JP2013507934A - 遺伝子療法適用のためのスーパーコイル状ミニサークルｄｎａ - Google Patents
遺伝子療法適用のためのスーパーコイル状ミニサークルｄｎａ Download PDFInfo
- Publication number
- JP2013507934A JP2013507934A JP2012534410A JP2012534410A JP2013507934A JP 2013507934 A JP2013507934 A JP 2013507934A JP 2012534410 A JP2012534410 A JP 2012534410A JP 2012534410 A JP2012534410 A JP 2012534410A JP 2013507934 A JP2013507934 A JP 2013507934A
- Authority
- JP
- Japan
- Prior art keywords
- minivector
- nucleic acid
- acid sequence
- dna
- gene
- 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
- 238000001415 gene therapy Methods 0.000 title claims abstract description 33
- 108091028043 Nucleic acid sequence Proteins 0.000 claims abstract description 104
- 150000007523 nucleic acids Chemical class 0.000 claims abstract description 82
- 238000000034 method Methods 0.000 claims abstract description 76
- 239000000203 mixture Substances 0.000 claims abstract description 10
- 108020004707 nucleic acids Proteins 0.000 claims abstract description 7
- 102000039446 nucleic acids Human genes 0.000 claims abstract description 7
- 108020004414 DNA Proteins 0.000 claims description 184
- 210000004027 cell Anatomy 0.000 claims description 131
- 239000004055 small Interfering RNA Substances 0.000 claims description 123
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 88
- 108090000623 proteins and genes Proteins 0.000 claims description 75
- 230000014509 gene expression Effects 0.000 claims description 53
- 230000030279 gene silencing Effects 0.000 claims description 52
- 239000002679 microRNA Substances 0.000 claims description 38
- 108700011259 MicroRNAs Proteins 0.000 claims description 29
- 102000004169 proteins and genes Human genes 0.000 claims description 22
- 206010025323 Lymphomas Diseases 0.000 claims description 18
- 239000000126 substance Substances 0.000 claims description 16
- 206010073478 Anaplastic large-cell lymphoma Diseases 0.000 claims description 15
- 208000032004 Large-Cell Anaplastic Lymphoma Diseases 0.000 claims description 15
- 241000124008 Mammalia Species 0.000 claims description 14
- 210000000170 cell membrane Anatomy 0.000 claims description 14
- 210000004962 mammalian cell Anatomy 0.000 claims description 14
- 108091070501 miRNA Proteins 0.000 claims description 9
- 239000007921 spray Substances 0.000 claims description 9
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical compound N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 claims description 6
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 6
- 206010028980 Neoplasm Diseases 0.000 claims description 5
- 208000015914 Non-Hodgkin lymphomas Diseases 0.000 claims description 5
- 108091034117 Oligonucleotide Proteins 0.000 claims description 4
- 201000011510 cancer Diseases 0.000 claims description 4
- 210000005260 human cell Anatomy 0.000 claims description 4
- 210000002345 respiratory system Anatomy 0.000 claims description 4
- 239000011616 biotin Substances 0.000 claims description 3
- 229960002685 biotin Drugs 0.000 claims description 3
- 235000020958 biotin Nutrition 0.000 claims description 3
- 235000012000 cholesterol Nutrition 0.000 claims description 3
- GNBHRKFJIUUOQI-UHFFFAOYSA-N fluorescein Chemical compound O1C(=O)C2=CC=CC=C2C21C1=CC=C(O)C=C1OC1=CC(O)=CC=C21 GNBHRKFJIUUOQI-UHFFFAOYSA-N 0.000 claims description 3
- 210000002850 nasal mucosa Anatomy 0.000 claims description 2
- 108091000080 Phosphotransferase Proteins 0.000 claims 2
- 239000013598 vector Substances 0.000 description 57
- 239000013612 plasmid Substances 0.000 description 52
- 108020004459 Small interfering RNA Proteins 0.000 description 41
- 238000001890 transfection Methods 0.000 description 39
- 101710168331 ALK tyrosine kinase receptor Proteins 0.000 description 25
- 238000012226 gene silencing method Methods 0.000 description 24
- 102100033793 ALK tyrosine kinase receptor Human genes 0.000 description 23
- 230000004083 survival effect Effects 0.000 description 23
- 239000013600 plasmid vector Substances 0.000 description 21
- 230000000694 effects Effects 0.000 description 19
- 239000000443 aerosol Substances 0.000 description 18
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 18
- 230000001225 therapeutic effect Effects 0.000 description 18
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 17
- 239000006199 nebulizer Substances 0.000 description 17
- 230000001413 cellular effect Effects 0.000 description 16
