JP2015508760A5 - - Google Patents
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- JP2015508760A5 JP2015508760A5 JP2014556826A JP2014556826A JP2015508760A5 JP 2015508760 A5 JP2015508760 A5 JP 2015508760A5 JP 2014556826 A JP2014556826 A JP 2014556826A JP 2014556826 A JP2014556826 A JP 2014556826A JP 2015508760 A5 JP2015508760 A5 JP 2015508760A5
- Authority
- JP
- Japan
- Prior art keywords
- paracrine
- vector
- nucleic acid
- gene
- cell
- 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.)
- Granted
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- 108090000623 proteins and genes Proteins 0.000 claims description 131
- 230000003076 paracrine Effects 0.000 claims description 124
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 115
- 150000007523 nucleic acids Chemical class 0.000 claims description 109
- 102000005630 Urocortins Human genes 0.000 claims description 106
- 108010059705 Urocortins Proteins 0.000 claims description 106
- 239000000777 urocortin Substances 0.000 claims description 106
- 108020004707 nucleic acids Proteins 0.000 claims description 105
- 102000039446 nucleic acids Human genes 0.000 claims description 105
- 239000013598 vector Substances 0.000 claims description 92
- 210000004027 cell Anatomy 0.000 claims description 89
- 238000002347 injection Methods 0.000 claims description 74
- 239000007924 injection Substances 0.000 claims description 74
- ZEBBPGHOLWPSGI-KPLDDXDLSA-N urocortin ii Chemical compound CC[C@H](C)[C@H](N)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CO)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](C(C)C)C(=O)N1CCC[C@H]1C(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](C)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C(C)C)C(=O)NCC(=O)N[C@H](C(=O)N[C@@H](CS)C(N)=O)CC1=CN=CN1 ZEBBPGHOLWPSGI-KPLDDXDLSA-N 0.000 claims description 71
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 66
- 229920001184 polypeptide Polymers 0.000 claims description 63
- 239000000203 mixture Substances 0.000 claims description 57
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- 239000003981 vehicle Substances 0.000 claims description 45
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- 230000022532 regulation of transcription, DNA-dependent Effects 0.000 claims description 27
- 238000007918 intramuscular administration Methods 0.000 claims description 26
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- FCENQCVTLJEGOT-KIHVXQRMSA-N stresscopin Chemical compound C([C@@H](C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCSC)C(=O)N[C@@H](C)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H]([C@@H](C)CC)C(N)=O)NC(=O)[C@H](C)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](C)NC(=O)[C@H](C)NC(=O)[C@H](C)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@H](C)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CCCCN)NC(=O)[C@H](C)NC(=O)[C@H](CCCCN)NC(=O)[C@H](C)NC(=O)[C@@H](NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CCSC)NC(=O)[C@@H](NC(=O)[C@H](CC(N)=O)NC(=O)[C@@H](NC(=O)[C@H]1N(CCC1)C(=O)[C@@H](NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CO)NC(=O)[C@H](CC(C)C)NC(=O)[C@@H](NC(=O)[C@@H](N)CC=1C=CC=CC=1)[C@@H](C)O)C(C)C)[C@@H](C)O)[C@@H](C)CC)[C@@H](C)CC)C1=CN=CN1 FCENQCVTLJEGOT-KIHVXQRMSA-N 0.000 claims description 16
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- 101001091088 Homo sapiens Prorelaxin H2 Proteins 0.000 claims description 12
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- 239000002502 liposome Substances 0.000 claims description 11
- 239000002105 nanoparticle Substances 0.000 claims description 11
- 102400000667 Brain natriuretic peptide 32 Human genes 0.000 claims description 10
- 101800000407 Brain natriuretic peptide 32 Proteins 0.000 claims description 10
