JP2012520085A - 生物活性ｒｎａの送達のための組成物及び方法 - Google Patents
生物活性ｒｎａの送達のための組成物及び方法 Download PDFInfo
- Publication number
- JP2012520085A JP2012520085A JP2011554275A JP2011554275A JP2012520085A JP 2012520085 A JP2012520085 A JP 2012520085A JP 2011554275 A JP2011554275 A JP 2011554275A JP 2011554275 A JP2011554275 A JP 2011554275A JP 2012520085 A JP2012520085 A JP 2012520085A
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- rna
- sequences
- cell
- polynucleotide
- 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
- 230000000975 bioactive effect Effects 0.000 title claims abstract description 296
- 238000000034 method Methods 0.000 title claims abstract description 145
- 239000000203 mixture Substances 0.000 title claims abstract description 62
- 102000040430 polynucleotide Human genes 0.000 claims abstract description 362
- 108091033319 polynucleotide Proteins 0.000 claims abstract description 362
- 239000002157 polynucleotide Substances 0.000 claims abstract description 362
- 108090000765 processed proteins & peptides Proteins 0.000 claims abstract description 318
- 150000007523 nucleic acids Chemical class 0.000 claims abstract description 222
- 102000039446 nucleic acids Human genes 0.000 claims abstract description 219
- 108020004707 nucleic acids Proteins 0.000 claims abstract description 219
- 102000004196 processed proteins & peptides Human genes 0.000 claims abstract description 169
- 230000014509 gene expression Effects 0.000 claims abstract description 121
- 229920001184 polypeptide Polymers 0.000 claims abstract description 103
- 239000013598 vector Substances 0.000 claims abstract description 83
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 554
- 239000013604 expression vector Substances 0.000 claims description 342
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 227
- 108090000623 proteins and genes Proteins 0.000 claims description 205
- 230000004570 RNA-binding Effects 0.000 claims description 161
- 230000028327 secretion Effects 0.000 claims description 88
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 77
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 77
- 230000003612 virological effect Effects 0.000 claims description 71
- 108010052104 Viral Regulatory and Accessory Proteins Proteins 0.000 claims description 39
- 108010059722 Viral Fusion Proteins Proteins 0.000 claims description 37
- 230000029812 viral genome replication Effects 0.000 claims description 34
- 108090000565 Capsid Proteins Proteins 0.000 claims description 32
- 239000003937 drug carrier Substances 0.000 claims description 31
- 230000003248 secreting effect Effects 0.000 claims description 26
- 101710141454 Nucleoprotein Proteins 0.000 claims description 24
- 241000700605 Viruses Species 0.000 claims description 19
- 101710132601 Capsid protein Proteins 0.000 claims description 10
- 101710094648 Coat protein Proteins 0.000 claims description 10
- 102100021181 Golgi phosphoprotein 3 Human genes 0.000 claims description 10
- 101710125418 Major capsid protein Proteins 0.000 claims description 10
- 101710083689 Probable capsid protein Proteins 0.000 claims description 10
- 108700026220 vif Genes Proteins 0.000 claims description 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract description 21
- 201000010099 disease Diseases 0.000 abstract description 19
- 239000012096 transfection reagent Substances 0.000 abstract description 19
- 150000001875 compounds Chemical class 0.000 abstract description 8
- 238000003745 diagnosis Methods 0.000 abstract description 2
- 208000035475 disorder Diseases 0.000 abstract description 2
- 230000002265 prevention Effects 0.000 abstract description 2
- 210000004027 cell Anatomy 0.000 description 505
- 239000004055 small Interfering RNA Substances 0.000 description 176
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 107
- 102000037865 fusion proteins Human genes 0.000 description 106
- 108020001507 fusion proteins Proteins 0.000 description 106
- 108091027967 Small hairpin RNA Proteins 0.000 description 105
- 210000004748 cultured cell Anatomy 0.000 description 87
- 108020004459 Small interfering RNA Proteins 0.000 description 77
- 108700011259 MicroRNAs Proteins 0.000 description 75
- 239000002679 microRNA Substances 0.000 description 75
- 238000007792 addition Methods 0.000 description 73
- 230000027455 binding Effects 0.000 description 58
- 238000009739 binding Methods 0.000 description 58
- 239000013612 plasmid Substances 0.000 description 56
- 235000018102 proteins Nutrition 0.000 description 54
- 102000004169 proteins and genes Human genes 0.000 description 54
- 239000002773 nucleotide Substances 0.000 description 49
- 125000003729 nucleotide group Chemical group 0.000 description 49
- 230000004927 fusion Effects 0.000 description 46
- 102000005962 receptors Human genes 0.000 description 46
- 108020003175 receptors Proteins 0.000 description 46
- 102100023387 Endoribonuclease Dicer Human genes 0.000 description 42
- 101000907904 Homo sapiens Endoribonuclease Dicer Proteins 0.000 description 42
- 230000032258 transport Effects 0.000 description 39
- 101100144701 Mus musculus Drosha gene Proteins 0.000 description 37
- 125000003275 alpha amino acid group Chemical group 0.000 description 36
- 235000001014 amino acid Nutrition 0.000 description 36
- 229940024606 amino acid Drugs 0.000 description 36
- 150000001413 amino acids Chemical class 0.000 description 36
- 230000008685 targeting Effects 0.000 description 34
- 108091023037 Aptamer Proteins 0.000 description 33
- 230000000694 effects Effects 0.000 description 31
- 108010037362 Extracellular Matrix Proteins Proteins 0.000 description 30
- 102000010834 Extracellular Matrix Proteins Human genes 0.000 description 30
- 241000725303 Human immunodeficiency virus Species 0.000 description 30
- 238000001727 in vivo Methods 0.000 description 27
