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
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- 102400000667 Brain natriuretic peptide 32 Human genes 0.000 claims description 10
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- 101800002247 Brain natriuretic peptide 45 Proteins 0.000 claims description 10
- 102000015846 Prostacyclin synthases Human genes 0.000 claims description 10
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- 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
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- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 6
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- 208000031220 Hemophilia Diseases 0.000 claims description 5
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- 239000000816 peptidomimetic Substances 0.000 claims description 5
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- 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
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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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- 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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- 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
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- 230000001839 systemic circulation Effects 0.000 claims description 2
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- 239000013607 AAV vector Substances 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 4
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- 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
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- 206010071436 Systolic dysfunction Diseases 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
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- 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 (enExample) | 2016-03-31 |
| JP6562632B2 JP6562632B2 (ja) | 2019-08-21 |
Family
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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 (enExample) |
| EP (2) | EP2814513B1 (enExample) |
| JP (3) | JP6562632B2 (enExample) |
| KR (1) | KR102107482B1 (enExample) |
| CN (2) | CN104220098B (enExample) |
| AU (2) | AU2013221615B2 (enExample) |
| BR (1) | BR112014020127A2 (enExample) |
| CA (1) | CA2864100A1 (enExample) |
| DK (1) | DK2814513T3 (enExample) |
| ES (1) | ES2660492T3 (enExample) |
| HK (1) | HK1254639A1 (enExample) |
| HU (1) | HUE036640T2 (enExample) |
| IL (3) | IL234007A0 (enExample) |
| IN (1) | IN2014DN06789A (enExample) |
| PL (1) | PL2814513T3 (enExample) |
| PT (1) | PT2814513T (enExample) |
| RU (1) | RU2642605C2 (enExample) |
| WO (1) | WO2013123094A2 (enExample) |
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 |
| PT2814513T (pt) * | 2012-02-14 | 2018-03-02 | Univ California | Entrega sistémica e expressão regulada de genes parácrinos para doenças cardiovasculares e outras doenças |
| CA2943751A1 (en) * | 2014-04-03 | 2015-10-08 | The Regents Of The University Of California | Systemic delivery of virus vectors encoding urocortin-2 and related genes to treat diabetes-related cardiac dysfunctions and congestive heart failure |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| JP7023108B2 (ja) | 2014-10-21 | 2022-02-21 | ユニバーシティ オブ マサチューセッツ | 組み換えａａｖバリアントおよびその使用 |
| BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
| US10570395B2 (en) | 2014-11-14 | 2020-02-25 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CA2967367C (en) | 2014-11-14 | 2023-05-16 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| CA2984769C (en) * | 2015-05-12 | 2023-03-21 | The Regents Of The University Of California | Peptide treatment for inflammation and fibrosis |
