111094A (en) * | 1990-08-14 | 2000-08-29 | Isis Pharmaceuticals Inc. | Enhanced antisense modulation of ICAM-1 |
| US20040033977A1 (en) * | 1990-08-14 | 2004-02-19 | Bennett C. Frank | Oligonucleotide modulation of cell adhesion |
| US6582908B2 (en) * | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
| AU4770093A (en) * | 1992-07-02 | 1994-01-31 | Hybridon, Inc. | Self-stabilized oligonucleotides as therapeutic agents |
| CA2167804A1 (en) * | 1993-07-20 | 1995-02-02 | Robert H. Singer | In vivo nucleic acid hybridization method |
| US5962332A (en) * | 1994-03-17 | 1999-10-05 | University Of Massachusetts | Detection of trinucleotide repeats by in situ hybridization |
| US5866331A (en) * | 1995-10-20 | 1999-02-02 | University Of Massachusetts | Single molecule detection by in situ hybridization |
| US5962675A (en) * | 1996-02-13 | 1999-10-05 | Ribozyme Pharmaceuticals, Inc. | Chemical syntheses of 2'-O-methoxy purine nucleosides |
| US6261836B1 (en) * | 1996-10-01 | 2001-07-17 | Geron Corporation | Telomerase |
| US6391543B2 (en) * | 1997-04-23 | 2002-05-21 | Abbott Laboratories | Reagents and methods useful for detecting diseases of the prostate |
| AU8280798A (en) * | 1997-07-03 | 1999-01-25 | Thomas Jefferson University | An improved method for design and selection of efficacious antisense oligonucleotides |
| WO1999010509A1 (en) * | 1997-08-22 | 1999-03-04 | The Government Of The United States Of America, Represented By The Secretary Of Health And Human Services, National Institutes Of Health | Polynucleotide inhibition of rna destabilization and sequestration |
| US6322978B1 (en) * | 1998-04-20 | 2001-11-27 | Joslin Diabetes Center, Inc. | Repeat polymorphism in the frataxin gene and uses therefore |
| EP1190047A1 (en) * | 1999-06-23 | 2002-03-27 | Angiogene, Inc. | Antisense oligonucleotide modulating cyclin e gene expression and therapeutic uses thereof |
| AU2074101A (en) * | 1999-12-08 | 2001-06-18 | Millennium Pharmaceuticals, Inc. | Compositions, kits, and methods for identification, assessment, prevention, and therapy of cervical cancer |
| EP1130121A3 (en) * | 2000-01-26 | 2003-04-16 | Nisshinbo Industries Inc. | Immobilized nucleic acid and method for detecting nucleic acid |
| US6503756B1 (en) * | 2000-09-22 | 2003-01-07 | Isis Pharmaceuticals, Inc. | Antisense modulation of syntaxin 4 interacting protein expression |
| US20030125273A1 (en) * | 2001-12-05 | 2003-07-03 | Isis Pharmaceuticals Inc, | Antisense modulation of MHC class II transactivator expression |
| GB0101397D0 (en) * | 2001-01-19 | 2001-03-07 | Amersham Pharm Biotech Uk Ltd | Suppression of non-specific nucleic acid amplication |
| US20050048529A1 (en) * | 2002-02-20 | 2005-03-03 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA) |
| US20030125241A1 (en) * | 2001-05-18 | 2003-07-03 | Margit Wissenbach | Therapeutic uses of LNA-modified oligonucleotides in infectious diseases |
| US7399590B2 (en) * | 2002-02-21 | 2008-07-15 | Asm Scientific, Inc. | Recombinase polymerase amplification |
| US20040023906A1 (en) * | 2002-08-01 | 2004-02-05 | Isis Pharmaceuticals Inc. | Antisense modulation of phosphotyrosyl phosphatase activator expression |
| DK2264172T3 (da) * | 2002-04-05 | 2017-11-27 | Roche Innovation Ct Copenhagen As | Oligomerforbindelser til modulering af HIF-1á-ekspression |
| US20040097441A1 (en) * | 2002-11-16 | 2004-05-20 | Isis Pharmaceuticals Inc. | Modulation of NIMA-related kinase 6 expression |
| US20040005565A1 (en) * | 2002-07-02 | 2004-01-08 | Isis Pharmaceuticals Inc. | Antisense modulation of livin expression |
| WO2004044136A2 (en) * | 2002-11-05 | 2004-05-27 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2’-modified nucleosides for use in gene modulation |