- 101900318283 Alfalfa mosaic virus Capsid protein Proteins 0.000 description 25
- 108010000521 Human Growth Hormone Proteins 0.000 description 25
- 102000002265 Human Growth Hormone Human genes 0.000 description 25
- 239000000854 Human Growth Hormone Substances 0.000 description 25
- 210000002744 extracellular matrix Anatomy 0.000 description 25
- 238000001890 transfection Methods 0.000 description 25
- 108090000994 Catalytic RNA Proteins 0.000 description 24
- 102000053642 Catalytic RNA Human genes 0.000 description 24
- 108091092562 ribozyme Proteins 0.000 description 24
- 239000013603 viral vector Substances 0.000 description 24
- 210000000805 cytoplasm Anatomy 0.000 description 23
- 108020004999 messenger RNA Proteins 0.000 description 23
- 230000010076 replication Effects 0.000 description 23
- 108020004414 DNA Proteins 0.000 description 21
- 229930027917 kanamycin Natural products 0.000 description 21
- 229960000318 kanamycin Drugs 0.000 description 21
- SBUJHOSQTJFQJX-NOAMYHISSA-N kanamycin Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CN)O[C@@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](N)[C@H](O)[C@@H](CO)O2)O)[C@H](N)C[C@@H]1N SBUJHOSQTJFQJX-NOAMYHISSA-N 0.000 description 21
- 229930182823 kanamycin A Natural products 0.000 description 21
- 241000124008 Mammalia Species 0.000 description 19
- 230000002950 deficient Effects 0.000 description 19
- 230000000670 limiting effect Effects 0.000 description 19
- 210000001519 tissue Anatomy 0.000 description 18
- 239000000047 product Substances 0.000 description 17
- 241001515965 unidentified phage Species 0.000 description 17
- 230000007246 mechanism Effects 0.000 description 16
- 125000005647 linker group Chemical group 0.000 description 15
- 238000004519 manufacturing process Methods 0.000 description 15
- 125000006850 spacer group Chemical group 0.000 description 15
- 230000000692 anti-sense effect Effects 0.000 description 14
- 238000000338 in vitro Methods 0.000 description 14
- 108010044467 Isoenzymes Proteins 0.000 description 13
- 238000010367 cloning Methods 0.000 description 13
- 238000004806 packaging method and process Methods 0.000 description 13
- 238000012360 testing method Methods 0.000 description 13
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 description 12
- 230000037361 pathway Effects 0.000 description 12
- 108091008146 restriction endonucleases Proteins 0.000 description 12
- 108091026890 Coding region Proteins 0.000 description 11
- 102000001301 EGF receptor Human genes 0.000 description 11
- 108060006698 EGF receptor Proteins 0.000 description 11
- 108091008103 RNA aptamers Proteins 0.000 description 11
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 description 11
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 description 11
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 description 11
- 238000003556 assay Methods 0.000 description 11
- 239000012634 fragment Substances 0.000 description 11
- 230000006870 function Effects 0.000 description 11
- 230000003993 interaction Effects 0.000 description 11
- 238000002156 mixing Methods 0.000 description 11
- 239000002245 particle Substances 0.000 description 10
- 230000001105 regulatory effect Effects 0.000 description 10
- 241000701161 unidentified adenovirus Species 0.000 description 10
- 238000011144 upstream manufacturing Methods 0.000 description 10
- 108091029499 Group II intron Proteins 0.000 description 9
- 108091027981 Response element Proteins 0.000 description 9
- 102100022013 U1 small nuclear ribonucleoprotein A Human genes 0.000 description 9
- 101710106597 U1 small nuclear ribonucleoprotein A Proteins 0.000 description 9
- 108091008605 VEGF receptors Proteins 0.000 description 9
- 102000009484 Vascular Endothelial Growth Factor Receptors Human genes 0.000 description 9
- 239000003814 drug Substances 0.000 description 9
- 238000013518 transcription Methods 0.000 description 9
- 230000035897 transcription Effects 0.000 description 9
- 229940124676 vascular endothelial growth factor receptor Drugs 0.000 description 9
- 101000742844 Homo sapiens RNA-binding motif protein, Y chromosome, family 1 member A1 Proteins 0.000 description 8
- 101000773184 Homo sapiens Twist-related protein 1 Proteins 0.000 description 8
- 102100021010 Nucleolin Human genes 0.000 description 8
- 102100038040 RNA-binding motif protein, Y chromosome, family 1 member A1 Human genes 0.000 description 8
- 102100030398 Twist-related protein 1 Human genes 0.000 description 8
- 230000000295 complement effect Effects 0.000 description 8
- 230000002401 inhibitory effect Effects 0.000 description 8
- 230000001404 mediated effect Effects 0.000 description 8
- 108010044762 nucleolin Proteins 0.000 description 8
- RXWNCPJZOCPEPQ-NVWDDTSBSA-N puromycin Chemical compound C1=CC(OC)=CC=C1C[C@H](N)C(=O)N[C@H]1[C@@H](O)[C@H](N2C3=NC=NC(=C3N=C2)N(C)C)O[C@@H]1CO RXWNCPJZOCPEPQ-NVWDDTSBSA-N 0.000 description 8
- 108010085238 Actins Proteins 0.000 description 7
- 102000007469 Actins Human genes 0.000 description 7
- 101100004408 Arabidopsis thaliana BIG gene Proteins 0.000 description 7
- 241000701022 Cytomegalovirus Species 0.000 description 7
- 101100485279 Drosophila melanogaster emb gene Proteins 0.000 description 7
- 102100029095 Exportin-1 Human genes 0.000 description 7
- 239000005089 Luciferase Substances 0.000 description 7
- 101100485284 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) CRM1 gene Proteins 0.000 description 7
- 101150094313 XPO1 gene Proteins 0.000 description 7
- 238000013459 approach Methods 0.000 description 7
- 239000002585 base Substances 0.000 description 7
- 230000015572 biosynthetic process Effects 0.000 description 7
- 230000001413 cellular effect Effects 0.000 description 7
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 description 7
- 108700002148 exportin 1 Proteins 0.000 description 7