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 description 15
- 230000009368 gene silencing by RNA Effects 0.000 description 15
- 238000001727 in vivo Methods 0.000 description 15
- 210000004072 lung Anatomy 0.000 description 13
- 238000000527 sonication Methods 0.000 description 13
- 230000015556 catabolic process Effects 0.000 description 12
- 238000006731 degradation reaction Methods 0.000 description 12
- 201000010099 disease Diseases 0.000 description 12
- 230000001404 mediated effect Effects 0.000 description 12
- 108060001084 Luciferase Proteins 0.000 description 11
- 210000001744 T-lymphocyte Anatomy 0.000 description 11
- 230000001580 bacterial effect Effects 0.000 description 11
- 210000004443 dendritic cell Anatomy 0.000 description 11
- 230000009467 reduction Effects 0.000 description 11
- 238000010008 shearing Methods 0.000 description 11
- 101150023956 ALK gene Proteins 0.000 description 10
- 239000012634 fragment Substances 0.000 description 10
- 239000000047 product Substances 0.000 description 10
- 230000006798 recombination Effects 0.000 description 10
- 238000005215 recombination Methods 0.000 description 10
- 239000005089 Luciferase Substances 0.000 description 9
- 238000002474 experimental method Methods 0.000 description 9
- 238000000684 flow cytometry Methods 0.000 description 9
- 238000002560 therapeutic procedure Methods 0.000 description 9
- 241000699670 Mus sp. Species 0.000 description 8
- 230000015572 biosynthetic process Effects 0.000 description 8
- 239000000523 sample Substances 0.000 description 8
- 238000005507 spraying Methods 0.000 description 8
- 101150066002 GFP gene Proteins 0.000 description 7
- 241000963438 Gaussia <copepod> Species 0.000 description 7
- 108020001507 fusion proteins Proteins 0.000 description 7
- 102000037865 fusion proteins Human genes 0.000 description 7
- 210000002966 serum Anatomy 0.000 description 7
- 238000011282 treatment Methods 0.000 description 7
- 108020004638 Circular DNA Proteins 0.000 description 6
- 229920002873 Polyethylenimine Polymers 0.000 description 6
- 208000035475 disorder Diseases 0.000 description 6
- 230000008569 process Effects 0.000 description 6
- 108700019146 Transgenes Proteins 0.000 description 5
- 238000012387 aerosolization Methods 0.000 description 5
- 230000008901 benefit Effects 0.000 description 5
- 230000004663 cell proliferation Effects 0.000 description 5
- 229940079593 drug Drugs 0.000 description 5
- 239000003814 drug Substances 0.000 description 5
- 230000002452 interceptive effect Effects 0.000 description 5
- 108020004999 messenger RNA Proteins 0.000 description 5
- 238000002663 nebulization Methods 0.000 description 5
- 230000008685 targeting Effects 0.000 description 5
- 241000283690 Bos taurus Species 0.000 description 4
- HEDRZPFGACZZDS-UHFFFAOYSA-N Chloroform Chemical compound ClC(Cl)Cl HEDRZPFGACZZDS-UHFFFAOYSA-N 0.000 description 4
- 241000283086 Equidae Species 0.000 description 4
- 102100034343 Integrase Human genes 0.000 description 4
- 108010061833 Integrases Proteins 0.000 description 4
- 241001494479 Pecora Species 0.000 description 4
- 230000001464 adherent effect Effects 0.000 description 4
- 230000003115 biocidal effect Effects 0.000 description 4
- 230000010261 cell growth Effects 0.000 description 4
- 238000001516 cell proliferation assay Methods 0.000 description 4
- 230000008859 change Effects 0.000 description 4
- 239000003795 chemical substances by application Substances 0.000 description 4
- 230000007423 decrease Effects 0.000 description 4
- 239000000975 dye Substances 0.000 description 4
- ZMMJGEGLRURXTF-UHFFFAOYSA-N ethidium bromide Chemical compound [Br-].C12=CC(N)=CC=C2C2=CC=C(N)C=C2[N+](CC)=C1C1=CC=CC=C1 ZMMJGEGLRURXTF-UHFFFAOYSA-N 0.000 description 4
- 229960005542 ethidium bromide Drugs 0.000 description 4
- MHMNJMPURVTYEJ-UHFFFAOYSA-N fluorescein-5-isothiocyanate Chemical compound O1C(=O)C2=CC(N=C=S)=CC=C2C21C1=CC=C(O)C=C1OC1=CC(O)=CC=C21 MHMNJMPURVTYEJ-UHFFFAOYSA-N 0.000 description 4
- 239000000499 gel Substances 0.000 description 4
- 238000010363 gene targeting Methods 0.000 description 4
- 230000012010 growth Effects 0.000 description 4
- 239000000463 material Substances 0.000 description 4
- 239000002773 nucleotide Substances 0.000 description 4
- 125000003729 nucleotide group Chemical group 0.000 description 4
- 230000010076 replication Effects 0.000 description 4
