JP2010540564A - 自己相補的なａａｖが媒介する眼の障害を処置または予防するための干渉ｒｎａ分子の送達 - Google Patents
自己相補的なａａｖが媒介する眼の障害を処置または予防するための干渉ｒｎａ分子の送達 Download PDFInfo
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- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
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- A61P27/04—Artificial tears; Irrigation solutions
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- A—HUMAN NECESSITIES
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- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/06—Antiglaucoma agents or miotics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P27/00—Drugs for disorders of the senses
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- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
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Applications Claiming Priority (2)
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| US97655207P | 2007-10-01 | 2007-10-01 | |
| PCT/US2008/078380 WO2009046059A1 (en) | 2007-10-01 | 2008-10-01 | Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders |
Publications (2)
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| JP2010540564A true JP2010540564A (ja) | 2010-12-24 |
| JP2010540564A5 JP2010540564A5 (https=) | 2012-09-13 |
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Country Status (12)
| Country | Link |
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| US (1) | US20090087413A1 (https=) |
| EP (1) | EP2192926A1 (https=) |
| JP (1) | JP2010540564A (https=) |
| KR (1) | KR20100061792A (https=) |
| CN (1) | CN101815536A (https=) |
| AU (1) | AU2008308784B2 (https=) |
| BR (1) | BRPI0817937A2 (https=) |
| CA (1) | CA2694091A1 (https=) |
| MX (1) | MX2010001608A (https=) |
| RU (1) | RU2010117178A (https=) |
| WO (1) | WO2009046059A1 (https=) |
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Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20170054493A (ko) * | 2014-09-16 | 2017-05-17 | 젠자임 코포레이션 | 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 |
| JP2021526839A (ja) * | 2018-06-12 | 2021-10-11 | ザ ユニバーシティ オブ ブリストル | 眼圧及び頭蓋内圧を調節するための材料及び方法 |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| EP2287323A1 (en) * | 2009-07-31 | 2011-02-23 | Association Institut de Myologie | Widespread gene delivery to the retina using systemic administration of AAV vectors |
| SG10201800541SA (en) | 2010-04-23 | 2018-03-28 | Univ Florida | Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1) |
| CN103505743A (zh) | 2012-06-21 | 2014-01-15 | 北京命码生科科技有限公司 | 含功能性microRNA/siRNA的细胞微粒子及其应用 |
| PL4012035T3 (pl) * | 2014-09-16 | 2025-03-24 | Genzyme Corporation | Wektory wirusowe związane z adenowirusami do leczenia jaskry miocylinowej (myoc) |