- 101800002247 Brain natriuretic peptide 45 Proteins 0.000 claims description 10
- 102000015846 Prostacyclin synthases Human genes 0.000 claims description 10
- 201000011510 cancer Diseases 0.000 claims description 10
- 210000000234 capsid Anatomy 0.000 claims description 10
- HPNRHPKXQZSDFX-OAQDCNSJSA-N nesiritide Chemical compound C([C@H]1C(=O)NCC(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCSC)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@H](C(N[C@@H](CO)C(=O)N[C@@H](CO)C(=O)N[C@@H](CO)C(=O)N[C@@H](CO)C(=O)NCC(=O)N[C@@H](CC(C)C)C(=O)NCC(=O)N[C@@H](CSSC[C@@H](C(=O)N1)NC(=O)CNC(=O)[C@H](CO)NC(=O)CNC(=O)[C@H](CCC(N)=O)NC(=O)[C@@H](NC(=O)[C@H](CCSC)NC(=O)[C@H](CCCCN)NC(=O)[C@H]1N(CCC1)C(=O)[C@@H](N)CO)C(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](CCCNC(N)=N)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC=1N=CNC=1)C(O)=O)=O)[C@@H](C)CC)C1=CC=CC=C1 HPNRHPKXQZSDFX-OAQDCNSJSA-N 0.000 claims description 10
- 108010064377 prostacyclin synthetase Proteins 0.000 claims description 10
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- 108010051696 Growth Hormone Proteins 0.000 claims description 8
- 102100038803 Somatotropin Human genes 0.000 claims description 8
- 101150084279 TM gene Proteins 0.000 claims description 8
- 230000003177 cardiotonic effect Effects 0.000 claims description 8
- 239000003102 growth factor Substances 0.000 claims description 8
- 239000000122 growth hormone Substances 0.000 claims description 8
- DTLOVISJEFBXLX-REAFJZEQSA-N relexan 2 Chemical compound C([C@H]1C(=O)N[C@H](C(=O)NCC(=O)N[C@H]2CSSC[C@@H](C(=O)N[C@H](C(N1)=O)CSSC[C@@H](C(NCC(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@H](C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@H](C(=O)N[C@@H](C)C(=O)N[C@H](C(=O)N[C@@H](CSSC[C@H](NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](C)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CO)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CCCCN)NC(=O)[C@H]([C@@H](C)O)NC2=O)C(O)=O)C(=O)NCC(=O)N[C@@H](CCSC)C(=O)N[C@@H](CO)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CO)C(O)=O)[C@@H](C)CC)[C@@H](C)CC)C(C)C)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CCCCN)NC(=O)[C@@H](NC(=O)[C@@H](NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCSC)NC(=O)[C@H](CC=1C2=CC=CC=C2NC=1)NC(=O)[C@H](CO)NC(=O)[C@@H](N)CC(O)=O)C(C)C)[C@@H](C)CC)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](C)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](C)NC(=O)[C@H](CO)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CC(C)C)NC(=O)[C@H]1NC(=O)CC1)C(C)C)C1=CN=CN1 DTLOVISJEFBXLX-REAFJZEQSA-N 0.000 claims description 8
- 229960002792 serelaxin Drugs 0.000 claims description 8
- 230000002459 sustained effect Effects 0.000 claims description 8
- 210000004556 brain Anatomy 0.000 claims description 7
- 239000012530 fluid Substances 0.000 claims description 7
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 6
- 241000702421 Dependoparvovirus Species 0.000 claims description 6
- 206010018429 Glucose tolerance impaired Diseases 0.000 claims description 6
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- 208000001280 Prediabetic State Diseases 0.000 claims description 6
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- 206010012601 diabetes mellitus Diseases 0.000 claims description 6
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- 208000002815 pulmonary hypertension Diseases 0.000 claims description 6
- 230000002103 transcriptional effect Effects 0.000 claims description 6
- 230000004584 weight gain Effects 0.000 claims description 6
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- 208000014644 Brain disease Diseases 0.000 claims description 5