- 239000012528 membrane Substances 0.000 description 7
- 108010043655 penetratin Proteins 0.000 description 7
- MCYTYTUNNNZWOK-LCLOTLQISA-N penetratin Chemical group C([C@H](NC(=O)[C@H](CC=1C2=CC=CC=C2NC=1)NC(=O)[C@H]([C@@H](C)CC)NC(=O)[C@H](CCCCN)NC(=O)[C@@H](NC(=O)[C@H](CCC(N)=O)NC(=O)[C@@H](N)CCCNC(N)=N)[C@@H](C)CC)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCSC)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCCCN)C(N)=O)C1=CC=CC=C1 MCYTYTUNNNZWOK-LCLOTLQISA-N 0.000 description 7
- 239000002953 phosphate buffered saline Substances 0.000 description 7
- 238000003757 reverse transcription PCR Methods 0.000 description 7
- 238000012546 transfer Methods 0.000 description 7
- 102100040006 Annexin A1 Human genes 0.000 description 6
- 108090000663 Annexin A1 Proteins 0.000 description 6
- 101150019028 Antp gene Proteins 0.000 description 6
- 101100339431 Arabidopsis thaliana HMGB2 gene Proteins 0.000 description 6
- 102000003971 Fibroblast Growth Factor 1 Human genes 0.000 description 6
- 108090000386 Fibroblast Growth Factor 1 Proteins 0.000 description 6
- 102000003974 Fibroblast growth factor 2 Human genes 0.000 description 6
- 108090000379 Fibroblast growth factor 2 Proteins 0.000 description 6
- 102000016621 Focal Adhesion Protein-Tyrosine Kinases Human genes 0.000 description 6
- 108010067715 Focal Adhesion Protein-Tyrosine Kinases Proteins 0.000 description 6
- 102000000802 Galectin 3 Human genes 0.000 description 6
- 108010001517 Galectin 3 Proteins 0.000 description 6
- 108700007698 Genetic Terminator Regions Proteins 0.000 description 6
- 108700010013 HMGB1 Proteins 0.000 description 6
- 101150021904 HMGB1 gene Proteins 0.000 description 6
- 102100037907 High mobility group protein B1 Human genes 0.000 description 6
- XQFRJNBWHJMXHO-RRKCRQDMSA-N IDUR Chemical compound C1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(I)=C1 XQFRJNBWHJMXHO-RRKCRQDMSA-N 0.000 description 6
- 102000001938 Plasminogen Activators Human genes 0.000 description 6
- 108010001014 Plasminogen Activators Proteins 0.000 description 6
- 101710192266 Tegument protein VP22 Proteins 0.000 description 6
- 102000002933 Thioredoxin Human genes 0.000 description 6
- 108060008539 Transglutaminase Proteins 0.000 description 6
- 230000015556 catabolic process Effects 0.000 description 6
- 238000006731 degradation reaction Methods 0.000 description 6
- 241001493065 dsRNA viruses Species 0.000 description 6
- 210000004940 nucleus Anatomy 0.000 description 6
- 229940127126 plasminogen activator Drugs 0.000 description 6
- 239000000758 substrate Substances 0.000 description 6
- 108060008226 thioredoxin Proteins 0.000 description 6
- 229940094937 thioredoxin Drugs 0.000 description 6
- 102000003601 transglutaminase Human genes 0.000 description 6
- 102100021569 Apoptosis regulator Bcl-2 Human genes 0.000 description 5
- 102100021663 Baculoviral IAP repeat-containing protein 5 Human genes 0.000 description 5
- 108060000903 Beta-catenin Proteins 0.000 description 5
- 102000015735 Beta-catenin Human genes 0.000 description 5
- 101150104494 CAV1 gene Proteins 0.000 description 5
- 241000450599 DNA viruses Species 0.000 description 5
- 206010059866 Drug resistance Diseases 0.000 description 5
- 101000971171 Homo sapiens Apoptosis regulator Bcl-2 Proteins 0.000 description 5
- 108010002350 Interleukin-2 Proteins 0.000 description 5
- 102000000588 Interleukin-2 Human genes 0.000 description 5
- 108060001084 Luciferase Proteins 0.000 description 5
- 206010028980 Neoplasm Diseases 0.000 description 5
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 description 5
- 101150001535 SRC gene Proteins 0.000 description 5
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 5
- 108010002687 Survivin Proteins 0.000 description 5
- 108010067674 Viral Nonstructural Proteins Proteins 0.000 description 5
- 230000004071 biological effect Effects 0.000 description 5
- 201000011510 cancer Diseases 0.000 description 5
- 238000004113 cell culture Methods 0.000 description 5
- 229940079593 drug Drugs 0.000 description 5
- 210000001163 endosome Anatomy 0.000 description 5
- 230000009368 gene silencing by RNA Effects 0.000 description 5
- 230000010534 mechanism of action Effects 0.000 description 5
- 230000008488 polyadenylation Effects 0.000 description 5
- 102000000844 Cell Surface Receptors Human genes 0.000 description 4
- 108010001857 Cell Surface Receptors Proteins 0.000 description 4
- 101150106019 Mmp2 gene Proteins 0.000 description 4
- 108010076504 Protein Sorting Signals Proteins 0.000 description 4
- 241000700584 Simplexvirus Species 0.000 description 4
- 210000004978 chinese hamster ovary cell Anatomy 0.000 description 4
- 230000030279 gene silencing Effects 0.000 description 4
- 238000009396 hybridization Methods 0.000 description 4
- 229950010131 puromycin Drugs 0.000 description 4
- 239000011780 sodium chloride Substances 0.000 description 4
- 230000014616 translation Effects 0.000 description 4
- 238000013519 translation Methods 0.000 description 4
- 108020004463 18S ribosomal RNA Proteins 0.000 description 3
- 108020005544 Antisense RNA Proteins 0.000 description 3
- 102000053602 DNA Human genes 0.000 description 3
- 101710177611 DNA polymerase II large subunit Proteins 0.000 description 3
- 101710184669 DNA polymerase II small subunit Proteins 0.000 description 3
- 241000702421 Dependoparvovirus Species 0.000 description 3
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 description 3
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 3
- 108091092195 Intron Proteins 0.000 description 3
- 241000713666 Lentivirus Species 0.000 description 3
- 108010085220 Multiprotein Complexes Proteins 0.000 description 3
- 102000007474 Multiprotein Complexes Human genes 0.000 description 3
- 102000000574 RNA-Induced Silencing Complex Human genes 0.000 description 3