- HRPVXLWXLXDGHG-UHFFFAOYSA-N Acrylamide Chemical compound NC(=O)C=C HRPVXLWXLXDGHG-UHFFFAOYSA-N 0.000 description 3
- 108020000946 Bacterial DNA Proteins 0.000 description 3
- 102000053602 DNA Human genes 0.000 description 3
- 241000588724 Escherichia coli Species 0.000 description 3
- 241000282412 Homo Species 0.000 description 3
- 241000699666 Mus <mouse, genus> Species 0.000 description 3
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 3
- 230000002159 abnormal effect Effects 0.000 description 3
- 230000006907 apoptotic process Effects 0.000 description 3
- 238000013459 approach Methods 0.000 description 3
- 238000003556 assay Methods 0.000 description 3
- 230000000875 corresponding effect Effects 0.000 description 3
- 231100000433 cytotoxic Toxicity 0.000 description 3
- 230000001472 cytotoxic effect Effects 0.000 description 3
- 230000028993 immune response Effects 0.000 description 3
- 208000032839 leukemia Diseases 0.000 description 3
- 230000007246 mechanism Effects 0.000 description 3
- 230000004048 modification Effects 0.000 description 3
- 238000012986 modification Methods 0.000 description 3
- 210000000056 organ Anatomy 0.000 description 3
- 230000037361 pathway Effects 0.000 description 3
- 230000032361 posttranscriptional gene silencing Effects 0.000 description 3
- 238000002360 preparation method Methods 0.000 description 3
- 231100000331 toxic Toxicity 0.000 description 3
- 230000002588 toxic effect Effects 0.000 description 3
- 239000013603 viral vector Substances 0.000 description 3
- 230000003612 virological effect Effects 0.000 description 3
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 2
- 241000282465 Canis Species 0.000 description 2
- 241000282472 Canis lupus familiaris Species 0.000 description 2
- 241000283707 Capra Species 0.000 description 2
- 241000700198 Cavia Species 0.000 description 2
- 102000012410 DNA Ligases Human genes 0.000 description 2
- 108010061982 DNA Ligases Proteins 0.000 description 2
- 230000033616 DNA repair Effects 0.000 description 2
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 description 2
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 description 2
- KCXVZYZYPLLWCC-UHFFFAOYSA-N EDTA Chemical compound OC(=O)CN(CC(O)=O)CCN(CC(O)=O)CC(O)=O KCXVZYZYPLLWCC-UHFFFAOYSA-N 0.000 description 2
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 2
- 241000282324 Felis Species 0.000 description 2
- 241000282326 Felis catus Species 0.000 description 2
- 101000907904 Homo sapiens Endoribonuclease Dicer Proteins 0.000 description 2
- 239000012097 Lipofectamine 2000 Substances 0.000 description 2
- 238000000134 MTT assay Methods 0.000 description 2
- 231100000002 MTT assay Toxicity 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 2
- LRHPLDYGYMQRHN-UHFFFAOYSA-N N-Butanol Chemical compound CCCCO LRHPLDYGYMQRHN-UHFFFAOYSA-N 0.000 description 2
- 101710147059 Nicking endonuclease Proteins 0.000 description 2
- 241000283973 Oryctolagus cuniculus Species 0.000 description 2
- 241000288906 Primates Species 0.000 description 2
- 241000700159 Rattus Species 0.000 description 2
- 241000283984 Rodentia Species 0.000 description 2
- 241000282887 Suidae Species 0.000 description 2
- 239000011543 agarose gel Substances 0.000 description 2
- 230000000692 anti-sense effect Effects 0.000 description 2
- 239000012620 biological material Substances 0.000 description 2
- 125000002091 cationic group Chemical group 0.000 description 2
- 230000000295 complement effect Effects 0.000 description 2
- 231100000135 cytotoxicity Toxicity 0.000 description 2
- 230000003013 cytotoxicity Effects 0.000 description 2
- 239000007857 degradation product Substances 0.000 description 2
- 238000002716 delivery method Methods 0.000 description 2
- 238000005516 engineering process Methods 0.000 description 2
- 210000002950 fibroblast Anatomy 0.000 description 2
- 238000000799 fluorescence microscopy Methods 0.000 description 2
- 238000001502 gel electrophoresis Methods 0.000 description 2
- PCHJSUWPFVWCPO-UHFFFAOYSA-N gold Chemical compound [Au] PCHJSUWPFVWCPO-UHFFFAOYSA-N 0.000 description 2
- 239000010931 gold Substances 0.000 description 2
- 229910052737 gold Inorganic materials 0.000 description 2
- 230000006872 improvement Effects 0.000 description 2
- 230000001965 increasing effect Effects 0.000 description 2
- 230000001939 inductive effect Effects 0.000 description 2
- 230000005764 inhibitory process Effects 0.000 description 2
