JP2019536462A5 - - Google Patents
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- Publication number
- JP2019536462A5 JP2019536462A5 JP2019530105A JP2019530105A JP2019536462A5 JP 2019536462 A5 JP2019536462 A5 JP 2019536462A5 JP 2019530105 A JP2019530105 A JP 2019530105A JP 2019530105 A JP2019530105 A JP 2019530105A JP 2019536462 A5 JP2019536462 A5 JP 2019536462A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid molecule
- genome editing
- cas9 protein
- sequence
- 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.)
- Granted
Links
- 150000007523 nucleic acids Chemical class 0.000 claims 90
- 108020004707 nucleic acids Proteins 0.000 claims 65
- 102000039446 nucleic acids Human genes 0.000 claims 65
- 108091033409 CRISPR Proteins 0.000 claims 58
- 238000010362 genome editing Methods 0.000 claims 40
- 150000001413 amino acids Chemical class 0.000 claims 29
- 108090000623 proteins and genes Proteins 0.000 claims 29
- 108020005004 Guide RNA Proteins 0.000 claims 27
- 235000018102 proteins Nutrition 0.000 claims 25
- 102000004169 proteins and genes Human genes 0.000 claims 25
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 24
- 235000001014 amino acid Nutrition 0.000 claims 24
- 238000003780 insertion Methods 0.000 claims 22
- 230000037431 insertion Effects 0.000 claims 22
- 239000002773 nucleotide Substances 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 12
- 230000001052 transient effect Effects 0.000 claims 8
- 108091026890 Coding region Proteins 0.000 claims 6
- 239000013598 vector Substances 0.000 claims 6
- 101710163270 Nuclease Proteins 0.000 claims 5
- 230000000694 effects Effects 0.000 claims 5
- 230000008685 targeting Effects 0.000 claims 5
- 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 4
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 4
- 230000000295 complement effect Effects 0.000 claims 4
- 239000013603 viral vector Substances 0.000 claims 4
- 230000014509 gene expression Effects 0.000 claims 3
- 229920001184 polypeptide Polymers 0.000 claims 3
- 102000004196 processed proteins & peptides Human genes 0.000 claims 3
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 238000006467 substitution reaction Methods 0.000 claims 3
- 108020004705 Codon Proteins 0.000 claims 2
- 239000004471 Glycine Substances 0.000 claims 2
- 230000003834 intracellular effect Effects 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 125000006850 spacer group Chemical group 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 claims 1
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 claims 1
- 101000897880 Spiroplasma virus SpV1-R8A2 B Putative capsid protein ORF9 Proteins 0.000 claims 1
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 235000004279 alanine Nutrition 0.000 claims 1
- 230000001413 cellular effect Effects 0.000 claims 1
- 238000010363 gene targeting Methods 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 229960000856 protein c Drugs 0.000 claims 1
- 230000010474 transient expression Effects 0.000 claims 1
- 239000004474 valine Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022208855A JP2023052089A (ja) | 2016-12-05 | 2022-12-26 | 内因性および起源DNAのワンショットガイドRNA(ogRNA)標的化のためのシステムおよび方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662430154P | 2016-12-05 | 2016-12-05 | |
| US62/430,154 | 2016-12-05 | ||
| US201762503640P | 2017-05-09 | 2017-05-09 | |
| US62/503,640 | 2017-05-09 | ||
| PCT/US2017/064720 WO2018106693A1 (en) | 2016-12-05 | 2017-12-05 | SYSTEMS AND METHODS FOR ONE-SHOT GUIDE RNA (ogRNA) TARGETING OF ENDOGENOUS AND SOURCE DNA |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022208855A Division JP2023052089A (ja) | 2016-12-05 | 2022-12-26 | 内因性および起源DNAのワンショットガイドRNA(ogRNA)標的化のためのシステムおよび方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019536462A JP2019536462A (ja) | 2019-12-19 |
| JP2019536462A5 true JP2019536462A5 (https=) | 2021-01-21 |
| JP7272952B2 JP7272952B2 (ja) | 2023-05-12 |
Family
ID=60972337
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019530105A Active JP7272952B2 (ja) | 2016-12-05 | 2017-12-05 | 内因性および起源DNAのワンショットガイドRNA(ogRNA)標的化のためのシステムおよび方法 |
| JP2022208855A Withdrawn JP2023052089A (ja) | 2016-12-05 | 2022-12-26 | 内因性および起源DNAのワンショットガイドRNA(ogRNA)標的化のためのシステムおよび方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022208855A Withdrawn JP2023052089A (ja) | 2016-12-05 | 2022-12-26 | 内因性および起源DNAのワンショットガイドRNA(ogRNA)標的化のためのシステムおよび方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (6) | US9963719B1 (https=) |
| EP (1) | EP3548614A1 (https=) |
| JP (2) | JP7272952B2 (https=) |
