JP2024019727A5 - - Google Patents

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Publication number
JP2024019727A5
JP2024019727A5 JP2023223638A JP2023223638A JP2024019727A5 JP 2024019727 A5 JP2024019727 A5 JP 2024019727A5 JP 2023223638 A JP2023223638 A JP 2023223638A JP 2023223638 A JP2023223638 A JP 2023223638A JP 2024019727 A5 JP2024019727 A5 JP 2024019727A5
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JP
Japan
Prior art keywords
sgrna
nme
nucleotides
length
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023223638A
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English (en)
Japanese (ja)
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JP2024019727A (ja
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Publication date
Priority claimed from JP2020525971A external-priority patent/JP2021502097A/ja
Application filed filed Critical
Publication of JP2024019727A publication Critical patent/JP2024019727A/ja
Publication of JP2024019727A5 publication Critical patent/JP2024019727A5/ja
Pending legal-status Critical Current

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

Applications Claiming Priority (8)

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
JP2020525971A JP2021502097A (ja) 2017-11-10 2018-11-09 標的化crispr送達プラットフォーム
PCT/US2018/060126 WO2019094791A2 (en) 2017-11-10 2018-11-09 Targeted crispr delivery platforms

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2020525971A Division JP2021502097A (ja) 2017-11-10 2018-11-09 標的化crispr送達プラットフォーム

Publications (2)

Publication Number Publication Date
JP2024019727A JP2024019727A (ja) 2024-02-09
JP2024019727A5 true JP2024019727A5 (enExample) 2025-04-17

Family

ID=66438139

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020525971A Withdrawn JP2021502097A (ja) 2017-11-10 2018-11-09 標的化crispr送達プラットフォーム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 Before (1)

Application Number Title Priority Date Filing Date
JP2020525971A Withdrawn JP2021502097A (ja) 2017-11-10 2018-11-09 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)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
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
EP3921638A4 (en) 2019-02-05 2022-08-24 AMMR Joint Venture EXPANDED CHOICE OF EFFICIENT TARGETED GENE MANIPULATORS
US20220298494A1 (en) * 2019-02-14 2022-09-22 Metagenomi Ip Technologies, Llc Enzymes with ruvc domains
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
AU2022335499A1 (en) 2021-08-27 2024-02-22 Metagenomi Therapeutics, Inc. Enzymes with ruvc domains
WO2023064813A2 (en) * 2021-10-13 2023-04-20 University Of Massachusetts Modified guide rnas for neisseria meningitidis cas9
CA3237303A1 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
IL312507A (en) * 2021-11-03 2024-07-01 Intellia Therapeutics Inc Modified guide rnas for gene editing
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
KR20250124819A (ko) 2022-12-21 2025-08-20 인텔리아 테라퓨틱스, 인크. 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법
TW202436622A (zh) 2023-03-06 2024-09-16 美商英特利亞醫療公司 用於b型肝炎病毒(hbv)基因體編輯之組合物及方法
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
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)
IL326191A (en) 2023-08-14 2026-03-01 Intellia Therapeutics Inc Compositions and methods for genetic modification of 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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
RU2716420C2 (ru) * 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
EP3666892A1 (en) * 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
CA2930877A1 (en) * 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
JP2016538001A (ja) * 2013-11-28 2016-12-08 ホライズン・ジェノミクス・ゲーエムベーハー 体細胞半数体ヒト細胞株
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
EP3080259B1 (en) * 2013-12-12 2023-02-01 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
WO2016179038A1 (en) * 2015-05-01 2016-11-10 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
BR112019028146A2 (pt) * 2017-07-31 2020-07-07 Sigma-Aldrich Co. Llc rna guia sintético para sistemas ativadores de crispr/cas

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