- PHTQWCKDNZKARW-UHFFFAOYSA-N isoamylol Chemical compound CC(C)CCO PHTQWCKDNZKARW-UHFFFAOYSA-N 0.000 description 2
- 210000003292 kidney cell Anatomy 0.000 description 2
- 238000002372 labelling Methods 0.000 description 2
- 230000007774 longterm Effects 0.000 description 2
- 238000009126 molecular therapy Methods 0.000 description 2
- 239000013642 negative control Substances 0.000 description 2
- 229920003023 plastic Polymers 0.000 description 2
- 229920002401 polyacrylamide Polymers 0.000 description 2
- 230000002829 reductive effect Effects 0.000 description 2
- 230000008439 repair process Effects 0.000 description 2
- 230000004044 response Effects 0.000 description 2
- PYWVYCXTNDRMGF-UHFFFAOYSA-N rhodamine B Chemical compound [Cl-].C=12C=CC(=[N+](CC)CC)C=C2OC2=CC(N(CC)CC)=CC=C2C=1C1=CC=CC=C1C(O)=O PYWVYCXTNDRMGF-UHFFFAOYSA-N 0.000 description 2
- 239000000243 solution Substances 0.000 description 2
- 238000010186 staining Methods 0.000 description 2
- 239000000725 suspension Substances 0.000 description 2
- 238000007910 systemic administration Methods 0.000 description 2
- 210000001519 tissue Anatomy 0.000 description 2
- 231100000419 toxicity Toxicity 0.000 description 2
- 230000001988 toxicity Effects 0.000 description 2
- 239000012096 transfection reagent Substances 0.000 description 2
- 230000007923 virulence factor Effects 0.000 description 2
- 239000000304 virulence factor Substances 0.000 description 2
- VGIRNWJSIRVFRT-UHFFFAOYSA-N 2',7'-difluorofluorescein Chemical compound OC(=O)C1=CC=CC=C1C1=C2C=C(F)C(=O)C=C2OC2=CC(O)=C(F)C=C21 VGIRNWJSIRVFRT-UHFFFAOYSA-N 0.000 description 1
- QEQDLKUMPUDNPG-UHFFFAOYSA-N 2-(7-amino-4-methyl-2-oxochromen-3-yl)acetic acid Chemical compound C1=C(N)C=CC2=C1OC(=O)C(CC(O)=O)=C2C QEQDLKUMPUDNPG-UHFFFAOYSA-N 0.000 description 1
- QKNYBSVHEMOAJP-UHFFFAOYSA-N 2-amino-2-(hydroxymethyl)propane-1,3-diol;hydron;chloride Chemical compound Cl.OCC(N)(CO)CO QKNYBSVHEMOAJP-UHFFFAOYSA-N 0.000 description 1
- MWBWWFOAEOYUST-UHFFFAOYSA-N 2-aminopurine Chemical compound NC1=NC=C2N=CNC2=N1 MWBWWFOAEOYUST-UHFFFAOYSA-N 0.000 description 1
- FWBHETKCLVMNFS-UHFFFAOYSA-N 4',6-Diamino-2-phenylindol Chemical compound C1=CC(C(=N)N)=CC=C1C1=CC2=CC=C(C(N)=N)C=C2N1 FWBHETKCLVMNFS-UHFFFAOYSA-N 0.000 description 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 1
- COCMHKNAGZHBDZ-UHFFFAOYSA-N 4-carboxy-3-[3-(dimethylamino)-6-dimethylazaniumylidenexanthen-9-yl]benzoate Chemical compound C=12C=CC(=[N+](C)C)C=C2OC2=CC(N(C)C)=CC=C2C=1C1=CC(C([O-])=O)=CC=C1C(O)=O COCMHKNAGZHBDZ-UHFFFAOYSA-N 0.000 description 1
- YMZMTOFQCVHHFB-UHFFFAOYSA-N 5-carboxytetramethylrhodamine Chemical compound C=12C=CC(N(C)C)=CC2=[O+]C2=CC(N(C)C)=CC=C2C=1C1=CC=C(C(O)=O)C=C1C([O-])=O YMZMTOFQCVHHFB-UHFFFAOYSA-N 0.000 description 1
- BZTDTCNHAFUJOG-UHFFFAOYSA-N 6-carboxyfluorescein Chemical compound C12=CC=C(O)C=C2OC2=CC(O)=CC=C2C11OC(=O)C2=CC=C(C(=O)O)C=C21 BZTDTCNHAFUJOG-UHFFFAOYSA-N 0.000 description 1
- 229920000936 Agarose Polymers 0.000 description 1
- 241000203069 Archaea Species 0.000 description 1
- 241000894006 Bacteria Species 0.000 description 1
- 108091032955 Bacterial small RNA Proteins 0.000 description 1
- 102100035875 C-C chemokine receptor type 5 Human genes 0.000 description 1
- 101710149870 C-C chemokine receptor type 5 Proteins 0.000 description 1
- 206010008025 Cerebellar ataxia Diseases 0.000 description 1
- 201000003883 Cystic fibrosis Diseases 0.000 description 1
- 102000003915 DNA Topoisomerases Human genes 0.000 description 1
- 108090000323 DNA Topoisomerases Proteins 0.000 description 1
- 238000007399 DNA isolation Methods 0.000 description 1
- 102000004163 DNA-directed RNA polymerases Human genes 0.000 description 1
- 108090000626 DNA-directed RNA polymerases Proteins 0.000 description 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 1
- 238000002965 ELISA Methods 0.000 description 1
- 102100023387 Endoribonuclease Dicer Human genes 0.000 description 1
- 241000620209 Escherichia coli DH5[alpha] Species 0.000 description 1
- 241000206602 Eukaryota Species 0.000 description 1
- 108060002716 Exonuclease Proteins 0.000 description 1
- 208000034951 Genetic Translocation Diseases 0.000 description 1
- 208000031448 Genomic Instability Diseases 0.000 description 1
- 101000914514 Homo sapiens T-cell-specific surface glycoprotein CD28 Proteins 0.000 description 1
- 208000019693 Lung disease Diseases 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 101710163270 Nuclease Proteins 0.000 description 1