| JP2006507841A (ja) * | 2002-11-14 | 2006-03-09 | ダーマコン， インコーポレイテッド | 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ |
| DK2284269T3 (en) * | 2002-11-18 | 2017-10-23 | Roche Innovation Ct Copenhagen As | Antisense design |
| US20040110153A1 (en) * | 2002-12-10 | 2004-06-10 | Affymetrix, Inc. | Compleixity management of genomic DNA by semi-specific amplification |
| US7750144B2 (en) * | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| US20050014168A1 (en) * | 2003-06-03 | 2005-01-20 | Arcturus Bioscience, Inc. | 3' biased microarrays |
| EP1668130A2 (en) * | 2003-09-18 | 2006-06-14 | Isis Pharmaceuticals, Inc. | Modulation of eif4e expression |
| US20050108783A1 (en) * | 2003-09-23 | 2005-05-19 | Chihiro Koike | Porcine invariant chain protein, full length cDNA, genomic organization, and regulatory region |
| US20050261217A1 (en) * | 2004-05-18 | 2005-11-24 | Isis Pharmaceuticals Inc. | Modulation of pumilio 1 expression |
| US20050261216A1 (en) * | 2004-05-18 | 2005-11-24 | Isis Pharmaceuticals Inc. | Modulation of Nanos 1 expression |
| EP1765415A4 (en) * | 2004-06-03 | 2010-03-24 | Isis Pharmaceuticals Inc | OLIGOMERIC COMPOUNDS FACILITATING THE "RISC" LOAD |
| US20050287539A1 (en) * | 2004-06-29 | 2005-12-29 | Emmanuel Labourier | Methods and compositions for preparing capped RNA |
| DE102004034987A1 (de) * | 2004-07-16 | 2006-02-02 | Carl Zeiss Jena Gmbh | Lichtrastermikroskop und Verwendung |
| US7718625B2 (en) * | 2005-01-27 | 2010-05-18 | University Of South Florida | Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof |
| WO2006082826A1 (ja) * | 2005-02-02 | 2006-08-10 | Eisai R & D Management Co., Ltd. | Corl2遺伝子を標的としたプルキンエ細胞識別方法 |
| WO2006130201A1 (en) * | 2005-03-14 | 2006-12-07 | Board Of Regents, The University Of Texas System | Antigene oligomers inhibit transcription |
| EP1957648B1 (en) * | 2005-11-17 | 2014-04-23 | Board of Regents, The University of Texas System | Modulation of gene expression by oligomers targeted to chromosomal dna |
| US7807652B2 (en) * | 2005-11-21 | 2010-10-05 | Isis Pharmaceuticals, Inc. | Modulation of eIF4E-BP2 expression |
| US8685899B2 (en) * | 2007-02-14 | 2014-04-01 | Genisphere Inc. | Methods, reagents and kits for detection of nucleic acid molecules |
| MX2009012568A (es) * | 2007-05-22 | 2009-12-08 | Mdrna Inc | Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico. |
| US20090082297A1 (en) * | 2007-06-25 | 2009-03-26 | Lioy Daniel T | Compositions and Methods for Regulating Gene Expression |
| EP2173913A4 (en) * | 2007-08-03 | 2010-09-01 | Biocept Inc | IN-SITU HYBRIDIZATION FOR THE DETECTION OF RNA AND DNA MARKERS |
| US20110009466A1 (en) * | 2007-08-29 | 2011-01-13 | President And Fellows Of Harvard College | Methods of increasing gene expression through rna protection |
| WO2009046397A2 (en) * | 2007-10-04 | 2009-04-09 | Board Of Regents, The University Of Texas System | Modulating gene expression with agrna and gapmers targeting antisense transcripts |
| UA100253C2 (uk) * | 2007-11-26 | 2012-12-10 | Сантаріс Фарма А/С | Lna-антагоністи андрогенного рецептора |
| EP2235033A4 (en) * | 2007-12-28 | 2011-11-02 | Univ California | METHOD AND COMPOSITIONS FOR INCREASING GENE EXPRESSION |
| US20100297750A1 (en) * | 2008-01-24 | 2010-11-25 | Toru Natsume | Polynucleotide or analogue thereof, and gene expression regulation method using the polynucleotide or the analogue thereof |
| US20110263687A1 (en) * | 2008-04-07 | 2011-10-27 | Riken | Rna molecules and uses thereof |
| US8669102B2 (en) * | 2008-08-14 | 2014-03-11 | Isis Pharmaceuticals, Inc. | Modulation of prion expression |
| US8815586B2 (en) * | 2009-04-24 | 2014-08-26 | The Board Of Regents Of The University Of Texas System | Modulation of gene expression using oligomers that target gene regions downstream of 3′ untranslated regions |
