JP2004535801A - 筋特異的発現ベクター - Google Patents
筋特異的発現ベクター Download PDFInfo
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- JP2004535801A JP2004535801A JP2002592469A JP2002592469A JP2004535801A JP 2004535801 A JP2004535801 A JP 2004535801A JP 2002592469 A JP2002592469 A JP 2002592469A JP 2002592469 A JP2002592469 A JP 2002592469A JP 2004535801 A JP2004535801 A JP 2004535801A
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| JP2021168663A (ja) * | 2014-01-21 | 2021-10-28 | フレイエ ユニヴェルシテイト ブリュッセルVrije Universiteit Brussel | 筋特異的核酸調節エレメント並びにその方法及び使用 |
| JP2022010079A (ja) * | 2015-09-17 | 2022-01-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2023543154A (ja) * | 2020-09-11 | 2023-10-13 | アルデブロン，エル．エル．シー． | 筋特異的ハイブリッドプロモーター |
| JP2024504412A (ja) * | 2021-01-27 | 2024-01-31 | ザ・ユニバーシティ・コート・オブ・ザ・ユニバーシティ・オブ・エディンバラ | 機能性核酸分子および方法 |
Families Citing this family (63)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
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| JP2025526502A (ja) * | 2022-07-11 | 2025-08-13 | ヴァイタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための治療用サテライト細胞の製造 |
| JP2025535203A (ja) | 2022-09-15 | 2025-10-23 | ボイジャー セラピューティクス インコーポレイテッド | タウ結合化合物 |
| AU2024287813A1 (en) | 2023-07-07 | 2026-01-22 | Kriya Therapeutics, Inc. | Periocular delivery of aav vectors for treating ophthalmic pathologies |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5266488A (en) * | 1988-04-06 | 1993-11-30 | The Regents Of The University Of California | Troponin T gene promoter and derivatives thereof |
| US5795872A (en) * | 1995-09-19 | 1998-08-18 | Pharmadigm, Inc. | DNA construct for immunization |
| US5846528A (en) * | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
| US5858351A (en) * | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
| DE69918090T2 (de) * | 1998-04-24 | 2005-06-16 | University Of Florida, Gainesville | Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie |
| WO2001083695A2 (en) * | 2000-04-28 | 2001-11-08 | Xiao Xiao | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| US6593110B2 (en) * | 2000-05-04 | 2003-07-15 | California Institute Of Technology | Checkpoint-activating oligonucleotides |
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2002
- 2002-05-22 EP EP02739449A patent/EP1390490B1/en not_active Expired - Lifetime
- 2002-05-22 AT AT02739449T patent/ATE428777T1/de not_active IP Right Cessation
- 2002-05-22 WO PCT/US2002/016748 patent/WO2002095006A2/en not_active Ceased
- 2002-05-22 DE DE60231975T patent/DE60231975D1/de not_active Expired - Lifetime
- 2002-05-22 JP JP2002592469A patent/JP2004535801A/ja active Pending
- 2002-05-22 EP EP08103273A patent/EP2017338A1/en not_active Withdrawn
- 2002-05-22 CA CA002448120A patent/CA2448120A1/en not_active Abandoned
- 2002-05-24 US US10/156,604 patent/US20030100526A1/en not_active Abandoned
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2007
- 2007-03-12 US US11/716,899 patent/US20110212529A1/en not_active Abandoned
Cited By (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021168663A (ja) * | 2014-01-21 | 2021-10-28 | フレイエ ユニヴェルシテイト ブリュッセルVrije Universiteit Brussel | 筋特異的核酸調節エレメント並びにその方法及び使用 |
| JP7235806B2 (ja) | 2014-01-21 | 2023-03-08 | フレイエ ユニヴェルシテイト ブリュッセル | 筋特異的核酸調節エレメント並びにその方法及び使用 |
| JP2023062192A (ja) * | 2014-01-21 | 2023-05-02 | フレイエ ユニヴェルシテイト ブリュッセル | 筋特異的核酸調節エレメント並びにその方法及び使用 |
| JP2022010079A (ja) * | 2015-09-17 | 2022-01-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2023543154A (ja) * | 2020-09-11 | 2023-10-13 | アルデブロン，エル．エル．シー． | 筋特異的ハイブリッドプロモーター |
| JP7846677B2 (ja) | 2020-09-11 | 2026-04-15 | アルデブロン，エル．エル．シー． | 筋特異的ハイブリッドプロモーター |
| JP2024504412A (ja) * | 2021-01-27 | 2024-01-31 | ザ・ユニバーシティ・コート・オブ・ザ・ユニバーシティ・オブ・エディンバラ | 機能性核酸分子および方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| EP1390490B1 (en) | 2009-04-15 |
| WO2002095006A2 (en) | 2002-11-28 |
| US20030100526A1 (en) | 2003-05-29 |
| DE60231975D1 (de) | 2009-05-28 |
| CA2448120A1 (en) | 2002-11-28 |
| ATE428777T1 (de) | 2009-05-15 |
| EP1390490A2 (en) | 2004-02-25 |
| WO2002095006A3 (en) | 2003-03-13 |
| EP1390490A4 (en) | 2005-05-11 |
| EP2017338A1 (en) | 2009-01-21 |
| US20110212529A1 (en) | 2011-09-01 |
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