- 108020004711 Nucleic Acid Probes Proteins 0.000 description 1
- ISWSIDIOOBJBQZ-UHFFFAOYSA-N Phenol Chemical compound OC1=CC=CC=C1 ISWSIDIOOBJBQZ-UHFFFAOYSA-N 0.000 description 1
- 108091081021 Sense strand Proteins 0.000 description 1
- 239000012506 Sephacryl® Substances 0.000 description 1
- 101150020431 Slk gene Proteins 0.000 description 1
- 208000010112 Spinocerebellar Degenerations Diseases 0.000 description 1
- NINIDFKCEFEMDL-UHFFFAOYSA-N Sulfur Chemical compound [S] NINIDFKCEFEMDL-UHFFFAOYSA-N 0.000 description 1
- 102100027213 T-cell-specific surface glycoprotein CD28 Human genes 0.000 description 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 description 1
- 108700005077 Viral Genes Proteins 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 238000002835 absorbance Methods 0.000 description 1
- 230000009471 action Effects 0.000 description 1
- 238000000137 annealing Methods 0.000 description 1
- 239000002246 antineoplastic agent Substances 0.000 description 1
- 229940041181 antineoplastic drug Drugs 0.000 description 1
- 239000003125 aqueous solvent Substances 0.000 description 1
- 239000012131 assay buffer Substances 0.000 description 1
- 208000006673 asthma Diseases 0.000 description 1
- 239000011324 bead Substances 0.000 description 1
- 230000009286 beneficial effect Effects 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- 108091092356 cellular DNA Proteins 0.000 description 1
- 230000033077 cellular process Effects 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 238000003776 cleavage reaction Methods 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 238000005056 compaction Methods 0.000 description 1
- 239000002131 composite material Substances 0.000 description 1
- 238000009833 condensation Methods 0.000 description 1
- 230000005494 condensation Effects 0.000 description 1
- 230000001276 controlling effect Effects 0.000 description 1
- 230000002596 correlated effect Effects 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 238000004163 cytometry Methods 0.000 description 1
- 210000000805 cytoplasm Anatomy 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 238000011033 desalting Methods 0.000 description 1
- 238000001514 detection method Methods 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 238000010790 dilution Methods 0.000 description 1
- 239000012895 dilution Substances 0.000 description 1
- 230000003292 diminished effect Effects 0.000 description 1
- 230000008034 disappearance Effects 0.000 description 1
- 238000012377 drug delivery Methods 0.000 description 1
- 230000002900 effect on cell Effects 0.000 description 1
- 238000001962 electrophoresis Methods 0.000 description 1
- 230000007613 environmental effect Effects 0.000 description 1
- 230000002255 enzymatic effect Effects 0.000 description 1
- VYXSBFYARXAAKO-UHFFFAOYSA-N ethyl 2-[3-(ethylamino)-6-ethylimino-2,7-dimethylxanthen-9-yl]benzoate;hydron;chloride Chemical compound [Cl-].C1=2C=C(C)C(NCC)=CC=2OC2=CC(=[NH+]CC)C(C)=CC2=C1C1=CC=CC=C1C(=O)OCC VYXSBFYARXAAKO-UHFFFAOYSA-N 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 238000001704 evaporation Methods 0.000 description 1
- 230000008020 evaporation Effects 0.000 description 1
- 230000005284 excitation Effects 0.000 description 1
- 230000007717 exclusion Effects 0.000 description 1
- 102000013165 exonuclease Human genes 0.000 description 1
- 238000000605 extraction Methods 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 238000001914 filtration Methods 0.000 description 1
- 238000001943 fluorescence-activated cell sorting Methods 0.000 description 1
- 239000007850 fluorescent dye Substances 0.000 description 1
- 108010055863 gene b exonuclease Proteins 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- 238000012239 gene modification Methods 0.000 description 1
- 230000037440 gene silencing effect Effects 0.000 description 1
- 230000005017 genetic modification Effects 0.000 description 1
- 235000013617 genetically modified food Nutrition 0.000 description 1
- 239000011521 glass Substances 0.000 description 1
- 230000009036 growth inhibition Effects 0.000 description 1
- 230000036541 health Effects 0.000 description 1
- 238000004128 high performance liquid chromatography Methods 0.000 description 1
- 231100000110 immunotoxic Toxicity 0.000 description 1
- 230000002625 immunotoxic effect Effects 0.000 description 1