| CN113999875A (zh) * | 2015-05-28 | 2022-02-01 | 康奈尔大学 | 腺相关病毒介导的c1ei递送作为用于血管性水肿的疗法 |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| RU2764587C2 (ru) | 2016-05-18 | 2022-01-18 | Вояджер Терапьютикс, Инк. | Способы и композиции для лечения хореи гентингтона |
| JP7066635B2 (ja) | 2016-05-18 | 2022-05-13 | ボイジャー セラピューティクス インコーポレイテッド | 調節性ポリヌクレオチド |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JP7577291B6 (ja) | 2016-10-13 | 2024-11-26 | ユニバーシティ オブ マサチューセッツ | Ａａｖカプシド設計 |
| BR112019015081A2 (pt) * | 2017-01-23 | 2020-03-10 | Rutgers, The State University Of New Jersey | Composições para reduzir a expressão da sarcolipina e prevenir e tratar a distrofia muscular e a cardiomiopatia e os métodos de utilização |
| EP4600365A3 (en) * | 2017-02-28 | 2025-12-10 | Genemedicine Co., Ltd. | Anticancer composition comprising tumor-specific oncolytic adenovirus and immune checkpoint inhibitor |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| US11760987B2 (en) | 2017-09-07 | 2023-09-19 | The Regents Of The University Of California | Compositions and methods for the treatment or prevention of heart failure |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| JP2021523914A (ja) | 2018-05-15 | 2021-09-09 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | パーキンソン病を治療するための組成物および方法 |
| EP3801465B1 (en) * | 2018-06-11 | 2024-07-24 | The Regents of the University of California | Viral vector encoding elovl2 protein for treating age-related macular degeneration |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2021287122C1 (en) * | 2020-06-12 | 2025-04-10 | Gencellmed Inc. | Recombinant Herpes Simplex Virus comprising glycoprotein GH that has been modified for retargeting, and use thereof |
| CN112826823B (zh) * | 2021-01-11 | 2022-03-08 | 中国人民解放军军事科学院军事医学研究院 | 多西环素在制备治疗和/或预防和/或缓解和/或改善心肌病药物中的应用 |
Family Cites Families (61)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5217879A (en) | 1989-01-12 | 1993-06-08 | Washington University | Infectious Sindbis virus vectors |
| US5733542A (en) | 1990-11-16 | 1998-03-31 | Haynesworth; Stephen E. | Enhancing bone marrow engraftment using MSCS |
| US5270192A (en) | 1991-02-07 | 1993-12-14 | Monsanto Company | Biological artificial liver |
| US5846743A (en) | 1995-02-22 | 1998-12-08 | Brigham And Women's Hospital, Inc. | Polyphoshoinositide binding peptides for intracellular drug delivery |
| US7361332B2 (en) | 1995-03-17 | 2008-04-22 | The Regents Of The University Of California | Treating tumors using implants comprising combinations of allogeneic cells |
| US6214797B1 (en) * | 1995-06-13 | 2001-04-10 | The Salk Institute For Biological Studies | Urocortin peptides, nucleic acid encoding same methods for using same |
| US5721118A (en) | 1995-10-31 | 1998-02-24 | The Regents Of The University Of California, San Diego | Mammalian artificial chromosomes and methods of using same |
| DE69636289T2 (de) | 1995-12-18 | 2007-05-10 | Angiodevice International Gmbh | Vernetzten polymerisatmassen und verfahren für ihre verwendung |
| US6025155A (en) | 1996-04-10 | 2000-02-15 | Chromos Molecular Systems, Inc. | Artificial chromosomes, uses thereof and methods for preparing artificial chromosomes |
| US6933331B2 (en) | 1998-05-22 | 2005-08-23 | Nanoproducts Corporation | Nanotechnology for drug delivery, contrast agents and biomedical implants |
| US5874268A (en) | 1996-09-23 | 1999-02-23 | Duke University | Method of introducing exogenous compounds into cells by electroporation and apparatus for same |
| WO1998020027A2 (en) * | 1996-11-01 | 1998-05-14 | Eurogene Limited | Therapeutic use of an agent that stimulates no or prostacyclin production and delivery device |