- 108010016790 RNA-Induced Silencing Complex Proteins 0.000 description 3
- 241000710960 Sindbis virus Species 0.000 description 3
- 108010067390 Viral Proteins Proteins 0.000 description 3
- 239000013592 cell lysate Substances 0.000 description 3
- 210000000170 cell membrane Anatomy 0.000 description 3
- 210000003855 cell nucleus Anatomy 0.000 description 3
- 238000006243 chemical reaction Methods 0.000 description 3
- 239000003184 complementary RNA Substances 0.000 description 3
- 239000008121 dextrose Substances 0.000 description 3
- 238000003197 gene knockdown Methods 0.000 description 3
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 3
- 238000001114 immunoprecipitation Methods 0.000 description 3
- 239000003446 ligand Substances 0.000 description 3
- 230000033001 locomotion Effects 0.000 description 3
- 238000002844 melting Methods 0.000 description 3
- 230000008018 melting Effects 0.000 description 3
- 230000036961 partial effect Effects 0.000 description 3
- 230000000149 penetrating effect Effects 0.000 description 3
- 239000013641 positive control Substances 0.000 description 3
- 230000032361 posttranscriptional gene silencing Effects 0.000 description 3
- 239000003531 protein hydrolysate Substances 0.000 description 3
- 238000003786 synthesis reaction Methods 0.000 description 3
- 241001430294 unidentified retrovirus Species 0.000 description 3
- 108091032955 Bacterial small RNA Proteins 0.000 description 2
- 102000014914 Carrier Proteins Human genes 0.000 description 2
- 102000004190 Enzymes Human genes 0.000 description 2
- 108090000790 Enzymes Proteins 0.000 description 2
- ZHNUHDYFZUAESO-UHFFFAOYSA-N Formamide Chemical compound NC=O ZHNUHDYFZUAESO-UHFFFAOYSA-N 0.000 description 2
- 108010048671 Homeodomain Proteins Proteins 0.000 description 2
- 102000009331 Homeodomain Proteins Human genes 0.000 description 2
- 241000713772 Human immunodeficiency virus 1 Species 0.000 description 2
- 241000714192 Human spumaretrovirus Species 0.000 description 2
- 238000000636 Northern blotting Methods 0.000 description 2
- 101710163270 Nuclease Proteins 0.000 description 2
- 108090000630 Oncostatin M Proteins 0.000 description 2
- 102000004140 Oncostatin M Human genes 0.000 description 2
- 108020005067 RNA Splice Sites Proteins 0.000 description 2
- -1 RNA-protein complex Proteins 0.000 description 2
- 101710100969 Receptor tyrosine-protein kinase erbB-3 Proteins 0.000 description 2
- 102100029986 Receptor tyrosine-protein kinase erbB-3 Human genes 0.000 description 2
- 239000004098 Tetracycline Substances 0.000 description 2
- 102000040945 Transcription factor Human genes 0.000 description 2
- 108091023040 Transcription factor Proteins 0.000 description 2
- 108020005202 Viral DNA Proteins 0.000 description 2
- 239000012190 activator Substances 0.000 description 2
- 239000011543 agarose gel Substances 0.000 description 2
- 229960000723 ampicillin Drugs 0.000 description 2
- AVKUERGKIZMTKX-NJBDSQKTSA-N ampicillin Chemical compound C1([C@@H](N)C(=O)N[C@H]2[C@H]3SC([C@@H](N3C2=O)C(O)=O)(C)C)=CC=CC=C1 AVKUERGKIZMTKX-NJBDSQKTSA-N 0.000 description 2
- 230000003321 amplification Effects 0.000 description 2
- 230000009830 antibody antigen interaction Effects 0.000 description 2
- 230000008901 benefit Effects 0.000 description 2
- 230000033228 biological regulation Effects 0.000 description 2
- 108010006025 bovine growth hormone Proteins 0.000 description 2
- 230000003197 catalytic effect Effects 0.000 description 2
- 239000006143 cell culture medium Substances 0.000 description 2
- 239000003153 chemical reaction reagent Substances 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 229960005091 chloramphenicol Drugs 0.000 description 2
- WIIZWVCIJKGZOK-RKDXNWHRSA-N chloramphenicol Chemical compound ClC(Cl)C(=O)N[C@H](CO)[C@H](O)C1=CC=C([N+]([O-])=O)C=C1 WIIZWVCIJKGZOK-RKDXNWHRSA-N 0.000 description 2
- 239000003593 chromogenic compound Substances 0.000 description 2
- 238000012217 deletion Methods 0.000 description 2
- 230000037430 deletion Effects 0.000 description 2
- 238000011161 development Methods 0.000 description 2
- 230000018109 developmental process Effects 0.000 description 2
- 238000010586 diagram Methods 0.000 description 2
- 238000010494 dissociation reaction Methods 0.000 description 2
- 230000005593 dissociations Effects 0.000 description 2
- 230000003828 downregulation Effects 0.000 description 2
- 238000009585 enzyme analysis Methods 0.000 description 2
- 230000007062 hydrolysis Effects 0.000 description 2
- 238000006460 hydrolysis reaction Methods 0.000 description 2
- 206010022000 influenza Diseases 0.000 description 2
- 239000002502 liposome Substances 0.000 description 2
- 210000004962 mammalian cell Anatomy 0.000 description 2
- 239000002609 medium Substances 0.000 description 2
- 239000013642 negative control Substances 0.000 description 2
- 210000004492 nuclear pore Anatomy 0.000 description 2
- 238000003199 nucleic acid amplification method Methods 0.000 description 2
- 150000004713 phosphodiesters Chemical group 0.000 description 2
- 230000019491 signal transduction Effects 0.000 description 2
- 239000000243 solution Substances 0.000 description 2
- 238000010561 standard procedure Methods 0.000 description 2
- 239000006228 supernatant Substances 0.000 description 2
- 230000002459 sustained effect Effects 0.000 description 2
- 229960002180 tetracycline Drugs 0.000 description 2
- 229930101283 tetracycline Natural products 0.000 description 2
- 235000019364 tetracycline Nutrition 0.000 description 2
- 150000003522 tetracyclines Chemical class 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 102100026802 72 kDa type IV collagenase Human genes 0.000 description 1
- 239000004475 Arginine Substances 0.000 description 1
- 241000972773 Aulopiformes Species 0.000 description 1
- 241000894006 Bacteria Species 0.000 description 1