- 102100022641 Coagulation factor IX Human genes 0.000 claims description 5
- 208000031220 Hemophilia Diseases 0.000 claims description 5
- 208000009292 Hemophilia A Diseases 0.000 claims description 5
- 230000006907 apoptotic process Effects 0.000 claims description 5
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- 210000002216 heart Anatomy 0.000 claims description 5
- 208000009429 hemophilia B Diseases 0.000 claims description 5
- 208000017169 kidney disease Diseases 0.000 claims description 5
- 210000004185 liver Anatomy 0.000 claims description 5
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- 239000000816 peptidomimetic Substances 0.000 claims description 5
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- 230000004936 stimulating effect Effects 0.000 claims description 5
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 4
- 241000283690 Bos taurus Species 0.000 claims description 4
- 241000283707 Capra Species 0.000 claims description 4
- 241000700199 Cavia porcellus Species 0.000 claims description 4
- 241000282693 Cercopithecidae Species 0.000 claims description 4
- 241000699800 Cricetinae Species 0.000 claims description 4
- 108010074864 Factor XI Proteins 0.000 claims description 4
- 241000282326 Felis catus Species 0.000 claims description 4
- 101000599951 Homo sapiens Insulin-like growth factor I Proteins 0.000 claims description 4
- 102100037852 Insulin-like growth factor I Human genes 0.000 claims description 4
- 241000282560 Macaca mulatta Species 0.000 claims description 4
- 241000283973 Oryctolagus cuniculus Species 0.000 claims description 4
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- 210000004413 cardiac myocyte Anatomy 0.000 claims description 4
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- 229960003722 doxycycline Drugs 0.000 claims description 4
- XQTWDDCIUJNLTR-CVHRZJFOSA-N doxycycline monohydrate Chemical compound O.O=C1C2=C(O)C=CC=C2[C@H](C)[C@@H]2C1=C(O)[C@]1(O)C(=O)C(C(N)=O)=C(O)[C@@H](N(C)C)[C@@H]1[C@H]2O XQTWDDCIUJNLTR-CVHRZJFOSA-N 0.000 claims description 4
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- 230000010247 heart contraction Effects 0.000 claims description 3
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- 241000282412 Homo Species 0.000 claims description 2
- 101000687346 Homo sapiens PR domain zinc finger protein 2 Proteins 0.000 claims description 2
- 102100024885 PR domain zinc finger protein 2 Human genes 0.000 claims description 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 2
- 108091023040 Transcription factor Proteins 0.000 claims description 2
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- 229940127249 oral antibiotic Drugs 0.000 claims description 2
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- ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 claims description 2
- 229960002930 sirolimus Drugs 0.000 claims description 2
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 claims description 2
- 239000011780 sodium chloride Substances 0.000 claims description 2
- 238000001179 sorption measurement Methods 0.000 claims description 2
- 230000001839 systemic circulation Effects 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- 239000013607 AAV vector Substances 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 4
- 230000000638 stimulation Effects 0.000 claims 4
- DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical compound C[C@@H]1CC[C@@]2(CC[C@@]3(C(=CC[C@H]4[C@]3(CC[C@@H]5[C@@]4(CC[C@@H](C5(C)C)OS(=O)(=O)[O-])C)C)[C@@H]2[C@]1(C)O)C)C(=O)O[C@H]6[C@@H]([C@H]([C@@H]([C@H](O6)CO)O)O)O.[Na+] DGAQECJNVWCQMB-PUAWFVPOSA-M 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 210000005260 human cell Anatomy 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 229910052708 sodium Inorganic materials 0.000 claims 2