| US6630137B1 (en) | 1997-04-28 | 2003-10-07 | Eli Lilly And Company | Activated protein C formulations |
| KR20070005030A (ko) * | 1997-05-06 | 2007-01-09 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 혈관형성성 트랜스유전자의 생체내 송달에 의한 심장 질환및 심실 리모델링을 치료하기 위한 방법 및 조성물 |
| US20050095227A1 (en) * | 1997-07-22 | 2005-05-05 | The General Hospital Corporation | Treating heart failure |
| SE9704076D0 (sv) | 1997-11-06 | 1997-11-06 | Holdingbolaget Vid Goeteborgs | Method for permeabilisation of cell structures and use thereof |
| US6886568B2 (en) | 1998-04-08 | 2005-05-03 | The Johns Hopkins University | Method for fabricating cell-containing implants |
| WO1999061601A2 (en) * | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| US6589503B1 (en) | 1998-06-20 | 2003-07-08 | Washington University | Membrane-permeant peptide complexes for medical imaging, diagnostics, and pharmaceutical therapy |
| US6958212B1 (en) | 1999-02-01 | 2005-10-25 | Eidgenossische Technische Hochschule Zurich | Conjugate addition reactions for the controlled delivery of pharmaceutically active compounds |
| JP2003513942A (ja) * | 1999-11-05 | 2003-04-15 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | インビボ遺伝子送達による心血管疾患を処理するための技術および組成物 |
| AU2460001A (en) * | 1999-12-27 | 2001-07-09 | Regents Of The University Of California, The | Gene therapy for congestive heart failure |
| WO2001077328A1 (en) | 2000-04-06 | 2001-10-18 | Franco Wayne P | Methods of using growth factors for treating heart disease |
| US7442390B2 (en) | 2000-06-05 | 2008-10-28 | University Of South Florida | Method for enhancing engraftment of cells using mesenchymal progenitor cells |
| NZ523803A (en) * | 2000-08-04 | 2004-08-27 | Res Dev Foundation | Human urocortin-related polypeptide (URP) and uses thereof to rreat a pathophysiological state, eg stress, anxiety and appetite dysfunction |
| AU8468301A (en) * | 2000-08-04 | 2002-02-18 | Res Dev Foundation | Urocortin proteins and uses thereof |
| EP1382672A4 (en) | 2001-04-24 | 2004-06-30 | Hokkaido Tech Licensing Office | COLONY RICH IN SMALL HEPATIC CELLS, METHOD FOR OBTAINING THIS COLONY, METHOD FOR INDUCING THE MATURATION OF THIS COLONY IN HEPATIC TISSUE AND METHOD FOR ESTIMATING THE EFFECT OF A MEDICAMENT USING THIS COLONY RICH IN SMALL MATURE HEPATIC CELLS |
| DK1453547T3 (en) * | 2001-12-17 | 2016-12-05 | Univ Pennsylvania | ADENOASSOCATED VIRUS (AAV) SEROTYPE 8 SEQUENCES, VECTORS CONTAINING THESE AND APPLICATIONS THEREOF |
| US6936585B2 (en) * | 2002-01-16 | 2005-08-30 | The Procter & Gamble Company | Corticotropin releasing factor 2 receptor agonists |
| US7166133B2 (en) | 2002-06-13 | 2007-01-23 | Kensey Nash Corporation | Devices and methods for treating defects in the tissue of a living being |
| US20040151766A1 (en) | 2003-01-30 | 2004-08-05 | Monahan Sean D. | Protein and peptide delivery to mammalian cells in vitro |
| US7241455B2 (en) | 2003-04-08 | 2007-07-10 | Boston Scientific Scimed, Inc. | Implantable or insertable medical devices containing radiation-crosslinked polymer for controlled delivery of a therapeutic agent |
| EP1648298A4 (en) | 2003-07-25 | 2010-01-13 | Dexcom Inc | OXYGEN-IMPROVED MEMBRANE SYSTEMS FOR IMPLANTABLE DEVICES |
| US7794706B2 (en) | 2003-10-14 | 2010-09-14 | Medivas, Llc | Bioactive wound dressings and implantable devices and methods of use |
| US20050136121A1 (en) | 2003-12-22 | 2005-06-23 | Shear/Kershman Laboratories, Inc. | Oral peptide delivery system with improved bioavailability |
| KR20060123384A (ko) | 2003-12-24 | 2006-12-01 | 가부시키가이샤 엘티티 바이오파마 | 약물을 함유하는 나노 입자 및 그 제조 방법, 그리고 당해나노 입자로 이루어지는 비경구 투여용 제제 |
