JP2018535689A5 - - Google Patents
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- JP2018535689A5 JP2018535689A5 JP2018529124A JP2018529124A JP2018535689A5 JP 2018535689 A5 JP2018535689 A5 JP 2018535689A5 JP 2018529124 A JP2018529124 A JP 2018529124A JP 2018529124 A JP2018529124 A JP 2018529124A JP 2018535689 A5 JP2018535689 A5 JP 2018535689A5
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- 108020004414 DNA Proteins 0.000 claims description 161
- 238000000034 method Methods 0.000 claims description 119
- 102000004169 proteins and genes Human genes 0.000 claims description 91
- 108090000623 proteins and genes Proteins 0.000 claims description 91
- 101710163270 Nuclease Proteins 0.000 claims description 81
- 102000039446 nucleic acids Human genes 0.000 claims description 62
- 108020004707 nucleic acids Proteins 0.000 claims description 62
- 150000007523 nucleic acids Chemical class 0.000 claims description 62
- 102000053602 DNA Human genes 0.000 claims description 60
- 230000008685 targeting Effects 0.000 claims description 50
- 239000012634 fragment Substances 0.000 claims description 48
- 108091033409 CRISPR Proteins 0.000 claims description 46
- 102000004190 Enzymes Human genes 0.000 claims description 42
- 108090000790 Enzymes Proteins 0.000 claims description 42
- 102100035102 E3 ubiquitin-protein ligase MYCBP2 Human genes 0.000 claims description 32
- 238000010354 CRISPR gene editing Methods 0.000 claims description 28
- 230000001105 regulatory effect Effects 0.000 claims description 22
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 20
- 230000000295 complement effect Effects 0.000 claims description 15
- 101150069031 CSN2 gene Proteins 0.000 claims description 11
- 101150055601 cops2 gene Proteins 0.000 claims description 11
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 10
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 10
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 10
- 108091008146 restriction endonucleases Proteins 0.000 claims description 9
- 238000004519 manufacturing process Methods 0.000 claims description 8
- 239000002773 nucleotide Substances 0.000 claims description 7
- 125000003729 nucleotide group Chemical group 0.000 claims description 7
- 102000004533 Endonucleases Human genes 0.000 claims description 5
- 108010042407 Endonucleases Proteins 0.000 claims description 5
- 238000011144 upstream manufacturing Methods 0.000 claims description 5
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 claims description 4
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 claims description 4
- 238000006243 chemical reaction Methods 0.000 claims description 4
- 238000003776 cleavage reaction Methods 0.000 claims description 4
- 230000007017 scission Effects 0.000 claims description 4
- FGUUSXIOTUKUDN-IBGZPJMESA-N C1(=CC=CC=C1)N1C2=C(NC([C@H](C1)NC=1OC(=NN=1)C1=CC=CC=C1)=O)C=CC=C2 Chemical compound C1(=CC=CC=C1)N1C2=C(NC([C@H](C1)NC=1OC(=NN=1)C1=CC=CC=C1)=O)C=CC=C2 FGUUSXIOTUKUDN-IBGZPJMESA-N 0.000 claims description 3
- 238000012408 PCR amplification Methods 0.000 claims description 3
- 230000000694 effects Effects 0.000 claims description 2
- 230000006862 enzymatic digestion Effects 0.000 claims description 2
- 238000006911 enzymatic reaction Methods 0.000 claims description 2
- 108010068698 spleen exonuclease Proteins 0.000 claims description 2
- 108020005004 Guide RNA Proteins 0.000 description 41
- 241000894007 species Species 0.000 description 20
- 210000004027 cell Anatomy 0.000 description 12
- 230000001580 bacterial effect Effects 0.000 description 7
- 108020004682 Single-Stranded DNA Proteins 0.000 description 6
- 108020005196 Mitochondrial DNA Proteins 0.000 description 5
- 108020001027 Ribosomal DNA Proteins 0.000 description 5
- 108091028113 Trans-activating crRNA Proteins 0.000 description 5
- 101150038500 cas9 gene Proteins 0.000 description 5
- 210000002230 centromere Anatomy 0.000 description 5
- 230000003612 virological effect Effects 0.000 description 5
- 241000894006 Bacteria Species 0.000 description 4
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 4
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 description 4
- 230000003252 repetitive effect Effects 0.000 description 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 4
- 241000196324 Embryophyta Species 0.000 description 3
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 3