| PL2499249T3 (pl) * | 2009-11-12 | 2019-03-29 | Univ Western Australia | Cząsteczki antysensowne i sposoby leczenia patologii |
| WO2011130371A1 (en) * | 2010-04-13 | 2011-10-20 | Life Technologies Corporation | Compositions and methods for inhibition of nucleic acids function |
| US20110306653A1 (en) * | 2010-05-14 | 2011-12-15 | Tagcyx Biotechnologies | Stabilization method of functional nucleic acid |
| GB201010557D0 (en) * | 2010-06-23 | 2010-08-11 | Mina Therapeutics Ltd | RNA molecules and uses thereof |
| WO2012122645A1 (en) * | 2011-03-11 | 2012-09-20 | Sarissa Inc. | Methods of treating cancer by inhibition of dna repair proteins |
| US9476043B2 (en) * | 2011-04-08 | 2016-10-25 | Rula Zain-Luqman | Diagnosis and treatment of friedreich's ataxia |
| CN103620036B (zh) * | 2011-06-09 | 2016-12-21 | 库尔纳公司 | 通过抑制共济蛋白(fxn)的天然反义转录物而治疗fxn 相关疾病 |
| DK2756080T3 (da) * | 2011-09-14 | 2019-05-20 | Translate Bio Ma Inc | Multimeriske oligonukleotidforbindelser |
| US20150247141A1 (en) * | 2012-09-14 | 2015-09-03 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| US20160032273A1 (en) * | 2013-03-15 | 2016-02-04 | Moderna Therapeutics, Inc. | Characterization of mrna molecules |
| CN105370259A (zh) * | 2014-08-29 | 2016-03-02 | 中国石油化工股份有限公司 | 水平井分段压裂方法 |
| CN107987053B (zh) * | 2017-12-08 | 2023-04-14 | 植恩生物技术股份有限公司 | 一种高纯度z型盐酸氟哌噻吨的制备方法 |
| CN108590349A (zh) * | 2018-06-11 | 2018-09-28 | 太仓市金毅电子有限公司 | 具有防撬功能的智能锁 |
-
2014
- 2014-08-15 JP JP2016534875A patent/JP2016528897A/ja active Pending
- 2014-08-15 US US14/461,317 patent/US20150050738A1/en not_active Abandoned
- 2014-08-15 SG SG11201600987TA patent/SG11201600987TA/en unknown
- 2014-08-15 EA EA201690403A patent/EA201690403A1/ru unknown
- 2014-08-15 KR KR1020167006517A patent/KR20160036065A/ko not_active Application Discontinuation
- 2014-08-15 CN CN201480056023.2A patent/CN105658797A/zh active Pending
- 2014-08-15 BR BR112016003127A patent/BR112016003127A2/pt not_active Application Discontinuation
- 2014-08-15 EP EP14835805.4A patent/EP3033424A4/en active Pending
- 2014-08-15 CA CA2921556A patent/CA2921556A1/en not_active Abandoned
- 2014-08-15 WO PCT/US2014/051331 patent/WO2015023975A1/en active Application Filing
- 2014-08-15 AU AU2014306416A patent/AU2014306416B2/en not_active Ceased
- 2014-08-15 MX MX2016002044A patent/MX2016002044A/es unknown
-
2015
- 2015-04-30 US US14/700,311 patent/US20150232844A1/en not_active Abandoned
- 2015-04-30 US US14/700,491 patent/US20150232846A1/en not_active Abandoned
- 2015-04-30 US US14/700,395 patent/US20150247145A1/en not_active Abandoned
- 2015-04-30 US US14/700,555 patent/US20150232847A1/en not_active Abandoned
- 2015-04-30 US US14/700,445 patent/US20170152511A9/en not_active Abandoned
- 2015-04-30 US US14/700,529 patent/US20150225715A1/en not_active Abandoned
- 2015-04-30 US US14/700,334 patent/US20150247144A1/en not_active Abandoned
-
2016
- 2016-02-11 IL IL244081A patent/IL244081A0/en unknown
-
2021
- 2021-05-18 AU AU2021203174A patent/AU2021203174A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016528897A5 (US07794700-20100914-C00152.png) | ||
| JP6647430B2 (ja) | アンチセンス核酸 | |
| JP2019519218A5 (US07794700-20100914-C00152.png) | ||
| JP2020518266A5 (US07794700-20100914-C00152.png) | ||
| KR102335801B1 (ko) | 안티센스 핵산 | |
| JP2024023899A (ja) | アンチセンス核酸 | |
| JP6384845B2 (ja) | アンチセンス核酸 | |
| JP2015523854A5 (US07794700-20100914-C00152.png) | ||
| JP2015523853A5 (US07794700-20100914-C00152.png) | ||
| JP2015518710A5 (US07794700-20100914-C00152.png) | ||
| CA3084170A1 (en) | Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage | |
| US11597926B2 (en) | Thiophosphoramidites | |
| US20230002762A1 (en) | Oligonucleotides comprising a phosphorodithioate internucleoside linkage |