| EP4012035B1 (en) * | 2014-09-16 | 2024-11-06 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| KR20220076510A (ko) | 2019-10-08 | 2022-06-08 | 트러스티스 오브 보스톤 칼리지 | 다수의 서로 다른 비천연 아미노산을 함유하는 단백질 및 이러한 단백질의 제조 및 사용 방법 |
| CN115484978A (zh) | 2020-03-05 | 2022-12-16 | 尼奥克斯医疗有限公司 | 使用免疫细胞治疗癌症的方法和组合物 |
| WO2021245224A1 (en) * | 2020-06-05 | 2021-12-09 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for treating ocular diseases |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE318923T1 (de) * | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| US20040198682A1 (en) * | 2001-11-30 | 2004-10-07 | Mcswiggen James | RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (siNA) |
| US20050048529A1 (en) * | 2002-02-20 | 2005-03-03 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA) |
| US20040209832A1 (en) * | 2001-11-30 | 2004-10-21 | Mcswiggen James | RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA) |
| WO2005019422A2 (en) * | 2003-08-13 | 2005-03-03 | The Board Of Trustees Of The University Of Illinois | Silencing of tgf-beta receptor type ii expression by sirna |
| WO2006042252A2 (en) * | 2004-10-08 | 2006-04-20 | Potentia Pharmeceuticals, Inc. | Viral complement control proteins for eye disorders |
| TWI401316B (zh) * | 2004-12-23 | 2013-07-11 | Alcon Inc | 用於治療青光眼之血清澱粉樣蛋白Ａ的ＲＮＡｉ抑制作用 |
| TWI386225B (zh) * | 2004-12-23 | 2013-02-21 | Alcon Inc | 用於治療眼睛病症的結締組織生長因子（ＣＴＧＦ）ＲＮＡ干擾（ＲＮＡｉ）抑制技術 |
| TW200639252A (en) * | 2005-02-01 | 2006-11-16 | Alcon Inc | RNAi-mediated inhibition of ocular hypertension targets |
| WO2006099353A1 (en) * | 2005-03-11 | 2006-09-21 | Alcon, Inc. | Rnai-mediated inhibition of frizzled related protein-1 for treatment of glaucoma |
| GB0521351D0 (en) * | 2005-10-20 | 2005-11-30 | Genomica Sau | Modulation of TRPV expression levels |
| AR057252A1 (es) * | 2005-12-27 | 2007-11-21 | Alcon Mfg Ltd | Inhibicion de rho quinasa mediada por arni para el tratamiento de trastornos oculares |
| TW200731980A (en) * | 2005-12-29 | 2007-09-01 | Alcon Mfg Ltd | RNAi-mediated inhibition of HIF1A for treatment of ocular angiogenesis |
| TW201336514A (zh) * | 2006-04-13 | 2013-09-16 | Alcon Res Ltd | ＲＮＡ干擾（ＲＮＡｉ）所媒介之與脾酪胺酸激酶相關之發炎症狀的抑制作用（二） |
| WO2007127428A2 (en) * | 2006-04-28 | 2007-11-08 | University Of Florida Research Foundation, Inc. | Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery |
| US7732421B2 (en) * | 2006-05-19 | 2010-06-08 | Alcon Research, Ltd. | RNAI-mediated inhibition of tumor necrosis factor α-related conditions |
| KR20090042297A (ko) * | 2006-08-24 | 2009-04-29 | 알콘 리서치, 리미티드 | ＩＯＰ―관련 증상을 치료하기 위한 그렘린의 ＲＮＡｉ 매개 억제 |
| US7872119B2 (en) * | 2007-02-26 | 2011-01-18 | Quark Pharmaceuticals, Inc. | Inhibitors of RTP801 and their use in disease treatment |
| US7973019B1 (en) * | 2007-10-03 | 2011-07-05 | Alcon Research, Ltd. | Transferrin/transferrin receptor-mediated siRNA delivery |
