JP2022512974A - 生体内crispr超抑制因子による合成免疫調節 - Google Patents

生体内crispr超抑制因子による合成免疫調節 Download PDF

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JP2022512974A
JP2022512974A JP2021525082A JP2021525082A JP2022512974A JP 2022512974 A JP2022512974 A JP 2022512974A JP 2021525082 A JP2021525082 A JP 2021525082A JP 2021525082 A JP2021525082 A JP 2021525082A JP 2022512974 A JP2022512974 A JP 2022512974A
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myd88
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サミラ キアニ
モ レザ エブラヒムカニ
ファルザネ モガダム
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アリゾナ ボード オブ リージェンツ オン ビハーフ オブ アリゾナ ステート ユニバーシティ
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JP2021525082A 2018-11-08 2019-11-07 生体内crispr超抑制因子による合成免疫調節 Pending JP2022512974A (ja)

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US201862757679P 2018-11-08 2018-11-08
US62/757,679 2018-11-08
US201962899584P 2019-09-12 2019-09-12
US62/899,584 2019-09-12
PCT/US2019/060285 WO2020097344A1 (en) 2018-11-08 2019-11-07 Synthetic immunomodulation with a crispr super-repressor in vivo

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Publication number Priority date Publication date Assignee Title
US11773407B2 (en) 2017-06-26 2023-10-03 Arizona Board Of Regents On Behalf Of Arizona State University CRISPR logic circuits for safer and controllable gene therapies
US20200123567A1 (en) 2017-06-26 2020-04-23 Samira Kiani A universal platform to enhance crispr-based gene editing for in vivo therapies
WO2023288306A2 (en) * 2021-07-16 2023-01-19 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeting myd88 gene in vitro and in vivo
EP4479538A4 (en) * 2022-02-17 2026-01-28 Syntax Bio Inc CELL PROGRAMMING SYSTEMS AND ASSOCIATED PROCESSES
EP4711456A1 (en) * 2024-09-11 2026-03-18 GenCC GmbH & Co. KG Method for the manufacture of individualized crispr/cas complexes and individualized crispr/cas complexes

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014506896A (ja) * 2011-03-02 2014-03-20 ▲華▼中科技大学同▲済▼医学院附属同▲済▼医院 アミノチアゾールmyd88特異的阻害剤の薬学的な使用
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016094872A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
JP2017506232A (ja) * 2014-02-07 2017-03-02 Sbiバイオテック株式会社 腫瘍を治療するための阻害性オリゴヌクレオチド
JP2017532001A (ja) * 2013-12-12 2017-11-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物
JP2018519244A (ja) * 2015-07-08 2018-07-19 リサーチ アンド ビジネス ファウンデーション ソンギュンクァン ユニバーシティ ピロリジンカルボキサミド誘導体ならびにその調製および使用方法

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20030148986A1 (en) * 2001-12-17 2003-08-07 Cedars-Sinai Medical Center Methods for treating vascular disease by inhibiting myeloid differentiation factor 88
US9228207B2 (en) * 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
TWI906646B (zh) * 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的crispr酶突變
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
US11439692B2 (en) * 2017-05-17 2022-09-13 Modalis Therapeutics Corporation Method of treating diseases associated with MYD88 pathways using CRISPR-GNDM system
US11773407B2 (en) * 2017-06-26 2023-10-03 Arizona Board Of Regents On Behalf Of Arizona State University CRISPR logic circuits for safer and controllable gene therapies

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014506896A (ja) * 2011-03-02 2014-03-20 ▲華▼中科技大学同▲済▼医学院附属同▲済▼医院 アミノチアゾールmyd88特異的阻害剤の薬学的な使用
JP2017532001A (ja) * 2013-12-12 2017-11-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物
JP2017506232A (ja) * 2014-02-07 2017-03-02 Sbiバイオテック株式会社 腫瘍を治療するための阻害性オリゴヌクレオチド
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016094872A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
JP2018519244A (ja) * 2015-07-08 2018-07-19 リサーチ アンド ビジネス ファウンデーション ソンギュンクァン ユニバーシティ ピロリジンカルボキサミド誘導体ならびにその調製および使用方法

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ACS CHEMICAL BIOLOGY, vol. 13, JPN6023040017, 16 October 2017 (2017-10-16), pages 406 - 416, ISSN: 0005162238 *
NATURE METHODS, vol. 15, JPN6023040018, August 2018 (2018-08-01), pages 611 - 616, ISSN: 0005162237 *
NATURE REVIEWS MOLECULAR CELL BIOLOGY, vol. 17, JPN6023040016, 2016, pages 5 - 15, ISSN: 0005162239 *

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EP3877398A1 (en) 2021-09-15
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CN113330023A (zh) 2021-08-31
KR20210102247A (ko) 2021-08-19
EP3877398A4 (en) 2022-08-17
US11884925B2 (en) 2024-01-30
WO2020097344A1 (en) 2020-05-14
US20210380990A1 (en) 2021-12-09
CA3119302A1 (en) 2020-05-14
AU2019375914A1 (en) 2021-06-03
US20240117364A1 (en) 2024-04-11

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