- 239000011734 sodium Substances 0.000 claims 2
- 238000013518 transcription Methods 0.000 claims 2
- 230000035897 transcription Effects 0.000 claims 2
- 206010071436 Systolic dysfunction Diseases 0.000 claims 1
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- 230000017531 blood circulation Effects 0.000 claims 1
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- 230000002265 prevention Effects 0.000 claims 1
- 230000000069 prophylactic effect Effects 0.000 claims 1
- 230000001681 protective effect Effects 0.000 claims 1
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- XZPMQCKVOWVETG-UHFFFAOYSA-J tetrasodium;2-[(3-carboxylato-3-sulfonatopropanoyl)-octadecylamino]butanedioate Chemical compound [Na+].[Na+].[Na+].[Na+].CCCCCCCCCCCCCCCCCCN(C(CC([O-])=O)C([O-])=O)C(=O)CC(C([O-])=O)S([O-])(=O)=O XZPMQCKVOWVETG-UHFFFAOYSA-J 0.000 claims 1
- 208000031229 Cardiomyopathies Diseases 0.000 description 1
- 206010020772 Hypertension Diseases 0.000 description 1
- YXLIYGUJLJFLJH-UHFFFAOYSA-L disodium;4-(octadecylamino)-4-oxo-2-sulfonatobutanoate Chemical compound [Na+].[Na+].CCCCCCCCCCCCCCCCCCNC(=O)CC(C([O-])=O)S([O-])(=O)=O YXLIYGUJLJFLJH-UHFFFAOYSA-L 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261598772P | 2012-02-14 | 2012-02-14 | |
| US61/598,772 | 2012-02-14 | ||
| PCT/US2013/025997 WO2013123094A2 (en) | 2012-02-14 | 2013-02-13 | Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017163244A Division JP2017203048A (ja) | 2012-02-14 | 2017-08-28 | 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現 |
| JP2019096752A Division JP2019135263A (ja) | 2012-02-14 | 2019-05-23 | 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015508760A JP2015508760A (ja) | 2015-03-23 |
| JP2015508760A5 true JP2015508760A5 (https=) | 2016-03-31 |
| JP6562632B2 JP6562632B2 (ja) | 2019-08-21 |
Family
ID=48984885
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014556826A Active JP6562632B2 (ja) | 2012-02-14 | 2013-02-13 | 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現 |
| JP2017163244A Pending JP2017203048A (ja) | 2012-02-14 | 2017-08-28 | 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現 |
| JP2019096752A Withdrawn JP2019135263A (ja) | 2012-02-14 | 2019-05-23 | 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017163244A Pending JP2017203048A (ja) | 2012-02-14 | 2017-08-28 | 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現 |
| JP2019096752A Withdrawn JP2019135263A (ja) | 2012-02-14 | 2019-05-23 | 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現 |
Country Status (18)
| Country | Link |
|---|---|
| US (3) | US20150118287A1 (https=) |
| EP (2) | EP2814513B1 (https=) |
| JP (3) | JP6562632B2 (https=) |
| KR (1) | KR102107482B1 (https=) |
| CN (2) | CN108310396B (https=) |
| AU (2) | AU2013221615B2 (https=) |
| BR (1) | BR112014020127A2 (https=) |
| CA (1) | CA2864100A1 (https=) |
| DK (1) | DK2814513T3 (https=) |
| ES (1) | ES2660492T3 (https=) |
| HK (1) | HK1254639A1 (https=) |
| HU (1) | HUE036640T2 (https=) |
| IL (3) | IL234007A0 (https=) |
| IN (1) | IN2014DN06789A (https=) |
| PL (1) | PL2814513T3 (https=) |
| PT (1) | PT2814513T (https=) |
| RU (1) | RU2642605C2 (https=) |
| WO (1) | WO2013123094A2 (https=) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| CN108310396B (zh) * | 2012-02-14 | 2022-07-15 | 加利福尼亚大学董事会 | 用于心血管疾病和其它病况的旁分泌基因的全身性递送和受调节的表达 |
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- 2013-02-13 CN CN201810214134.1A patent/CN108310396B/zh active Active
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- 2013-02-13 CA CA2864100A patent/CA2864100A1/en not_active Abandoned
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