| TW200932274A (en) * | 2007-12-18 | 2009-08-01 | Alcon Res Ltd | Interfering RNA delivery system and uses thereof |
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2008
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- 2008-10-01 WO PCT/US2008/078380 patent/WO2009046059A1/en not_active Ceased
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- 2008-10-01 MX MX2010001608A patent/MX2010001608A/es not_active Application Discontinuation
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2010
- 2010-01-20 ZA ZA2010/00423A patent/ZA201000423B/en unknown
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| JPN6013023940; 'Ｓｕｐｐｒｅｓｓｉｏｎ ｏｆ ｍｏｕｓｅ ｒｈｏｄｏｐｓｉｎ ｅｘｐｒｅｓｓｉｏｎ ｉｎ ｖｉｖｏ ｂｙ ＡＡＶ ｍｅｄｉａｔｅｄ ｓｉＲＮＡ ｄｅｌｉｖｅｒｙ．' Ｖｉｓｉｏｎ Ｒｅｓ． ４７（９）, 2007, １２０２-１２０８ * |
| JPN6013023942; 'Ｏｃｕｌａｒ ｇｅｎｅ ｔｒａｎｓｆｅｒ ｗｉｔｈ ｓｅｌｆ-ｃｏｍｐｌｅｍｅｎｔａｒｙ ＡＡＶ ｖｅｃｔｏｒｓ．' Ｉｎｖｅｓｔ Ｏｐｈｔｈａｌｍｏｌ Ｖｉｓ Ｓｃｉ． ４８（７）, 2007, ３３２４-３３２８ * |
| JPN6013023944; 'Ｍｅｃｈａｎｉｓｍｓ ｏｆ ＡＡＶ ｔｒａｎｓｄｕｃｔｉｏｎ ｉｎ ｇｌａｕｃｏｍａ-ａｓｓｏｃｉａｔｅｄ ｈｕｍａｎ ｔｒａｂｅｃｕｌａｒ ｍｅｓｈｗｏｒｋ ｃｅｌｌｓ．' Ｊ Ｇｅｎｅ Ｍｅｄ． ８（５）, 2006, ５８９-６０２ * |
| JPN6013023945; 'ＡＡＶ ｈｙｂｒｉｄ ｓｅｒｏｔｙｐｅｓ： ｉｍｐｒｏｖｅｄ ｖｅｃｔｏｒｓ ｆｏｒ ｇｅｎｅ ｄｅｌｉｖｅｒｙ．' Ｃｕｒｒ Ｇｅｎｅ Ｔｈｅｒ． ５（３）, 2005, ２９９-３１０ * |
| JPN6013023948; 'Ｉｎｈｉｂｉｔｉｏｎ ｏｆ Ｏｃｕｌａｒ Ａｎｇｉｏｇｅｎｅｓｉｓ ｂｙ ｓｉＲＮＡ Ｔａｒｇｅｔｉｎｇ Ｖａｓｃｕｌａｒ Ｅｎｄｏｔｈｅｌｉａｌ Ｇｒｏｗｔｈ Ｆａｃｔｏｒ Ｐａｔｈｗａｙ Ｇｅｎｅ' Ａｍ Ｊ Ｐａｔｈｏｌ． １６５（６）, 2004, ２１７７-２１８５ * |
| JPN6013023949; 'Ｐｏｔｅｎｔｉａｌ ａｐｐｌｉｃａｔｉｏｎｓ ｆｏｒ ＲＮＡｉ ｔｏ ｐｒｏｂｅ ｐａｔｈｏｇｅｎｅｓｉｓ ａｎｄ ｄｅｖｅｌｏｐ ｎｅｗ ｔｒｅａｔｍｅｎｔｓ ｆｏｒ ｏｃｕｌａｒ ｄｉｓｏｒｄｅｒ' Ｇｅｎｅ Ｔｈｅｒａｐｙ １３（６）, 2006, ５５９-５６２ * |
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| KR20170054493A (ko) * | 2014-09-16 | 2017-05-17 | 젠자임 코포레이션 | 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 |
| JP2017529395A (ja) * | 2014-09-16 | 2017-10-05 | ジェンザイム・コーポレーション | ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター |
| KR102612871B1 (ko) | 2014-09-16 | 2023-12-13 | 젠자임 코포레이션 | 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 |
| KR20230172610A (ko) * | 2014-09-16 | 2023-12-22 | 젠자임 코포레이션 | 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 |
| KR102916865B1 (ko) | 2014-09-16 | 2026-01-23 | 젠자임 코포레이션 | 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 |
| JP2021526839A (ja) * | 2018-06-12 | 2021-10-11 | ザ ユニバーシティ オブ ブリストル | 眼圧及び頭蓋内圧を調節するための材料及び方法 |
| JP7430917B2 (ja) | 2018-06-12 | 2024-02-14 | ザ ユニバーシティ オブ ブリストル | 眼圧及び頭蓋内圧を調節するための材料及び方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| KR20100061792A (ko) | 2010-06-09 |
| CN101815536A (zh) | 2010-08-25 |
| CA2694091A1 (en) | 2009-04-09 |
| US20090087413A1 (en) | 2009-04-02 |
| AU2008308784A1 (en) | 2009-04-09 |
| ZA201000423B (en) | 2011-03-30 |
| BRPI0817937A2 (pt) | 2015-04-07 |
| RU2010117178A (ru) | 2011-11-10 |
| EP2192926A1 (en) | 2010-06-09 |
| AU2008308784B2 (en) | 2013-07-18 |
| WO2009046059A1 (en) | 2009-04-09 |
| MX2010001608A (es) | 2010-03-15 |
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