- 241000283690 Bos taurus Species 0.000 description 1
- 241000282472 Canis lupus familiaris Species 0.000 description 1
- 108010078791 Carrier Proteins Proteins 0.000 description 1
- 102000003727 Caveolin 1 Human genes 0.000 description 1
- 108090000026 Caveolin 1 Proteins 0.000 description 1
- 241000282693 Cercopithecidae Species 0.000 description 1
- 108020004705 Codon Proteins 0.000 description 1
- 101710081103 Cuticular glutathione peroxidase Proteins 0.000 description 1
- 102000009024 Epidermal Growth Factor Human genes 0.000 description 1
- 241000283086 Equidae Species 0.000 description 1
- 102000047351 Exportin-5 Human genes 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 108010001498 Galectin 1 Proteins 0.000 description 1
- 102100021736 Galectin-1 Human genes 0.000 description 1
- 101710114816 Gene 41 protein Proteins 0.000 description 1
- 102400000322 Glucagon-like peptide 1 Human genes 0.000 description 1
- DTHNMHAUYICORS-KTKZVXAJSA-N Glucagon-like peptide 1 Chemical compound C([C@@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](C)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CCCCN)C(=O)NCC(=O)N[C@@H](CCCNC(N)=N)C(N)=O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCCCN)NC(=O)[C@H](C)NC(=O)[C@H](C)NC(=O)[C@H](CCC(N)=O)NC(=O)CNC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@H](CO)NC(=O)[C@@H](NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CO)NC(=O)[C@@H](NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@@H](NC(=O)CNC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](C)NC(=O)[C@@H](N)CC=1N=CNC=1)[C@@H](C)O)[C@@H](C)O)C(C)C)C1=CC=CC=C1 DTHNMHAUYICORS-KTKZVXAJSA-N 0.000 description 1
- 101800000224 Glucagon-like peptide 1 Proteins 0.000 description 1
- 102000009465 Growth Factor Receptors Human genes 0.000 description 1
- 108010009202 Growth Factor Receptors Proteins 0.000 description 1
- 101710154606 Hemagglutinin Proteins 0.000 description 1
- 108091006054 His-tagged proteins Proteins 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101000627872 Homo sapiens 72 kDa type IV collagenase Proteins 0.000 description 1
- 101000718525 Homo sapiens Alpha-galactosidase A Proteins 0.000 description 1
- 101000847058 Homo sapiens Exportin-5 Proteins 0.000 description 1
- 101001075374 Homo sapiens Gamma-glutamyl hydrolase Proteins 0.000 description 1
- 101001059150 Homo sapiens Gelsolin Proteins 0.000 description 1
- 101000623901 Homo sapiens Mucin-16 Proteins 0.000 description 1
- 101000664737 Homo sapiens Somatotropin Proteins 0.000 description 1
- 108091006905 Human Serum Albumin Proteins 0.000 description 1
- 102000008100 Human Serum Albumin Human genes 0.000 description 1
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 description 1
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 description 1
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 description 1
- 108091026898 Leader sequence (mRNA) Proteins 0.000 description 1
- 239000000232 Lipid Bilayer Substances 0.000 description 1
- 206010025323 Lymphomas Diseases 0.000 description 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 description 1
- 239000004472 Lysine Substances 0.000 description 1
- 108010036176 Melitten Proteins 0.000 description 1
- 108090000301 Membrane transport proteins Proteins 0.000 description 1
- 102000003939 Membrane transport proteins Human genes 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 241000737052 Naso hexacanthus Species 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 108091034117 Oligonucleotide Proteins 0.000 description 1
- 101710093908 Outer capsid protein VP4 Proteins 0.000 description 1
- 101710135467 Outer capsid protein sigma-1 Proteins 0.000 description 1
- 238000012408 PCR amplification Methods 0.000 description 1
- 238000010222 PCR analysis Methods 0.000 description 1
- 239000002033 PVDF binder Substances 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 102000004160 Phosphoric Monoester Hydrolases Human genes 0.000 description 1
- 108090000608 Phosphoric Monoester Hydrolases Proteins 0.000 description 1
- 108010072866 Prostate-Specific Antigen Proteins 0.000 description 1
- 101710176177 Protein A56 Proteins 0.000 description 1
- 101710150344 Protein Rev Proteins 0.000 description 1
- 101710185720 Putative ethidium bromide resistance protein Proteins 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- 108020005091 Replication Origin Proteins 0.000 description 1
- 241001068295 Replication defective viruses Species 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 241000710961 Semliki Forest virus Species 0.000 description 1
- 108091027568 Single-stranded nucleotide Proteins 0.000 description 1
- 101100289792 Squirrel monkey polyomavirus large T gene Proteins 0.000 description 1
- 101710172711 Structural protein Proteins 0.000 description 1
- 102000006601 Thymidine Kinase Human genes 0.000 description 1
- 108020004440 Thymidine kinase Proteins 0.000 description 1
- 102000044209 Tumor Suppressor Genes Human genes 0.000 description 1
- 108700025716 Tumor Suppressor Genes Proteins 0.000 description 1
- 108090000848 Ubiquitin Proteins 0.000 description 1
- 102000044159 Ubiquitin Human genes 0.000 description 1
- 108700005077 Viral Genes Proteins 0.000 description 1
- 108020000999 Viral RNA Proteins 0.000 description 1
- 238000009825 accumulation Methods 0.000 description 1
- 230000009471 action Effects 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 230000008485 antagonism Effects 0.000 description 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 1
- 210000003719 b-lymphocyte Anatomy 0.000 description 1
- 108091008324 binding proteins Proteins 0.000 description 1
- 239000000872 buffer Substances 0.000 description 1
- 239000000969 carrier Substances 0.000 description 1
- 230000003915 cell function Effects 0.000 description 1
