JP2015533786A - 遺伝子疾患の治療のための方法および組成物 - Google Patents

遺伝子疾患の治療のための方法および組成物 Download PDF

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Publication number
JP2015533786A
JP2015533786A JP2015530017A JP2015530017A JP2015533786A JP 2015533786 A JP2015533786 A JP 2015533786A JP 2015530017 A JP2015530017 A JP 2015530017A JP 2015530017 A JP2015530017 A JP 2015530017A JP 2015533786 A JP2015533786 A JP 2015533786A
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globin
gene
cell
sequence
cells
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JP2015533786A5 (enExample
Inventor
グレゴリー ジェイ． コスト，
グレゴリー ジェイ． コスト，
フィリップ ディー． グレゴリー，
フィリップ ディー． グレゴリー，
ドミトリー ガスチン，
ドミトリー ガスチン，
マイケル シー． ホームズ，
マイケル シー． ホームズ，
ジェフリー シー． ミラー，
ジェフリー シー． ミラー，
デイビッド パッション，
デイビッド パッション，
エドワード ジェイ． リーバー，
エドワード ジェイ． リーバー，
アンドレアス ライク，
アンドレアス ライク，
フィオードル ウルノフ，
フィオードル ウルノフ，
レイ ザン，
レイ ザン，
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C12N15/09—Recombinant DNA-technology
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    • C12N9/14—Hydrolases (3)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2015530017A 2012-08-29 2013-08-29 遺伝子疾患の治療のための方法および組成物 Pending JP2015533786A (ja)

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US201261694693P 2012-08-29 2012-08-29
US61/694,693 2012-08-29
PCT/US2013/057214 WO2014036219A2 (en) 2012-08-29 2013-08-29 Methods and compositions for treatment of a genetic condition

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JP2015530017A Pending JP2015533786A (ja) 2012-08-29 2013-08-29 遺伝子疾患の治療のための方法および組成物
JP2017180131A Pending JP2017217012A (ja) 2012-08-29 2017-09-20 遺伝子疾患の治療のための方法および組成物
JP2018093930A Expired - Fee Related JP6629917B2 (ja) 2012-08-29 2018-05-15 遺伝子疾患の治療のための方法および組成物
JP2021169438A Pending JP2022001072A (ja) 2012-08-29 2021-10-15 遺伝子疾患の治療のための方法および組成物

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JP2018093930A Expired - Fee Related JP6629917B2 (ja) 2012-08-29 2018-05-15 遺伝子疾患の治療のための方法および組成物
JP2021169438A Pending JP2022001072A (ja) 2012-08-29 2021-10-15 遺伝子疾患の治療のための方法および組成物

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CN (1) CN104704110B (enExample)
AU (1) AU2013308770B2 (enExample)
CA (2) CA3171494A1 (enExample)
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Cited By (2)

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JP2022528744A (ja) * 2019-04-12 2022-06-15 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作された造血幹細胞によるベータサラセミア表現型の補正
JP2022548399A (ja) * 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド 肝細胞核因子４－アルファ（ＨＮＦ４α）遺伝子発現をモジュレートするための組成物および方法

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JP2022548399A (ja) * 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド 肝細胞核因子４－アルファ（ＨＮＦ４α）遺伝子発現をモジュレートするための組成物および方法

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