- 238000011534 incubation Methods 0.000 description 1
- 230000006698 induction Effects 0.000 description 1
- 230000028709 inflammatory response Effects 0.000 description 1
- 239000000138 intercalating agent Substances 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 230000002045 lasting effect Effects 0.000 description 1
- 231100001231 less toxic Toxicity 0.000 description 1
- 210000004185 liver Anatomy 0.000 description 1
- 210000005265 lung cell Anatomy 0.000 description 1
- 239000012139 lysis buffer Substances 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 210000004379 membrane Anatomy 0.000 description 1
- 239000012528 membrane Substances 0.000 description 1
- CAAULPUQFIIOTL-UHFFFAOYSA-N methyl dihydrogen phosphate Chemical compound COP(O)(O)=O CAAULPUQFIIOTL-UHFFFAOYSA-N 0.000 description 1
- 238000010369 molecular cloning Methods 0.000 description 1
- 239000000178 monomer Substances 0.000 description 1
- ZIUHHBKFKCYYJD-UHFFFAOYSA-N n,n'-methylenebisacrylamide Chemical compound C=CC(=O)NCNC(=O)C=C ZIUHHBKFKCYYJD-UHFFFAOYSA-N 0.000 description 1
- 239000002853 nucleic acid probe Substances 0.000 description 1
- 238000002515 oligonucleotide synthesis Methods 0.000 description 1
- VYNDHICBIRRPFP-UHFFFAOYSA-N pacific blue Chemical compound FC1=C(O)C(F)=C2OC(=O)C(C(=O)O)=CC2=C1 VYNDHICBIRRPFP-UHFFFAOYSA-N 0.000 description 1
- 230000036961 partial effect Effects 0.000 description 1
- 230000001717 pathogenic effect Effects 0.000 description 1
- 230000027086 plasmid maintenance Effects 0.000 description 1
- 238000002264 polyacrylamide gel electrophoresis Methods 0.000 description 1
- 230000035755 proliferation Effects 0.000 description 1
- 230000000069 prophylactic effect Effects 0.000 description 1
- 238000011321 prophylaxis Methods 0.000 description 1
- 230000001681 protective effect Effects 0.000 description 1
- 238000011002 quantification Methods 0.000 description 1
- 230000003252 repetitive effect Effects 0.000 description 1
- 238000011160 research Methods 0.000 description 1
- 108091008146 restriction endonucleases Proteins 0.000 description 1
- 238000005070 sampling Methods 0.000 description 1
- 230000007017 scission Effects 0.000 description 1
- 238000012163 sequencing technique Methods 0.000 description 1
- 238000001228 spectrum Methods 0.000 description 1
- 229910052717 sulfur Inorganic materials 0.000 description 1
- 239000011593 sulfur Substances 0.000 description 1
- 230000009469 supplementation Effects 0.000 description 1
- 208000024891 symptom Diseases 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- MPLHNVLQVRSVEE-UHFFFAOYSA-N texas red Chemical compound [O-]S(=O)(=O)C1=CC(S(Cl)(=O)=O)=CC=C1C(C1=CC=2CCCN3CCCC(C=23)=C1O1)=C2C1=C(CCC1)C3=[N+]1CCCC3=C2 MPLHNVLQVRSVEE-UHFFFAOYSA-N 0.000 description 1
- 230000036962 time dependent Effects 0.000 description 1
- 238000013518 transcription Methods 0.000 description 1
- 230000035897 transcription Effects 0.000 description 1
- 230000002103 transcriptional effect Effects 0.000 description 1
- 230000001052 transient effect Effects 0.000 description 1
- PIEPQKCYPFFYMG-UHFFFAOYSA-N tris acetate Chemical compound CC(O)=O.OCC(N)(CO)CO PIEPQKCYPFFYMG-UHFFFAOYSA-N 0.000 description 1
- 230000003827 upregulation Effects 0.000 description 1
- 230000001018 virulence Effects 0.000 description 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 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
- C12N15/63—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
-
- 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
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
- A61P35/02—Antineoplastic agents specific for leukemia
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
-
- 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
- C12N15/86—Viral vectors
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/90—Vectors containing a transposable element
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- Hematology (AREA)
- Oncology (AREA)
- Pulmonology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US25245509P | 2009-10-16 | 2009-10-16 | |
| US61/252,455 | 2009-10-16 | ||
| PCT/US2010/052920 WO2011047318A2 (en) | 2009-10-16 | 2010-10-15 | Supercoiled minicircle dna for gene therapy applications |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016214390A Division JP2017046710A (ja) | 2009-10-16 | 2016-11-01 | 遺伝子療法適用のためのスーパーコイル状ミニサークルｄｎａ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2013507934A true JP2013507934A (ja) | 2013-03-07 |
