JP2021502097A5 - - Google Patents

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Publication number
JP2021502097A5
JP2021502097A5 JP2020525971A JP2020525971A JP2021502097A5 JP 2021502097 A5 JP2021502097 A5 JP 2021502097A5 JP 2020525971 A JP2020525971 A JP 2020525971A JP 2020525971 A JP2020525971 A JP 2020525971A JP 2021502097 A5 JP2021502097 A5 JP 2021502097A5
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JP
Japan
Prior art keywords
nucleotides
sgrna
sequence
composition
shortened
Prior art date
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JP2020525971A
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English (en)
Japanese (ja)
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JP2021502097A (ja
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Priority claimed from PCT/US2018/060126 external-priority patent/WO2019094791A2/en
Publication of JP2021502097A publication Critical patent/JP2021502097A/ja
Publication of JP2021502097A5 publication Critical patent/JP2021502097A5/ja
Priority to JP2023223638A priority Critical patent/JP2024019727A/ja
Withdrawn legal-status Critical Current

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JP2020525971A 2017-11-10 2018-11-09 標的化ｃｒｉｓｐｒ送達プラットフォーム Withdrawn JP2021502097A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023223638A JP2024019727A (ja) 2017-11-10 2023-12-28 標的化ｃｒｉｓｐｒ送達プラットフォーム

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762584310P 2017-11-10 2017-11-10
US62/584,310 2017-11-10
US201762596375P 2017-12-08 2017-12-08
US62/596,375 2017-12-08
US201862667084P 2018-05-04 2018-05-04
US62/667,084 2018-05-04
PCT/US2018/060126 WO2019094791A2 (en) 2017-11-10 2018-11-09 Targeted crispr delivery platforms

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023223638A Division JP2024019727A (ja) 2017-11-10 2023-12-28 標的化ｃｒｉｓｐｒ送達プラットフォーム

Publications (2)

Publication Number Publication Date
JP2021502097A JP2021502097A (ja) 2021-01-28
JP2021502097A5 true JP2021502097A5 (enExample) 2021-12-02

Family

ID=66438139

Family Applications (2)

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JP2020525971A Withdrawn JP2021502097A (ja) 2017-11-10 2018-11-09 標的化ｃｒｉｓｐｒ送達プラットフォーム
JP2023223638A Pending JP2024019727A (ja) 2017-11-10 2023-12-28 標的化ｃｒｉｓｐｒ送達プラットフォーム

Family Applications After (1)

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JP2023223638A Pending JP2024019727A (ja) 2017-11-10 2023-12-28 標的化ｃｒｉｓｐｒ送達プラットフォーム

Country Status (13)

Country Link
US (3) US20220389447A9 (enExample)
EP (1) EP3707254A4 (enExample)
JP (2) JP2021502097A (enExample)
KR (2) KR102922730B1 (enExample)
CN (1) CN111868240A (enExample)
AU (2) AU2018364993B2 (enExample)
BR (1) BR112020009268A2 (enExample)
CA (1) CA3082370A1 (enExample)
CO (1) CO2020007046A2 (enExample)
IL (2) IL317505A (enExample)
MX (1) MX2020004777A (enExample)
SG (1) SG11202005103RA (enExample)
WO (1) WO2019094791A2 (enExample)

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Publication number Priority date Publication date Assignee Title
JP7250031B2 (ja) * 2018-09-25 2023-03-31 公益財団法人微生物化学研究会 新規ウイルスベクターおよびその製造方法と使用方法
CN113661392B (zh) 2019-02-05 2025-05-27 卡蒂亚生物公司 有效靶向基因组操纵剂的增强选择
CN113728097A (zh) * 2019-02-14 2021-11-30 宏基因组学知识产权技术有限责任公司 具有ruvc结构域的酶
CN110257406B (zh) * 2019-06-21 2022-07-22 安徽省农业科学院水稻研究所 密码子植物化改造的Plant Nme2Cas9基因及其应用
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
WO2021122944A1 (en) 2019-12-18 2021-06-24 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
CN113652411B (zh) * 2021-07-30 2025-01-10 复旦大学 Cas9蛋白、含有Cas9蛋白的基因编辑系统及应用
CN119452085A (zh) 2021-08-27 2025-02-14 宏基因组学公司 具有ruvc结构域的酶
US20230183687A1 (en) * 2021-10-13 2023-06-15 University Of Massachusetts Modified guide rnas for neisseria meningitidis cas9
EP4426836A1 (en) * 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
EP4426822A2 (en) 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
JP2026506287A (ja) 2022-12-21 2026-02-24 インテリア セラピューティクス，インコーポレイテッド プロタンパク質転換酵素サブチリシンケキシン９（ｐｃｓｋ９）編集のための組成物及び方法
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
TW202515994A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對ｃｄ７０進行基因修飾之組合物及方法
WO2025038648A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2026163162A1 (en) 2025-01-31 2026-08-06 Intellia Therapeutics, Inc. Enhanced cas9 variants and uses thereof

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2728782T3 (es) * 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
CN105517579B (zh) * 2013-07-10 2019-11-15 哈佛大学校长及研究员协会 用于RNA向导的基因调节和编辑的正交Cas9蛋白
EP3760719A1 (en) * 2013-11-18 2021-01-06 CRISPR Therapeutics AG Crispr-cas system materials and methods
US10450586B2 (en) * 2013-11-28 2019-10-22 Horizon Discovery Limited Somatic haploid human cell line
JP6793547B2 (ja) * 2013-12-12 2020-12-02 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
WO2015089473A1 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
US20160346359A1 (en) * 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
US11732258B2 (en) * 2016-11-02 2023-08-22 President And Fellows Of Harvard College Engineered guide RNA sequences for in situ detection and sequencing
SG11201912024RA (en) * 2017-07-31 2020-02-27 Sigma Aldrich Co Llc Synthetic guide rna for crispr/cas activator systems

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