JP2001506133A - 組換えaavベクターの産生における使用のための、aavスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株 - Google Patents
組換えaavベクターの産生における使用のための、aavスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株Info
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Abstract
Description
Claims (1)
- 【特許請求の範囲】 1.組換えアデノ随伴ウイルス(rAAV)ベクターの高効率パッケージングに有用な 哺乳動物細胞であって、該細胞は以下のAAVスプリットパッケージング遺伝子: (i)AAV split-cap遺伝子であって、ここで、AAVrep遺伝子のRep78-特異的配 列から分離し、そして該split-cap遺伝子が異種プロモーターに作動可能に連結 したAAVsplit-cap遺伝子;および (ii)AAVrep78遺伝子であって、ここでAAV cap遺伝子のCap特異的配列から分 離し、そして該rep78遺伝子が異種プロモーターに作動可能に連結したAAV rep78 遺伝子、 の各々の少なくとも一つのコピーを含む、哺乳動物細胞。 2.AAVrep52遺伝子をさらに含む、請求項1に記載の哺乳動物細胞であって、該 rep52遺伝子が、AAVrep遺伝子のRep78特異的配列から分離し、そして該rep52遺 伝子がプロモーターに作動可能に連結した、哺乳動物細胞。 3.前記プロモーターの少なくとも一つが誘導性の異種プロモーターである、請 求項1に記載の哺乳動物細胞。 4.前記AAVスプリットパッケージング遺伝子の少なくとも一つが、異種エンハ ンサーに作動可能に連結した、請求項1に記載の哺乳動物細胞。 5.前記AAVスプリットパッケージング遺伝子の少なくとも一つが、前記細胞に 安定に組み込まれた、請求項1に記載の哺乳動物細胞。 6.少なくとも二つの異なるAAVスプリットパッケージング遺伝子が、前記細胞 中に安定に組み込まれた、請求項1に記載の哺乳動物細胞。 7.少なくとも三つの異なるAAVスプリットパッケージング遺伝子が前記細胞中 に安定に組み込まれた、請求項1に記載の哺乳動物細胞。 8.組換えAAVベクターをさらに含む、請求項1に記載の哺乳動物細胞。 9.前記目的の異種遺伝子が治療遺伝子である、請求項8に記載の哺乳動物細胞 。 10.前記治療遺伝子が嚢包性繊維症膜貫通調節タンパク質をコードする、請求 項9に記載の哺乳動物細胞。 11.前記哺乳動物細胞が、1細胞当たり少なくとも約100個の組換えAAV粒子を 産生し得る、請求項1に記載の哺乳動物細胞。 12.前記哺乳動物細胞が、1細胞当たり少なくとも約200個の組換えAAV粒子を 産生し得る、請求項1に記載の哺乳動物細胞。 13.前記細胞が、1細胞当たり少なくとも約400個の組換えAAV粒子を産生し得 る、請求項1に記載の哺乳動物細胞。 14.前記哺乳動物細胞が、ヒトの細胞である、請求項1に記載の哺乳動物細胞 。 15.AAVスプリットパッケージング細胞の調製に有用であるポリヌクレオチド 発現ベクターであって、該発現ベクターは、選択マーカーおよび以下からなる群 より選択される少なくとも一つのAAVスプリットパッケージング遺伝子: (i)AAV sprit cap遺伝子であって、該split-cap遺伝子がAAV rep遺伝子のRep 78特異的配列から分離し、そして該split-cap遺伝子が異種プロモーターに作動 可能に連結した、AAV split-cap遺伝子; (ii)AAV rep78遺伝子であって、該rep78遺伝子がAAV cap遺伝子のCap特異的 配列から分離し、そして該rep78遺伝子が異種誘導性プロモーターに作動可能に 連結した、AAV rep78遺伝子;および (iii)AAV rep52遺伝子であって、該rep52遺伝子がAAV rep遺伝子のRep78特 異的配列から分離し、そして該rep52遺伝子が異種プロモーターに作動可能に連 結した、AAV rep52遺伝子; を含む、ポリヌクレオチド発現ベクター。 16.異種プロモーターに作動可能に連結した、AAV split-cap遺伝子を含む、 請求項15に記載のポリヌクレオチド発現ベクター。 17.異種プロモーターに作動可能に連結したAAV rep78遺伝子を含む、請求項 15に記載のポリヌクレオチド発現ベクター。 18.異種プロモーターに作動可能に連結したAAV rep52遺伝子を含む、請求項 15に記載のポリヌクレオチド発現ベクター。 19.前記発現ベクターが、以下の二つのAAVスプリットパッケージング遺伝子 : (i)AAV split-cap遺伝子であって、該split-cap遺伝子がAAV rep遺伝子のRep 78特異的配列から分離し、そして該split-cap遺伝子が異種プロモーターに作動 可能に連結した、AAV split-cap遺伝子;および (ii)AAV rep52遺伝子であって、該rep52遺伝子がAAV rep遺伝子のRep78特異 的配列から分離し、そして該rep52遺伝子が異種プロモーターに作動可能に連結 した、AAV rep52遺伝子; を含む、請求項15に記載のポリヌクレオチド発現ベクター。 20.前記AAV split-cap遺伝子および前記AAVrep52遺伝子が、縦列の転写配向 に配置された、請求項19に記載のポリヌクレオチド発現ベクター。 21.前記AAVsplit-cap遺伝子および前記AAV rep52遺伝子が、異なる(divergen t)転写の配向に配置された、請求項19に記載のポリヌクレオチド発現ベクター 。 22.組換えアデノ随伴ウイルス(rAAV)ベクターの高効率パッケージングに有用 である哺乳動物細胞を産生する方法であって、請求項15に記載のポリヌクレオ チド発現ベクターを該細胞へ導入する工程を包含する、方法。 23.rAAVベクターの高効率パッケージングに有用である哺乳動物細胞を産生す る方法であって、請求項15に記載の少なくとも二つの異なるポリヌクレオチド 発現ベクターを該細胞へ導入する工程を包含する、方法。 