JP2021526860A - 適応免疫をモジュレートするための組成物および方法 - Google Patents

適応免疫をモジュレートするための組成物および方法 Download PDF

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JP2021526860A
JP2021526860A JP2021518054A JP2021518054A JP2021526860A JP 2021526860 A JP2021526860 A JP 2021526860A JP 2021518054 A JP2021518054 A JP 2021518054A JP 2021518054 A JP2021518054 A JP 2021518054A JP 2021526860 A JP2021526860 A JP 2021526860A
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sequence
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polypeptide
grna
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デイビッド エー. ネルズ,
ランジャン バトラ,
ユージーン イェオ,
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ロックアネイビオ, インコーポレイテッド
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    • C12N15/1136Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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JP2021518054A 2018-06-08 2019-06-07 適応免疫をモジュレートするための組成物および方法 Pending JP2021526860A (ja)

Applications Claiming Priority (3)

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US201862682276P 2018-06-08 2018-06-08
US62/682,276 2018-06-08
PCT/US2019/036050 WO2019236998A1 (en) 2018-06-08 2019-06-07 Compositions and methods for the modulation of adaptive immunity

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JP2021526860A true JP2021526860A (ja) 2021-10-11
JPWO2019236998A5 JPWO2019236998A5 (https=) 2022-06-15
JP2021526860A5 JP2021526860A5 (https=) 2022-06-15

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US (2) US20190382759A1 (https=)
EP (1) EP3801641A4 (https=)
JP (1) JP2021526860A (https=)
KR (1) KR20210060429A (https=)
CN (1) CN113286619A (https=)
AU (1) AU2019281006A1 (https=)
CA (1) CA3102783A1 (https=)
SG (1) SG11202012015YA (https=)
WO (1) WO2019236998A1 (https=)

Families Citing this family (14)

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Publication number Priority date Publication date Assignee Title
US9499805B2 (en) 2010-06-18 2016-11-22 The University Of North Carolina At Chapel Hill Methods and compositions for synthetic RNA endonucleases
US20190284553A1 (en) 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
JP2021526858A (ja) 2018-06-08 2021-10-11 ロックアネイビオ, インコーポレイテッド Rna標的化融合タンパク質組成物および使用方法
AU2020208346B2 (en) 2019-01-14 2026-01-29 University Of Rochester Targeted nuclear RNA cleavage and polyadenylation with CRISPR-cas
WO2021011504A1 (en) * 2019-07-12 2021-01-21 Duke University Nanoparticle systems for targeted delivery of crispr/cas13 and methods of using same
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
AU2021392032A1 (en) 2020-12-03 2023-06-22 Century Therapeutics, Inc. Genetically engineered cells and uses thereof
CN113136376B (zh) * 2021-05-26 2022-10-21 武汉大学 一种Cas12a变体及其在基因编辑中的应用
WO2023150131A1 (en) * 2022-02-01 2023-08-10 The Regents Of The University Of California Method of regulating alternative polyadenylation in rna
CN114848808B (zh) * 2022-03-24 2023-04-25 四川大学 基于阳离子脂多肽及细胞因子的免疫增强剂及制法、应用
CN114720699A (zh) * 2022-04-22 2022-07-08 浙江大学 一种结直肠癌早期预警血清指标及其检测方法和应用
CN116949011A (zh) * 2022-04-26 2023-10-27 中国科学院动物研究所 经分离的Cas13蛋白、基于它的基因编辑系统及其用途
CN115068632A (zh) * 2022-06-22 2022-09-20 华中科技大学同济医学院附属同济医院 Ago2在制备治疗心衰的药物方面的用途及其蛋白、基因、转化体、药物与制备方法
WO2024264035A2 (en) * 2023-06-22 2024-12-26 The General Hospital Corporation Compositions and methods of generating endogenous tdr molecules

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170145394A1 (en) * 2015-11-23 2017-05-25 The Regents Of The University Of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12180499B2 (en) * 2016-10-31 2024-12-31 University Of Florida Research Foundation, Inc. Compositions and methods for impeding transcription of expanded microsatellite repeats
AU2018234825B2 (en) * 2017-03-15 2020-12-17 Massachusetts Institute Of Technology Novel CAS13B orthologues CRISPR enzymes and systems
US11168322B2 (en) * 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170145394A1 (en) * 2015-11-23 2017-05-25 The Regents Of The University Of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
WO2017091630A1 (en) * 2015-11-23 2017-06-01 The Regents Of The University Of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
CELL, vol. 173, JPN6024011778, 19 April 2018 (2018-04-19), pages 665 - 676, ISSN: 0005494732 *
CLIN CANCER RES., vol. 23, no. 9, JPN6023028518, 2017, pages 2255 - 2266, ISSN: 0005494731 *
MARY GEARING: "Cas13d: Small RNA-targeting CRISPR enzymes for transcriptome engineering", ADDGENE BLOG, インターネット, <URL: HTTPS://BLOG.ADDGENE.ORG/CAS13D-SMALL-EFFICIENT-RNA-TARGETING-C, JPN6024011777, ISSN: 0005494733 *

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US20240344060A1 (en) 2024-10-17
EP3801641A1 (en) 2021-04-14
SG11202012015YA (en) 2021-01-28
WO2019236998A1 (en) 2019-12-12
CN113286619A (zh) 2021-08-20
US20190382759A1 (en) 2019-12-19
CA3102783A1 (en) 2019-12-12
KR20210060429A (ko) 2021-05-26
EP3801641A4 (en) 2022-09-28
AU2019281006A1 (en) 2021-01-28

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