| KR (1) | KR102604903B1 (https=) |
| CN (1) | CN110168084A (https=) |
| AU (1) | AU2017373797B2 (https=) |
| CA (1) | CA3046076A1 (https=) |
| MX (2) | MX2019006475A (https=) |
| WO (1) | WO2018106693A1 (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014346559B2 (en) | 2013-11-07 | 2020-07-09 | Editas Medicine,Inc. | CRISPR-related methods and compositions with governing gRNAs |
| WO2017075475A1 (en) * | 2015-10-30 | 2017-05-04 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| MA50877A (fr) | 2017-11-21 | 2020-09-30 | Bayer Healthcare Llc | Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante |
| EP3723813A4 (en) * | 2017-12-15 | 2021-10-06 | Regents of the University of Minnesota | GENOMIC CRISPR MEDIATION EDITING WITH VECTORS |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| WO2020055748A1 (en) * | 2018-09-10 | 2020-03-19 | The Regents Of The University Of California | Modulators of cas9 polypeptides and methods of use thereof |
| WO2020176552A1 (en) | 2019-02-25 | 2020-09-03 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
| WO2020186059A2 (en) * | 2019-03-12 | 2020-09-17 | Crispr Therapeutics Ag | Novel high fidelity rna-programmable endonuclease systems and uses thereof |
| EP3990636A1 (en) | 2019-06-28 | 2022-05-04 | CRISPR Therapeutics AG | Materials and methods for controlling gene editing |
| WO2021053582A1 (en) * | 2019-09-18 | 2021-03-25 | Crispr Therapeutics Ag | All-in-one self inactivating crispr vectors |
| WO2021062201A1 (en) * | 2019-09-25 | 2021-04-01 | Spotlight Therapeutics | Compositions and methods for nucleoprotein targeting and expression |
| US20230203220A1 (en) * | 2020-05-29 | 2023-06-29 | Regents Of The University Of Minnesota | Copolymers for intracellular therapeutic nucleic acid payload delivery |
| CA3200815A1 (en) * | 2020-12-03 | 2022-06-09 | Benjamin OAKES | Compositions and methods for the targeting of bcl11a |
| EP4323522A1 (en) | 2021-04-16 | 2024-02-21 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
| WO2025179057A1 (en) * | 2024-02-21 | 2025-08-28 | The Regents Of The University Of California | In vivo modification of cell genomes |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014346559B2 (en) | 2013-11-07 | 2020-07-09 | Editas Medicine,Inc. | CRISPR-related methods and compositions with governing gRNAs |
| MX2016007325A (es) | 2013-12-12 | 2017-07-19 | Broad Inst Inc | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
| WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| CA2963820A1 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
| EP3265559B1 (en) * | 2015-03-03 | 2021-01-06 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| US20190002920A1 (en) | 2015-04-30 | 2019-01-03 | The Brigham And Women's Hospital, Inc. | Methods and kits for cloning-free genome editing |
| WO2016183438A1 (en) | 2015-05-14 | 2016-11-17 | Massachusetts Institute Of Technology | Self-targeting genome editing system |
| WO2016186772A2 (en) | 2015-05-16 | 2016-11-24 | Genzyme Corporation | Gene editing of deep intronic mutations |
| US11530421B2 (en) * | 2016-02-01 | 2022-12-20 | The Regents Of The University Of California | Self-inactivating endonuclease-encoding nucleic acids and methods of using the same |
| WO2017173004A1 (en) * | 2016-03-30 | 2017-10-05 | Mikuni Takayasu | A method for in vivo precise genome editing |
-
2017
- 2017-12-05 WO PCT/US2017/064720 patent/WO2018106693A1/en not_active Ceased
- 2017-12-05 EP EP17829373.4A patent/EP3548614A1/en active Pending
- 2017-12-05 CN CN201780082711.XA patent/CN110168084A/zh active Pending
- 2017-12-05 CA CA3046076A patent/CA3046076A1/en active Pending
- 2017-12-05 JP JP2019530105A patent/JP7272952B2/ja active Active
- 2017-12-05 MX MX2019006475A patent/MX2019006475A/es unknown
- 2017-12-05 US US15/832,567 patent/US9963719B1/en active Active
- 2017-12-05 KR KR1020197019386A patent/KR102604903B1/ko active Active
- 2017-12-05 AU AU2017373797A patent/AU2017373797B2/en active Active
-
2018
- 2018-03-23 US US15/934,750 patent/US10006054B1/en active Active
- 2018-05-15 US US15/980,450 patent/US10494649B2/en active Active
-
2019
- 2019-06-03 MX MX2024002327A patent/MX2024002327A/es unknown
- 2019-10-29 US US16/666,982 patent/US11028411B2/en active Active
-
2021
- 2021-05-27 US US17/332,764 patent/US11692205B2/en active Active
-
2022
- 2022-12-26 JP JP2022208855A patent/JP2023052089A/ja not_active Withdrawn
-
2023
- 2023-05-12 US US18/316,880 patent/US12264331B2/en active Active
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