JP2020500539A - 標的特異的ヌクレアーゼの送達 - Google Patents

標的特異的ヌクレアーゼの送達 Download PDF

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JP2020500539A
JP2020500539A JP2019530472A JP2019530472A JP2020500539A JP 2020500539 A JP2020500539 A JP 2020500539A JP 2019530472 A JP2019530472 A JP 2019530472A JP 2019530472 A JP2019530472 A JP 2019530472A JP 2020500539 A JP2020500539 A JP 2020500539A
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formula
alkyl
lnp
independently
gene
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JP2020500539A5 (https=
Inventor
スティーブン エム． アンセル，
スティーブン エム． アンセル，
クリストファー バルボーサ，
クリストファー バルボーサ，
アンソニー コンウェイ，
アンソニー コンウェイ，
シンヤオ ドゥ，
シンヤオ ドゥ，
マイケル ジェイ． ホープ，
マイケル ジェイ． ホープ，
マイケル シー． ホームズ，
マイケル シー． ホームズ，
ゲイリー ケー． リー，
ゲイリー ケー． リー，
パウロ ジア チン リン，
パウロ ジア チン リン，
トーマス マッデン，
トーマス マッデン，
バーバラ ムイ，
バーバラ ムイ，
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Acuitas Therapeutics Inc
Sangamo Therapeutics Inc
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Acuitas Therapeutics Inc
Sangamo Therapeutics Inc
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Publication of JP2020500539A publication Critical patent/JP2020500539A/ja
Publication of JP2020500539A5 publication Critical patent/JP2020500539A5/ja
Priority to JP2022101160A priority Critical patent/JP2022121531A/ja
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2019530472A 2016-12-09 2017-12-08 標的特異的ヌクレアーゼの送達 Pending JP2020500539A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022101160A JP2022121531A (ja) 2016-12-09 2022-06-23 標的特異的ヌクレアーゼの送達

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662432042P 2016-12-09 2016-12-09
US62/432,042 2016-12-09
US201762458373P 2017-02-13 2017-02-13
US62/458,373 2017-02-13
US201762503470P 2017-05-09 2017-05-09
US62/503,470 2017-05-09
US201762559186P 2017-09-15 2017-09-15
US62/559,186 2017-09-15
PCT/US2017/065303 WO2018107026A1 (en) 2016-12-09 2017-12-08 Delivery of target specific nucleases

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JP2020500539A true JP2020500539A (ja) 2020-01-16
JP2020500539A5 JP2020500539A5 (https=) 2021-01-21

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JP2022101160A Pending JP2022121531A (ja) 2016-12-09 2022-06-23 標的特異的ヌクレアーゼの送達

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US (1) US20180185516A1 (https=)
EP (1) EP3551169A4 (https=)
JP (2) JP2020500539A (https=)
AU (1) AU2017374042C1 (https=)
CA (1) CA3045122A1 (https=)
WO (1) WO2018107026A1 (https=)

Cited By (5)

* Cited by examiner, † Cited by third party
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JP2020517694A (ja) * 2017-04-28 2020-06-18 アクイタス セラピューティクス インコーポレイテッド 新規なカルボニル脂質、および核酸を送達するための脂質ナノ粒子製剤
JP2020531418A (ja) * 2017-08-16 2020-11-05 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤における使用のための脂質
JP2023524666A (ja) * 2020-04-28 2023-06-13 インテリア セラピューティクス，インコーポレイテッド インビトロ細胞送達の方法
JP2023534035A (ja) * 2020-07-15 2023-08-07 ザ ジャクソン ラボラトリー ＳＡＲＳ－ＣｏＶ－２感染のためのヒト化マウスモデル
JP2023549011A (ja) * 2020-09-15 2023-11-22 ヴァーヴ・セラピューティクス，インコーポレーテッド 遺伝子編集のための脂質製剤

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PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
EP3995574A1 (en) 2016-08-24 2022-05-11 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
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