- 230000009087 cell motility Effects 0.000 description 1
- 108091092328 cellular RNA Proteins 0.000 description 1
- 230000007248 cellular mechanism Effects 0.000 description 1
- 230000002860 competitive effect Effects 0.000 description 1
- 239000002299 complementary DNA Substances 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 238000011109 contamination Methods 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 230000006735 deficit Effects 0.000 description 1
- 229940124447 delivery agent Drugs 0.000 description 1
- 238000001514 detection method Methods 0.000 description 1
- 229960000633 dextran sulfate Drugs 0.000 description 1
- 230000029087 digestion Effects 0.000 description 1
- 239000012636 effector Substances 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
- 210000003989 endothelium vascular Anatomy 0.000 description 1
- 238000013401 experimental design Methods 0.000 description 1
- 238000002474 experimental method Methods 0.000 description 1
- 238000010195 expression analysis Methods 0.000 description 1
- 239000013613 expression plasmid Substances 0.000 description 1
- 210000001723 extracellular space Anatomy 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- 238000010363 gene targeting Methods 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 239000001963 growth medium Substances 0.000 description 1
- 238000011534 incubation Methods 0.000 description 1
- 229910052738 indium Inorganic materials 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- 238000003780 insertion Methods 0.000 description 1
- 230000037431 insertion Effects 0.000 description 1
- 230000010354 integration Effects 0.000 description 1
- 230000003834 intracellular effect Effects 0.000 description 1
- 238000002955 isolation Methods 0.000 description 1
- 230000004807 localization Effects 0.000 description 1
- 239000003550 marker Substances 0.000 description 1
- VDXZNPDIRNWWCW-JFTDCZMZSA-N melittin Chemical compound NCC(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](C)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H]([C@@H](C)O)C(=O)NCC(=O)N[C@@H](CC(C)C)C(=O)N1CCC[C@H]1C(=O)N[C@@H](C)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CO)C(=O)N[C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](CCC(N)=O)C(N)=O)CC1=CNC2=CC=CC=C12 VDXZNPDIRNWWCW-JFTDCZMZSA-N 0.000 description 1
- 230000000051 modifying effect Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 230000009871 nonspecific binding Effects 0.000 description 1
- 230000030147 nuclear export Effects 0.000 description 1
- 229920002704 polyhistidine Polymers 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 229920002981 polyvinylidene fluoride Polymers 0.000 description 1
- 230000000861 pro-apoptotic effect Effects 0.000 description 1
- 230000008569 process Effects 0.000 description 1
- 230000001737 promoting effect Effects 0.000 description 1
- 235000004252 protein component Nutrition 0.000 description 1
- 108020001580 protein domains Proteins 0.000 description 1
- 230000004850 protein–protein interaction Effects 0.000 description 1
- 238000000746 purification Methods 0.000 description 1
- 230000014493 regulation of gene expression Effects 0.000 description 1
- 230000008844 regulatory mechanism Effects 0.000 description 1
- 230000003362 replicative effect Effects 0.000 description 1
- 230000002441 reversible effect Effects 0.000 description 1
- 235000019515 salmon Nutrition 0.000 description 1
- 230000009962 secretion pathway Effects 0.000 description 1
- 239000001509 sodium citrate Substances 0.000 description 1
- NLJMYIDDQXHKNR-UHFFFAOYSA-K sodium citrate Chemical compound O.O.[Na+].[Na+].[Na+].[O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O NLJMYIDDQXHKNR-UHFFFAOYSA-K 0.000 description 1
- 239000001488 sodium phosphate Substances 0.000 description 1
- 229910000162 sodium phosphate Inorganic materials 0.000 description 1
- 239000007858 starting material Substances 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 239000012134 supernatant fraction Substances 0.000 description 1
- 229920001897 terpolymer Polymers 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 238000010361 transduction Methods 0.000 description 1
- 230000026683 transduction Effects 0.000 description 1
- 238000003146 transient transfection Methods 0.000 description 1
- 230000014621 translational initiation Effects 0.000 description 1
- 230000005945 translocation Effects 0.000 description 1
- 230000007723 transport mechanism Effects 0.000 description 1
- RYFMWSXOAZQYPI-UHFFFAOYSA-K trisodium phosphate Chemical compound [Na+].[Na+].[Na+].[O-]P([O-])([O-])=O RYFMWSXOAZQYPI-UHFFFAOYSA-K 0.000 description 1
- 230000002792 vascular Effects 0.000 description 1
- 230000007332 vesicle formation Effects 0.000 description 1
- 230000007484 viral process Effects 0.000 description 1
- 238000001262 western blot Methods 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
Landscapes
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US16028809P | 2009-03-13 | 2009-03-13 | |
| US16028709P | 2009-03-13 | 2009-03-13 | |
| US61/160,288 | 2009-03-13 | ||
| US61/160,287 | 2009-03-13 | ||
| PCT/US2010/027365 WO2010105277A1 (en) | 2009-03-13 | 2010-03-15 | Compositions and methods for the delivery of biologically active rnas |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2012520085A true JP2012520085A (ja) | 2012-09-06 |
| JP2012520085A5 JP2012520085A5 (enExample) | 2013-02-21 |
Family
ID=42728852
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011554275A Pending JP2012520085A (ja) | 2009-03-13 | 2010-03-15 | 生物活性ｒｎａの送達のための組成物及び方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US8569065B2 (enExample) |
| EP (1) | EP2406379A4 (enExample) |
| JP (1) | JP2012520085A (enExample) |
| KR (1) | KR20110128345A (enExample) |
| CN (1) | CN102625842A (enExample) |
| AU (1) | AU2010223888A1 (enExample) |
