JP7520826B2 - 導入遺伝子を送達するための組成物および方法 - Google Patents

導入遺伝子を送達するための組成物および方法 Download PDF

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JP7520826B2
JP7520826B2 JP2021521116A JP2021521116A JP7520826B2 JP 7520826 B2 JP7520826 B2 JP 7520826B2 JP 2021521116 A JP2021521116 A JP 2021521116A JP 2021521116 A JP2021521116 A JP 2021521116A JP 7520826 B2 JP7520826 B2 JP 7520826B2
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nucleic acid
sequence
protein
grna
cell
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JP2022505173A (ja
JPWO2020081843A5 (https=
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リチャード ブルックス，アラン
ヴォー，カレン
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CRISPR Therapeutics AG
Bayer Healthcare LLC
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CRISPR Therapeutics AG
Bayer Healthcare LLC
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2021521116A 2018-10-17 2019-10-17 導入遺伝子を送達するための組成物および方法 Active JP7520826B2 (ja)

Priority Applications (1)

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JP2024110847A JP2024161350A (ja) 2018-10-17 2024-07-10 導入遺伝子を送達するための組成物および方法

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US201862747128P 2018-10-17 2018-10-17
US62/747,128 2018-10-17
PCT/US2019/056779 WO2020081843A1 (en) 2018-10-17 2019-10-17 Compositions and methods for delivering transgenes

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JP2022505173A JP2022505173A (ja) 2022-01-14
JPWO2020081843A5 JPWO2020081843A5 (https=) 2022-11-30
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JP7520826B2 true JP7520826B2 (ja) 2024-07-23

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CN (1) CN113366106A (https=)
AU (1) AU2019362000A1 (https=)
BR (1) BR112021007403A2 (https=)
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US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
IL302335A (en) * 2020-10-26 2023-06-01 Res Inst Nationwide Childrens Hospital NK cells with chimeric antigen receptor (CAR) and uses thereof
CN114496063B (zh) * 2022-01-13 2023-05-23 北京博康健基因科技有限公司 基于天然氨基酸序列的信号肽设计与二级结构从头算建模方法和装置
US20250382640A1 (en) * 2022-06-29 2025-12-18 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids
CN117384880A (zh) * 2022-07-08 2024-01-12 北京干细胞与再生医学研究院 工程化的核酸修饰编辑器
CN117947040A (zh) * 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用
WO2024157194A1 (en) * 2023-01-25 2024-08-02 Crispr Therapeutics Ag Methods and assays for off-target analysis
CN116970087B (zh) * 2023-05-11 2025-01-24 百葵锐(深圳)生物科技有限公司 一种可用于植物叶面定向结合及递送的蛋白rna复合物
EP4574975A1 (en) 2023-12-20 2025-06-25 Bayer AG Compositions and methods for delivering transgenes
WO2026064425A2 (en) 2024-09-17 2026-03-26 Dxome Co., Ltd. Mirror image crispr-cas compositions

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JP2016521975A (ja) 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ， インコーポレイテッド 遺伝的状態の処置のための方法および組成物
JP2021500072A (ja) 2017-10-17 2021-01-07 クリスパー セラピューティクス アクチェンゲゼルシャフト 血友病ａに対する遺伝子編集用組成物及び方法

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