| JP2013507934A5 JP2013507934A5 (enExample) | 2013-12-05 |
Family
ID=43876905
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012534410A Pending JP2013507934A (ja) | 2009-10-16 | 2010-10-15 | 遺伝子療法適用のためのスーパーコイル状ミニサークルｄｎａ |
| JP2016214390A Pending JP2017046710A (ja) | 2009-10-16 | 2016-11-01 | 遺伝子療法適用のためのスーパーコイル状ミニサークルｄｎａ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016214390A Pending JP2017046710A (ja) | 2009-10-16 | 2016-11-01 | 遺伝子療法適用のためのスーパーコイル状ミニサークルｄｎａ |
Country Status (10)
| Country | Link |
|---|---|
| US (3) | US8460924B2 (enExample) |
| EP (1) | EP2488641B1 (enExample) |
| JP (2) | JP2013507934A (enExample) |
| KR (1) | KR20120094166A (enExample) |
| AU (1) | AU2010306645B2 (enExample) |
| BR (1) | BR112012011576A2 (enExample) |
| CA (1) | CA2777835A1 (enExample) |
| MX (1) | MX2012004447A (enExample) |
| NZ (1) | NZ599998A (enExample) |
| WO (1) | WO2011047318A2 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020508654A (ja) * | 2017-02-16 | 2020-03-26 | ザ・ユニバーシティ・オブ・シェフィールド | 安定型ｖａｍｐレポーターアッセイ |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101510830B1 (ko) | 2012-05-11 | 2015-04-10 | 가톨릭대학교 산학협력단 | 생리활성 단백질을 발현하는 미니서클을 이용하여 형질전환된 줄기세포를 통한 치료법 |
| WO2013181440A1 (en) * | 2012-05-30 | 2013-12-05 | Baylor College Of Medicine | Supercoiled minivectors as a tool for dna repair, alteration and replacement |
| EP2735614A1 (en) * | 2012-11-22 | 2014-05-28 | Centre National De La Recherche Scientifique | In vitro production of DNA minicircles comprising less than 250 base pairs |
| US10604771B2 (en) | 2013-05-10 | 2020-03-31 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| US20180305701A1 (en) | 2017-03-14 | 2018-10-25 | Baylor College Of Medicine | Targeting minivectors to specific tissue using shape |
| US20200048716A1 (en) | 2017-11-03 | 2020-02-13 | Twister Biotech, Inc | Using minivectors to treat ovarian cancer |
| WO2021055760A1 (en) | 2019-09-18 | 2021-03-25 | Intergalactic Therapeutics, Inc. | Synthetic dna vectors and methods of use |
| US20240100189A1 (en) | 2021-09-10 | 2024-03-28 | Twister Biotech, Inc | Using minivectors to treat idiopathic pulmonary fibrosis |
| US20240093226A1 (en) | 2021-09-10 | 2024-03-21 | Baylor College Of Medicine | Ultrapure minivectors for gene therapy |
| US20230190955A1 (en) | 2021-10-21 | 2023-06-22 | Baylor College Of Medicine | Treatment of liver cancer or liver fibrosis |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2007523593A (ja) * | 2003-06-10 | 2007-08-23 | モロゲン・アーゲー | 遺伝子治療に用いるための製品 |
| JP2008526229A (ja) * | 2005-01-06 | 2008-07-24 | ベニテック，インコーポレーテッド | 幹細胞の維持のためのＲＮＡｉ剤 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2626584B1 (fr) * | 1988-01-28 | 1990-07-13 | Agronomique Inst Nat Rech | Sequence ars efficace chez yarrowia lipolytica et procede pour sa preparation |
| WO1994009127A2 (en) | 1992-10-16 | 1994-04-28 | The Government Of The United States Of America As Represented By The Secretary, Department Of Healthand Human Services | Supercoiled minicircle dna as as a unitary promoter vector |
| US6964861B1 (en) * | 1998-11-13 | 2005-11-15 | Invitrogen Corporation | Enhanced in vitro recombinational cloning of using ribosomal proteins |
| EP2105496B1 (en) | 2000-12-08 | 2013-02-20 | Life Technologies Corporation | Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites |
| GB0108968D0 (en) * | 2001-04-10 | 2001-05-30 | Imp College Innovations Ltd | Methods |
| WO2002083889A2 (en) | 2001-04-10 | 2002-10-24 | Imperial College Innovations Limited | Methods for the production of minicircles |
| WO2003064623A2 (en) | 2002-01-31 | 2003-08-07 | Washington State University Research Foundation | Methods and vectors for facilitating site-specific recombination |
| AU2003273995A1 (en) | 2002-06-05 | 2003-12-22 | Invitrogen Corporation | Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites |
| EP2801614B1 (en) * | 2002-08-29 | 2019-05-15 | The Board of Trustees of the Leland Stanford Junior University | Circular nucleic acid vectors and methods for making and using the same |
| US7288529B2 (en) * | 2003-04-24 | 2007-10-30 | New York University | ALK protein tyrosine kinase, cells and methods embodying and using same |
| US7622252B2 (en) * | 2005-06-10 | 2009-11-24 | Baylor College Of Medicine | Generation of minicircle DNA with physiological supercoiling |