- 108091023040 Transcription factor Proteins 0.000 description 3
- 102000040945 Transcription factor Human genes 0.000 description 3
- 241000700605 Viruses Species 0.000 description 3
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 description 2
- 229930024421 Adenine Natural products 0.000 description 2
- 241000206602 Eukaryota Species 0.000 description 2
- 241000124008 Mammalia Species 0.000 description 2
- 229960000643 adenine Drugs 0.000 description 2
- 239000003153 chemical reaction reagent Substances 0.000 description 2
- 229940104302 cytosine Drugs 0.000 description 2
- 230000037452 priming Effects 0.000 description 2
- 229940113082 thymine Drugs 0.000 description 2
- 229940035893 uracil Drugs 0.000 description 2
- 241000251468 Actinopterygii Species 0.000 description 1
- 101150050733 Gnas gene Proteins 0.000 description 1
- 241000238631 Hexapoda Species 0.000 description 1
- 241001465754 Metazoa Species 0.000 description 1
- 108091081062 Repeated sequence (DNA) Proteins 0.000 description 1
- 210000004102 animal cell Anatomy 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 238000000638 solvent extraction Methods 0.000 description 1
- 239000000758 substrate Substances 0.000 description 1
- 230000002194 synthesizing effect Effects 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021103316A JP2021141904A (ja) | 2015-12-07 | 2021-06-22 | ガイド核酸を作製および使用するための方法および組成物 |
| JP2024030061A JP2024051149A (ja) | 2015-12-07 | 2024-02-29 | ガイド核酸を作製および使用するための方法および組成物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562264262P | 2015-12-07 | 2015-12-07 | |
| US62/264,262 | 2015-12-07 | ||
| US201662298963P | 2016-02-23 | 2016-02-23 | |
| US62/298,963 | 2016-02-23 | ||
| PCT/US2016/065420 WO2017100343A1 (en) | 2015-12-07 | 2016-12-07 | Methods and compositions for the making and using of guide nucleic acids |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021103316A Division JP2021141904A (ja) | 2015-12-07 | 2021-06-22 | ガイド核酸を作製および使用するための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018535689A JP2018535689A (ja) | 2018-12-06 |
| JP2018535689A5 true JP2018535689A5 (enExample) | 2020-01-16 |
| JP6995751B2 JP6995751B2 (ja) | 2022-02-04 |
Family
ID=59013518
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529124A Active JP6995751B2 (ja) | 2015-12-07 | 2016-12-07 | ガイド核酸を作製および使用するための方法および組成物 |
| JP2021103316A Withdrawn JP2021141904A (ja) | 2015-12-07 | 2021-06-22 | ガイド核酸を作製および使用するための方法および組成物 |
| JP2024030061A Pending JP2024051149A (ja) | 2015-12-07 | 2024-02-29 | ガイド核酸を作製および使用するための方法および組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021103316A Withdrawn JP2021141904A (ja) | 2015-12-07 | 2021-06-22 | ガイド核酸を作製および使用するための方法および組成物 |
| JP2024030061A Pending JP2024051149A (ja) | 2015-12-07 | 2024-02-29 | ガイド核酸を作製および使用するための方法および組成物 |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US10787662B2 (enExample) |
| EP (2) | EP3871695A1 (enExample) |
| JP (3) | JP6995751B2 (enExample) |
| CN (1) | CN109310784B (enExample) |
| AU (1) | AU2016365720B2 (enExample) |
| CA (1) | CA3006781A1 (enExample) |
| DK (1) | DK3386550T3 (enExample) |
| WO (1) | WO2017100343A1 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT3105328T (lt) | 2014-02-11 | 2020-07-10 | The Regents Of The University Of Colorado, A Body Corporate | Daugiakomponentis genomo konstravimas, sudarantis sąlygas crispr |
| AU2016365720B2 (en) | 2015-12-07 | 2020-11-26 | Arc Bio, Llc | Methods and compositions for the making and using of guide nucleic acids |
| CN109688820B (zh) | 2016-06-24 | 2023-01-10 | 科罗拉多州立大学董事会（法人团体） | 用于生成条形码化组合文库的方法 |
| JP7383478B2 (ja) * | 2016-12-22 | 2023-11-20 | インテリア セラピューティクス，インコーポレイテッド | アルファ１アンチトリプシン欠乏症を治療するための組成物および方法 |
| EP3635114B1 (en) * | 2017-06-07 | 2024-11-06 | Arc Bio, LLC | Creation and use of guide nucleic acids |
| IT201700067084A1 (it) * | 2017-06-16 | 2018-12-16 | Univ Degli Studi Di Palermo | Metodo in vitro per l’arricchimento in sequenze genomiche bersaglio mediante sistema CRISPR-Cas |
| US10011849B1 (en) | 2017-06-23 | 2018-07-03 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| US9982279B1 (en) | 2017-06-23 | 2018-05-29 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| EP3640333A4 (en) * | 2017-07-14 | 2020-12-30 | Cure Genetics Co., Ltd | GENE EDITING SYSTEM AND GENE EDITING METHOD |