| US20050244463A1 (en) | 2004-04-30 | 2005-11-03 | Allergan, Inc. | Sustained release intraocular implants and methods for treating ocular vasculopathies |
| US8658203B2 (en) | 2004-05-03 | 2014-02-25 | Merrimack Pharmaceuticals, Inc. | Liposomes useful for drug delivery to the brain |
| JP4758915B2 (ja) | 2004-08-06 | 2011-08-31 | バイオスペクトラム，インコーポレイテッド | 多重層リポソームおよびその製造方法 |
| US7442389B2 (en) | 2004-08-20 | 2008-10-28 | Artes Medical, Inc. | Methods of administering microparticles combined with autologous body components |
| US7919112B2 (en) | 2004-08-26 | 2011-04-05 | Pathak Holdings, Llc | Implantable tissue compositions and method |
| US7351423B2 (en) | 2004-09-01 | 2008-04-01 | Depuy Spine, Inc. | Musculo-skeletal implant having a bioactive gradient |
| US7501486B2 (en) | 2004-09-07 | 2009-03-10 | Burnham Institute For Medical Research | Peptides that selectively home to heart vasculature and related conjugates and methods |
| US7521415B2 (en) | 2004-10-18 | 2009-04-21 | Nitto Denko Corporation | Methods of intracellular peptide delivery |
| CN101137668B (zh) * | 2005-02-08 | 2011-08-17 | 研究发展基金会 | 与可溶性G蛋白偶联受体（sGPCR）有关的组合物与方法 |
| US7759120B2 (en) | 2005-03-02 | 2010-07-20 | Kps Bay Medical, Inc. | Seeding implantable medical devices with cells |
| WO2006121532A2 (en) * | 2005-03-31 | 2006-11-16 | Mytogen, Inc. | Treatment for heart disease |
| EP1937213B1 (en) | 2005-07-27 | 2017-10-25 | Protiva Biotherapeutics Inc. | Systems and methods for manufacturing liposomes |
| US20070265221A1 (en) * | 2006-05-09 | 2007-11-15 | Weiss Robert G | Methods to improve creatine kinase metabolism and contractile function in cardiac muscle for the treatment of heart failure |
| EP2114437A2 (en) * | 2006-10-16 | 2009-11-11 | ConjuChem Biotechnologies Inc. | Modified corticotropin releasing factor peptides and uses thereof |
| US20080119909A1 (en) | 2006-11-21 | 2008-05-22 | Neurotrophincell Pty Limited | Cell implantation to prevent and/or treat hearing loss |
| CA2705862C (en) * | 2007-11-16 | 2018-03-27 | San Diego State University Research Foundation | Compositions and method for manipulating pim-1 activity in circulatory system cells |
| WO2009088786A1 (en) | 2007-12-31 | 2009-07-16 | Nanocor Therapeutics, Inc. | Rna interference for the treatment of heart failure |
| US20110144019A1 (en) * | 2008-05-16 | 2011-06-16 | Elaine Unemori | Method of treating chronic heart failure |
| TWI386202B (zh) * | 2008-05-16 | 2013-02-21 | Corthera Inc | 治療與急性心臟衰竭相關之呼吸困難的方法 |
| EA201170647A1 (ru) * | 2008-11-04 | 2011-12-30 | Янссен Фармацевтика Нв | Пептиды-агонисты crhr2 и их использование |
| WO2010138263A2 (en) * | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US20110105397A1 (en) * | 2009-11-04 | 2011-05-05 | Gengo Peter J | Method for treating heart failure with stresscopin-like peptides |
| PT2814513T (pt) * | 2012-02-14 | 2018-03-02 | Univ California | Entrega sistémica e expressão regulada de genes parácrinos para doenças cardiovasculares e outras doenças |
| AU2017209189B2 (en) * | 2016-01-19 | 2024-03-28 | The Regents Of The University Of California | Methods for the treatment of Danon disease and other disorders of autophagy |
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- 2013-02-13 RU RU2014137169A patent/RU2642605C2/ru active
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- 2013-02-13 CA CA2864100A patent/CA2864100A1/en not_active Abandoned
- 2013-02-13 WO PCT/US2013/025997 patent/WO2013123094A2/en not_active Ceased
- 2013-02-13 EP EP17208610.0A patent/EP3311830A1/en not_active Withdrawn
- 2013-02-13 BR BR112014020127-7A patent/BR112014020127A2/pt not_active Application Discontinuation
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