24.rAAVベクターの高効率パッケージングに有用である哺乳動物細胞を産生す る方法であって、請求項15に記載の少なくとも二つの異なるポリヌクレオチド 発現ベクターを該細胞へ導入する工程を包含する、方法。 25.組換えアデノ随伴ウイルス(rAAV)ベクターの高効率パッケージングに有用 である哺乳動物細胞を産生する方法であって、請求項15に記載のポリヌクレオ チド発現ベクターを該細胞へ安定に導入する工程を包含する、方法。 26.rAAVベクターの高効率パッケージングに有用である哺乳動物細胞を産生す る方法であって、請求項15に記載の少なくとも二つの異なるポリヌクレオチド 発現ベクターを該細胞へ安定に導入する工程を包含する、方法。 27.rAAVベクターの高効率パッケージングに有用である哺乳動物細胞を産生す る方法であって、請求項15に記載の少なくとも二つの異なるポリヌクレオチド 発現ベクターを該細胞へ安定に導入する工程を包含する、方法。 28.請求項22に記載の方法により産生される、哺乳動物細胞。
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
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US4160996P | 1996-12-18 | 1996-12-18 | |
US60/041,609 | 1996-12-18 | ||
PCT/US1997/023247 WO1998027204A2 (en) | 1996-12-18 | 1997-12-12 | Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors |
Publications (2)
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JP2001506133A true JP2001506133A (ja) | 2001-05-15 |
JP2001506133A5 JP2001506133A5 (ja) | 2005-08-11 |
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Application Number | Title | Priority Date | Filing Date |
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JP52792698A Ceased JP2001506133A (ja) | 1996-12-18 | 1997-12-12 | 組換えaavベクターの産生における使用のための、aavスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株 |
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Country | Link |
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US (1) | US6541258B2 (ja) |
EP (1) | EP0950091A2 (ja) |
JP (1) | JP2001506133A (ja) |
AU (1) | AU741605B2 (ja) |
CA (1) | CA2270285A1 (ja) |
WO (1) | WO1998027204A2 (ja) |
Cited By (1)
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FR2716682B1 (fr) * | 1994-01-28 | 1996-04-26 | Centre Nat Rech Scient | Procédé de préparation de virus adéno-associés (AAV) recombinants et utilisations. |
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1997
- 1997-12-12 EP EP97952482A patent/EP0950091A2/en not_active Withdrawn
- 1997-12-12 WO PCT/US1997/023247 patent/WO1998027204A2/en not_active Application Discontinuation
- 1997-12-12 US US08/981,172 patent/US6541258B2/en not_active Expired - Fee Related
- 1997-12-12 CA CA002270285A patent/CA2270285A1/en not_active Abandoned
- 1997-12-12 JP JP52792698A patent/JP2001506133A/ja not_active Ceased
- 1997-12-12 AU AU56076/98A patent/AU741605B2/en not_active Ceased
Cited By (1)
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WO2023182476A1 (ja) * | 2022-03-25 | 2023-09-28 | 学校法人自治医科大学 | アデノ随伴ウイルス(aav)ベクターの効率の良い産生システム |
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CA2270285A1 (en) | 1998-06-25 |
US20020081721A1 (en) | 2002-06-27 |
AU741605B2 (en) | 2001-12-06 |
EP0950091A2 (en) | 1999-10-20 |
AU5607698A (en) | 1998-07-15 |
US6541258B2 (en) | 2003-04-01 |
WO1998027204A3 (en) | 1998-07-30 |
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