| CA (1) | CA2755245A1 (enExample) |
| WO (1) | WO2010105277A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2017505638A (ja) * | 2014-02-14 | 2017-02-23 | シンヴィヴォ コーポレイション | ハイブリッドタンパクおよびその使用 |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2525780B1 (en) | 2010-01-19 | 2022-09-07 | Northwestern University | Synthetic nanostructures including nucleic acids and/or other entities |
| US20130101512A1 (en) * | 2010-03-12 | 2013-04-25 | Chad A. Mirkin | Crosslinked polynucleotide structure |
| WO2011130749A2 (en) | 2010-04-16 | 2011-10-20 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Identification of mutations in herpes simplex virus envelope glycoproteins that enable or enhance vector retargeting to novel non-hsv receptors |
| CA2807552A1 (en) | 2010-08-06 | 2012-02-09 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| EP2857499A1 (en) | 2010-10-01 | 2015-04-08 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| CA2831613A1 (en) | 2011-03-31 | 2012-10-04 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| WO2012159164A1 (en) * | 2011-05-23 | 2012-11-29 | Phylogica Limited | Method of determining, identifying or isolating cell-penetrating peptides |
| US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| PT3682905T (pt) | 2011-10-03 | 2022-04-07 | Modernatx Inc | Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações |
| ES2923757T3 (es) | 2011-12-16 | 2022-09-30 | Modernatx Inc | Composiciones de ARNm modificado |
| WO2013096958A1 (en) * | 2011-12-23 | 2013-06-27 | Egen, Inc. | Compositions and methods for the delivery of biologically active rnas |
| US20140371302A1 (en) * | 2011-12-29 | 2014-12-18 | Modema Therapeutics, Inc. | Modified mrnas encoding cell-penetrating polypeptides |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
| US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| PT2922554T (pt) | 2012-11-26 | 2022-06-28 | Modernatx Inc | Arn modificado nas porções terminais |
| WO2014144423A2 (en) * | 2013-03-15 | 2014-09-18 | Techulon Inc. | Antisense molecules for treatment of staphylococcus aureus infection |
| US8980864B2 (en) | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| WO2015002956A1 (en) * | 2013-07-01 | 2015-01-08 | Ohio State Innovation Foundation | Exosome delivery system |
| CA2919268C (en) | 2013-07-25 | 2023-09-05 | Exicure, Inc. | Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use |
| US10568898B2 (en) | 2013-08-13 | 2020-02-25 | Northwestern University | Lipophilic nanoparticles for drug delivery |
| US10023626B2 (en) | 2013-09-30 | 2018-07-17 | Modernatx, Inc. | Polynucleotides encoding immune modulating polypeptides |
| KR20160067219A (ko) | 2013-10-03 | 2016-06-13 | 모더나 세라퓨틱스, 인코포레이티드 | 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드 |
| DK3184641T3 (da) | 2013-10-28 | 2020-08-03 | Univ Of Pittsburgh - Of The Commonwealth System Of Higher Education | Onkolytisk hsv-vektor |
| AU2015269412B2 (en) | 2014-06-04 | 2020-03-12 | Exicure Operating Company | Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications |
| EP3183007B1 (en) | 2014-08-19 | 2020-06-17 | Northwestern University | Protein/oligonucleotide core-shell nanoparticle therapeutics |
| BR112017007012A2 (pt) | 2014-10-06 | 2018-03-06 | Exicure Inc | compostos anti-tnf |
| US9816080B2 (en) * | 2014-10-31 | 2017-11-14 | President And Fellows Of Harvard College | Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs) |
| US10078092B2 (en) | 2015-03-18 | 2018-09-18 | Northwestern University | Assays for measuring binding kinetics and binding capacity of acceptors for lipophilic or amphiphilic molecules |
| AU2017212713B2 (en) | 2016-01-27 | 2023-01-19 | Oncorus, Inc. | Oncolytic viral vectors and uses thereof |
| US11639371B2 (en) * | 2016-02-17 | 2023-05-02 | The Chinese University Of Hong Kong | Peptidylic inhibitors of nucleolin (NCL) targeting CAG-repeat RNA toxicity and methods for reducing polyQ-mediated toxicity in polyQ diseases |
| JP2019515654A (ja) | 2016-03-16 | 2019-06-13 | ザ ジェイ． デヴィッド グラッドストーン インスティテューツ | 肥満及び／又は糖尿病を処置するための方法及び組成物、並びに候補処置薬剤を識別するための方法及び組成物 |
| EP3478321A4 (en) | 2016-06-30 | 2020-04-22 | Oncorus, Inc. | PSEUDOTYPIZED ONCOLYTIC VIRAL ADMINISTRATION OF THERAPEUTIC POLYPEPTIDES |
| MX2020000993A (es) | 2017-07-26 | 2020-07-22 | Oncorus Inc | Vectores virales oncolíticos y usos de estos. |
| WO2019133847A1 (en) | 2017-12-29 | 2019-07-04 | Oncorus, Inc. | Oncolytic viral delivery of therapeutic polypeptides |
| US20210139892A1 (en) * | 2018-07-05 | 2021-05-13 | The Regents Of The University Of California | Compositions and methods for delivery of rna to a cell |
| EP4034088A4 (en) | 2019-09-26 | 2023-10-11 | President and Fellows of Harvard College | Minimal arrestin domain containing protein 1 (arrdc1) constructs |
| WO2023244760A1 (en) * | 2022-06-15 | 2023-12-21 | Duke University | Protein library display systems and methods thereof |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2000039311A1 (en) * | 1998-12-31 | 2000-07-06 | Advanced Research And Technology Institute | Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2 |
| WO2003060101A2 (en) * | 2002-01-11 | 2003-07-24 | University Of Southern California | Gene delivery system and methods of use |
| WO2004041852A2 (en) * | 2002-11-05 | 2004-05-21 | Glaxo Group Limited | Hiv vaccine |
| WO2008098933A1 (en) * | 2007-02-15 | 2008-08-21 | Dsm Ip Assets B.V. | A recombinant host cell for the production of a compound of interest |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5585263A (en) | 1994-05-20 | 1996-12-17 | University Of Alabama At Birmingham Research Foundation | Purified retroviral constitutive transport enhancer and its use to facilitate mRNA transport, and to produce recombinant, attenuated HIV |
| US6451592B1 (en) * | 1996-04-05 | 2002-09-17 | Chiron Corporation | Recombinant alphavirus-based vectors with reduced inhibition of cellular macromolecular synthesis |