| WO2007130604A2 (en) * | 2006-05-04 | 2007-11-15 | Baylor Research Institute | Anti-tumor activity of an oncolytic adenovirus-delivered oncogene sirna |
| EP2178896A1 (en) | 2007-07-31 | 2010-04-28 | The Johns Hopkins University | Polypeptide-nucleic acid conjugate for immunoprophylaxis or immunotherapy for neoplastic or infectious disorders |
-
2010
- 2010-10-15 US US12/905,612 patent/US8460924B2/en active Active
- 2010-10-15 KR KR20127012633A patent/KR20120094166A/ko not_active Withdrawn
- 2010-10-15 BR BR112012011576A patent/BR112012011576A2/pt not_active IP Right Cessation
- 2010-10-15 WO PCT/US2010/052920 patent/WO2011047318A2/en not_active Ceased
- 2010-10-15 MX MX2012004447A patent/MX2012004447A/es active IP Right Grant
- 2010-10-15 US US13/502,162 patent/US8729044B2/en active Active
- 2010-10-15 CA CA 2777835 patent/CA2777835A1/en not_active Abandoned
- 2010-10-15 NZ NZ59999810A patent/NZ599998A/en not_active IP Right Cessation
- 2010-10-15 JP JP2012534410A patent/JP2013507934A/ja active Pending
- 2010-10-15 AU AU2010306645A patent/AU2010306645B2/en not_active Ceased
- 2010-10-15 EP EP10824202.5A patent/EP2488641B1/en active Active
-
2013
- 2013-04-30 US US13/873,737 patent/US9267150B2/en active Active
-
2016
- 2016-11-01 JP JP2016214390A patent/JP2017046710A/ja active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2007523593A (ja) * | 2003-06-10 | 2007-08-23 | モロゲン・アーゲー | 遺伝子治療に用いるための製品 |
| JP2008526229A (ja) * | 2005-01-06 | 2008-07-24 | ベニテック，インコーポレーテッド | 幹細胞の維持のためのＲＮＡｉ剤 |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020508654A (ja) * | 2017-02-16 | 2020-03-26 | ザ・ユニバーシティ・オブ・シェフィールド | 安定型ｖａｍｐレポーターアッセイ |
Also Published As
| Publication number | Publication date |
|---|---|
| US20110160284A1 (en) | 2011-06-30 |
| WO2011047318A2 (en) | 2011-04-21 |
| EP2488641A2 (en) | 2012-08-22 |
| US8729044B2 (en) | 2014-05-20 |
| US20130316449A1 (en) | 2013-11-28 |
| WO2011047318A3 (en) | 2011-09-09 |
| US20120302625A1 (en) | 2012-11-29 |
| MX2012004447A (es) | 2012-10-15 |
| US9267150B2 (en) | 2016-02-23 |
| EP2488641A4 (en) | 2013-11-13 |
| BR112012011576A2 (pt) | 2015-09-15 |
| EP2488641B1 (en) | 2019-01-23 |
| US8460924B2 (en) | 2013-06-11 |
| AU2010306645B2 (en) | 2017-03-23 |
| KR20120094166A (ko) | 2012-08-23 |
| NZ599998A (en) | 2014-11-28 |
| CA2777835A1 (en) | 2011-04-21 |
| AU2010306645A1 (en) | 2012-06-07 |
| WO2011047318A8 (en) | 2011-06-09 |
| JP2017046710A (ja) | 2017-03-09 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2017046710A (ja) | 遺伝子療法適用のためのスーパーコイル状ミニサークルｄｎａ | |
| AU2021201683B2 (en) | Novel CAS13B orthologues CRISPR enzymes and systems | |
| US11667903B2 (en) | Tracking and manipulating cellular RNA via nuclear delivery of CRISPR/CAS9 | |
| KR102418185B1 (ko) | 단일 가닥 rna-편집 올리고뉴클레오타이드 | |
| US11884918B2 (en) | Systems and methods for modulating CRISPR activity | |
| CA2915845A1 (en) | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells | |
| EP4711451A1 (en) | Cas protein, corresponding gene editing system thereof and use thereof | |
| Tou et al. | Immune evasive DNA donors and recombinases license kilobase-scale writing | |
| US12421507B2 (en) | Methods and compositions for optochemical control of CRISPR-CAS9 | |
| CN119630786A (zh) | 用于位点特异性基因组修饰的多组件系统 | |
| US20130085173A1 (en) | Supercoiled minicircle dna for gene therapy applications | |
| JP6795492B2 (ja) | ＣＬＣＮ７（ＡＤＯ２ ＣＬＣＮ７依存性）遺伝子変異によって引き起こされる常染色体優性大理石骨病２型（ＡＤＯ２）療法のための短鎖干渉ＲＮＡ（ｓｉＲＮＡ） | |
| HK40024463A (en) | Novel type vi crispr orthologs and systems | |
| HK40022746A (en) | Novel cas13b orthologues crispr enzymes and systems | |
| HK40005508A (en) | Lipid nanoparticle formulations for crispr/cas components |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20131015 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20131015 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20141219 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20150319 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20150417 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20150518 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A821 Effective date: 20150518 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20151104 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20160204 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20160303 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20160401 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20160701 |