| AU2018320864B2 (en) | 2017-08-22 | 2024-02-22 | Napigen, Inc. | Organelle genome modification using polynucleotide guided endonuclease |
| CN112654710A (zh) | 2018-05-16 | 2021-04-13 | 辛瑟高公司 | 用于指导rna设计和使用的方法和系统 |
| AU2019282812B2 (en) | 2018-06-07 | 2025-08-14 | Arc Bio, Llc | Compositions and methods for making guide nucleic acids |
| MX2021001070A (es) * | 2018-07-31 | 2021-05-27 | Intellia Therapeutics Inc | Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1). |
| KR102200109B1 (ko) * | 2018-09-12 | 2021-01-08 | 한국과학기술정보연구원 | 표적 서열 증폭용 올리고 뉴클레오타이드 및 이를 이용한 표적 서열의 증폭 방법 |
| EP3861135B1 (en) | 2018-10-04 | 2023-08-02 | Arc Bio, LLC | Normalization controls for managing low sample inputs in next generation sequencing |
| ES2927412T3 (es) | 2018-11-08 | 2022-11-04 | Siemens Healthcare Gmbh | Secuenciación directa de nanoporos de ARN con la ayuda de un polinucleótido de horquilla |
| EP4317950B1 (en) | 2019-03-18 | 2026-01-28 | Regeneron Pharmaceuticals, Inc. | Crispr/cas screening platform to identify genetic modifiers of tau seeding or aggregation |
| JP7389135B2 (ja) | 2019-03-18 | 2023-11-29 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | タウ凝集に関連する遺伝的脆弱性を明らかにするためのｃｒｉｓｐｒ／ｃａｓドロップアウトスクリーニングプラットフォーム |
| AU2020272770B2 (en) | 2019-04-09 | 2026-01-22 | Arc Bio, Llc | Compositions and methods for nucleotide modification-based depletion |
| KR20220024053A (ko) | 2019-06-14 | 2022-03-03 | 리제너론 파마슈티칼스 인코포레이티드 | 타우병증의 모델 |
| AU2022272723A1 (en) * | 2021-05-14 | 2023-11-30 | Becton, Dickinson And Company | Methods for making libraries for nucleic acid sequencing |
| CN114293264B (zh) * | 2021-12-21 | 2024-12-27 | 翌圣生物科技(上海)股份有限公司 | 酶法靶序列随机sgRNA文库的制备方法 |
| CN114277447A (zh) * | 2021-12-21 | 2022-04-05 | 翌圣生物科技(上海)股份有限公司 | 靶序列随机sgRNA全覆盖组的制备方法 |
| WO2023122762A1 (en) | 2021-12-22 | 2023-06-29 | Camp4 Therapeutics Corporation | Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas |
| WO2024187091A1 (en) * | 2023-03-08 | 2024-09-12 | Seek Labs, Inc. | Compositions and methods of isothermal nucleic acid amplification |
| CN116617412A (zh) * | 2023-04-20 | 2023-08-22 | 浙江工业大学 | 一种靶向编辑mtDNA的CRISPR-Cas9递送载体的制备方法 |
| EP4771158A1 (en) * | 2023-09-01 | 2026-07-08 | Script Biosciences, Inc. | Compositions and methods for targeted modification of msh3 |
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| US5948902A (en) | 1997-11-20 | 1999-09-07 | South Alabama Medical Science Foundation | Antisense oligonucleotides to human serine/threonine protein phosphatase genes |
| US20050233340A1 (en) | 2004-04-20 | 2005-10-20 | Barrett Michael T | Methods and compositions for assessing CpG methylation |
| US8323930B2 (en) | 2007-07-28 | 2012-12-04 | Dna Twopointo, Inc. | Methods, compositions and kits for one-step DNA cloning using DNA topoisomerase |
| WO2009133466A2 (en) | 2008-04-30 | 2009-11-05 | Population Genetics Technologies Ltd. | Asymmetric adapter library construction |
| EP2393940B1 (en) | 2009-02-03 | 2014-12-17 | New England Biolabs, Inc. | Generation of random double-strand breaks in dna using enzymes |
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| AU2016365720B2 (en) | 2015-12-07 | 2020-11-26 | Arc Bio, Llc | Methods and compositions for the making and using of guide nucleic acids |
| CA3015360A1 (en) | 2016-02-23 | 2017-08-31 | Arc Bio, Llc | Methods and compositions for target detection |
| EP3635114B1 (en) | 2017-06-07 | 2024-11-06 | Arc Bio, LLC | Creation and use of guide nucleic acids |
-
2016
- 2016-12-07 AU AU2016365720A patent/AU2016365720B2/en active Active
- 2016-12-07 EP EP21152335.2A patent/EP3871695A1/en active Pending
- 2016-12-07 EP EP16873782.3A patent/EP3386550B1/en active Active
- 2016-12-07 JP JP2018529124A patent/JP6995751B2/ja active Active
- 2016-12-07 DK DK16873782.3T patent/DK3386550T3/da active
- 2016-12-07 WO PCT/US2016/065420 patent/WO2017100343A1/en not_active Ceased
- 2016-12-07 US US15/742,862 patent/US10787662B2/en active Active
- 2016-12-07 CN CN201680081179.5A patent/CN109310784B/zh active Active
- 2016-12-07 CA CA3006781A patent/CA3006781A1/en active Pending
-
2020
- 2020-08-17 US US16/995,761 patent/US20210207130A1/en not_active Abandoned
-
2021
- 2021-06-22 JP JP2021103316A patent/JP2021141904A/ja not_active Withdrawn
-
2024
- 2024-02-29 JP JP2024030061A patent/JP2024051149A/ja active Pending
- 2024-10-22 US US18/923,525 patent/US20250230432A1/en active Pending
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