| US20040241845A1 (en) * | 1998-05-22 | 2004-12-02 | Luc Desgroseillers | Mammalian staufen and use thereof |
| AU2001294872A1 (en) | 2000-09-28 | 2002-04-08 | Cellomics, Inc. | Methods and reagents for live-cell gene expression quantification |
| CU23225A1 (es) * | 2001-12-20 | 2007-08-30 | Ct Ingenieria Genetica Biotech | PéPTIDOS PARA EL TRATAMIENTO DEL CáNCER ASOCIADO AL VIRUS PAPILOMA HUMANO (VPH) Y DE OTROS TUMORES EPITELIALES |
| AU2003228301A1 (en) * | 2002-03-06 | 2003-09-22 | Rigel Pharmaceuticals, Inc. | Novel method for delivery and intracellular synthesis of sirna molecules |
| US20050074865A1 (en) | 2002-08-27 | 2005-04-07 | Compound Therapeutics, Inc. | Adzymes and uses thereof |
| EP1658304A4 (en) | 2002-09-28 | 2009-01-14 | Massachusetts Inst Technology | THERAPY ANTIGRIPPALE |
| US20040147027A1 (en) * | 2003-01-28 | 2004-07-29 | Troy Carol M. | Complex for facilitating delivery of dsRNA into a cell and uses thereof |
| US20070036773A1 (en) | 2005-08-09 | 2007-02-15 | City Of Hope | Generation and application of universal T cells for B-ALL |
| WO2007092222A2 (en) * | 2006-02-02 | 2007-08-16 | Regenetech, Inc. | Method of characterizing a biologically active compound |
| JP2009532017A (ja) * | 2006-02-10 | 2009-09-10 | ザ レジェンツ オブ ザ ユニヴァースティ オブ カリフォルニア | ＰＴＤ／ＣＰＰＳへのｄｓＲＮＡ結合ドメイン融合体によるｓｉＲＮＡの導入可能な送達 |
| US9085778B2 (en) | 2006-05-03 | 2015-07-21 | VL27, Inc. | Exosome transfer of nucleic acids to cells |
| CN100549179C (zh) * | 2006-08-07 | 2009-10-14 | 汪运山 | 胃癌靶向AFP基因的siRNAs表达载体及其用途 |
| PH12012501596A1 (en) | 2010-02-24 | 2017-02-03 | Arrowhead Res Corporation | Compositions for targeted delivery of sirna |
-
2010
- 2010-03-15 CA CA2755245A patent/CA2755245A1/en not_active Abandoned
- 2010-03-15 JP JP2011554275A patent/JP2012520085A/ja active Pending
- 2010-03-15 KR KR1020117024110A patent/KR20110128345A/ko not_active Ceased
- 2010-03-15 AU AU2010223888A patent/AU2010223888A1/en not_active Abandoned
- 2010-03-15 EP EP10751551A patent/EP2406379A4/en not_active Withdrawn
- 2010-03-15 WO PCT/US2010/027365 patent/WO2010105277A1/en not_active Ceased
- 2010-03-15 CN CN2010800210226A patent/CN102625842A/zh active Pending
- 2010-03-15 US US12/724,408 patent/US8569065B2/en active Active
-
2013
- 2013-03-11 US US13/793,961 patent/US20140005254A1/en not_active Abandoned
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2000039311A1 (en) * | 1998-12-31 | 2000-07-06 | Advanced Research And Technology Institute | Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2 |
| WO2003060101A2 (en) * | 2002-01-11 | 2003-07-24 | University Of Southern California | Gene delivery system and methods of use |
| WO2004041852A2 (en) * | 2002-11-05 | 2004-05-21 | Glaxo Group Limited | Hiv vaccine |
| WO2008098933A1 (en) * | 2007-02-15 | 2008-08-21 | Dsm Ip Assets B.V. | A recombinant host cell for the production of a compound of interest |
Non-Patent Citations (4)
| Title |
|---|
| BIOCONJUGATE CHEM., 2008, VOL.19, P.1017-1024, JPN6014026576, ISSN: 0002841311 * |
| MOLECULAR THERAPY, 2005, VOL.12, NO.5, P.842-851, JPN6014026570, ISSN: 0002841309 * |
| NATURE BIOTECHNOLOGY, 2006, VOL.24, NO.6, P.697-702, JPN6014026573, ISSN: 0002841310 * |
| ウイルス, 2002, VOL.52, NO.1, P.89-94, JPN6014026578, ISSN: 0002841312 * |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2017505638A (ja) * | 2014-02-14 | 2017-02-23 | シンヴィヴォ コーポレイション | ハイブリッドタンパクおよびその使用 |
Also Published As
| Publication number | Publication date |
|---|---|
| US8569065B2 (en) | 2013-10-29 |
| CA2755245A1 (en) | 2010-09-16 |
| KR20110128345A (ko) | 2011-11-29 |
| EP2406379A4 (en) | 2012-09-26 |
| WO2010105277A1 (en) | 2010-09-16 |
| US20140005254A1 (en) | 2014-01-02 |
| CN102625842A (zh) | 2012-08-01 |
| EP2406379A1 (en) | 2012-01-18 |
| US20100233141A1 (en) | 2010-09-16 |
| AU2010223888A1 (en) | 2011-10-06 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US8569065B2 (en) | Compositions and methods for the delivery of biologically active RNAs | |
| JP7820434B2 (ja) | アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 | |
| KR102957630B1 (ko) | 핵산 구조체 및 사용 방법 | |
| JP2015507474A (ja) | 生物活性ｒｎａの送達のための組成物及び方法 | |
| US12264331B2 (en) | Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA | |
| JP2021500863A (ja) | ゲノム編集用のポリヌクレオチド、組成物および方法 | |
| JP6608284B2 (ja) | 癌の治療に使用されるｒｎａトランススプライシング分子（ｒｔｍ） | |
| KR20170116055A (ko) | 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도 | |
| US20110207226A1 (en) | Non-integrating lenti/adeno-associated virus hybrid vector system | |
| US20200172913A1 (en) | Peptides and nanoparticles for intracellular delivery of virus | |
| WO2015002956A1 (en) | Exosome delivery system | |
| CN118632622A (zh) | 突变型肌纤蛋白疾病模型及其用途 | |
| US12584145B2 (en) | Conjugate systems and methods of use thereof | |
| CN121464217A (zh) | 用于治疗KIF1A相关神经紊乱的靶向KIF1A错义突变的RNAi | |
| WO2023147558A2 (en) | Crispr methods for correcting bag3 gene mutations in vivo | |
| US20250163474A1 (en) | Systems, methods, and compositions for targeted gene manipulation and uses thereof | |
| US20250135032A1 (en) | Crispr methods for correcting bag3 gene mutations in vivo | |
| US20260083861A1 (en) | Crispr-related methods and compositions targeting cd70 expression | |
| KR20260046119A (ko) | 단방향 유전자 삽입 동안 도입유전자 발현을 증진시키는 bGH-SV40L 직렬 PolyA의 용도 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20130104 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20130104 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20140627 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20140925 |
|
| A602 | Written permission of extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A602 Effective date: 20141002 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20141226 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20150507 |