JPWO2023275601A5 - - Google Patents
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- JPWO2023275601A5 JPWO2023275601A5 JP2024500032A JP2024500032A JPWO2023275601A5 JP WO2023275601 A5 JPWO2023275601 A5 JP WO2023275601A5 JP 2024500032 A JP2024500032 A JP 2024500032A JP 2024500032 A JP2024500032 A JP 2024500032A JP WO2023275601 A5 JPWO2023275601 A5 JP WO2023275601A5
- Authority
- JP
- Japan
- Prior art keywords
- protein
- tnpb
- item
- sequence
- rna
- 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.)
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- 108090000623 proteins and genes Proteins 0.000 claims description 167
- 102000004169 proteins and genes Human genes 0.000 claims description 165
- 238000000034 method Methods 0.000 claims description 142
- 239000012636 effector Substances 0.000 claims description 104
- 102000040430 polynucleotide Human genes 0.000 claims description 67
- 108091033319 polynucleotide Proteins 0.000 claims description 67
- 239000002157 polynucleotide Substances 0.000 claims description 67
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 40
- 108020001507 fusion proteins Proteins 0.000 claims description 30
- 102000037865 fusion proteins Human genes 0.000 claims description 30
- 230000002068 genetic effect Effects 0.000 claims description 22
- 101150044011 tnpB gene Proteins 0.000 claims description 18
- 239000002773 nucleotide Substances 0.000 claims description 14
- 125000003729 nucleotide group Chemical group 0.000 claims description 14
- 101710163270 Nuclease Proteins 0.000 claims description 9
- 230000008685 targeting Effects 0.000 claims description 8
- 241000192091 Deinococcus radiodurans Species 0.000 claims description 5
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 4
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 4
- 108010042407 Endonucleases Proteins 0.000 claims description 4
- 108010092799 RNA-directed DNA polymerase Proteins 0.000 claims description 4
- 102000018120 Recombinases Human genes 0.000 claims description 4
- 108010091086 Recombinases Proteins 0.000 claims description 4
- 102000006382 Ribonucleases Human genes 0.000 claims description 4
- 108010083644 Ribonucleases Proteins 0.000 claims description 4
- 102000008579 Transposases Human genes 0.000 claims description 4
- 108010020764 Transposases Proteins 0.000 claims description 4
- 230000001973 epigenetic effect Effects 0.000 claims description 4
- 238000002372 labelling Methods 0.000 claims description 4
- 239000003607 modifier Substances 0.000 claims description 4
- 102000004533 Endonucleases Human genes 0.000 claims description 3
- 230000035772 mutation Effects 0.000 claims description 2
- 102100034343 Integrase Human genes 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 89
- 108020004414 DNA Proteins 0.000 description 49
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- 239000013598 vector Substances 0.000 description 19
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- 238000003745 diagnosis Methods 0.000 description 7
- 210000000130 stem cell Anatomy 0.000 description 7
- 238000001890 transfection Methods 0.000 description 6
- 239000013607 AAV vector Substances 0.000 description 4
- 102100026846 Cytidine deaminase Human genes 0.000 description 4
- 108010031325 Cytidine deaminase Proteins 0.000 description 4
- 102100031780 Endonuclease Human genes 0.000 description 4
- 108091081548 Palindromic sequence Proteins 0.000 description 4
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 4
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 description 4
- 239000002502 liposome Substances 0.000 description 4
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 4
- 230000003612 virological effect Effects 0.000 description 4
- 208000009869 Neu-Laxova syndrome Diseases 0.000 description 3
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 3
- 108020004682 Single-Stranded DNA Proteins 0.000 description 3
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- 230000001177 retroviral effect Effects 0.000 description 3
- 108091006106 transcriptional activators Proteins 0.000 description 3
- 108091006107 transcriptional repressors Proteins 0.000 description 3
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 description 2
- 102000055025 Adenosine deaminases Human genes 0.000 description 2
- 229940113491 Glycosylase inhibitor Drugs 0.000 description 2
- 208000009889 Herpes Simplex Diseases 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 2
- 210000004102 animal cell Anatomy 0.000 description 2
- 239000000872 buffer Substances 0.000 description 2
- 229910000389 calcium phosphate Inorganic materials 0.000 description 2
- 239000001506 calcium phosphate Substances 0.000 description 2
- 235000011010 calcium phosphates Nutrition 0.000 description 2
- 229920006317 cationic polymer Polymers 0.000 description 2
- 210000000170 cell membrane Anatomy 0.000 description 2
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- 201000010099 disease Diseases 0.000 description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 2
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 description 2
- 238000004520 electroporation Methods 0.000 description 2
- 210000003527 eukaryotic cell Anatomy 0.000 description 2
- 210000001808 exosome Anatomy 0.000 description 2
- 210000003754 fetus Anatomy 0.000 description 2
- 102000034287 fluorescent proteins Human genes 0.000 description 2
- 108091006047 fluorescent proteins Proteins 0.000 description 2
- 238000010362 genome editing Methods 0.000 description 2
- 210000004602 germ cell Anatomy 0.000 description 2
- 210000005260 human cell Anatomy 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 210000004263 induced pluripotent stem cell Anatomy 0.000 description 2
- 238000001638 lipofection Methods 0.000 description 2
- 238000000520 microinjection Methods 0.000 description 2
- 238000012986 modification Methods 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 239000002245 particle Substances 0.000 description 2
- 230000000149 penetrating effect Effects 0.000 description 2
- 108090000765 processed proteins & peptides Proteins 0.000 description 2
- 102000004196 processed proteins & peptides Human genes 0.000 description 2
- 210000001236 prokaryotic cell Anatomy 0.000 description 2
- 230000001105 regulatory effect Effects 0.000 description 2
- 239000000126 substance Substances 0.000 description 2
- 210000003014 totipotent stem cell Anatomy 0.000 description 2
- 238000013518 transcription Methods 0.000 description 2
- 230000035897 transcription Effects 0.000 description 2
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 2
- 229940035893 uracil Drugs 0.000 description 2
- 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 description 1
- 108091033409 CRISPR Proteins 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 108091092566 Extrachromosomal DNA Proteins 0.000 description 1
- 108091092584 GDNA Proteins 0.000 description 1
- 102100025169 Max-binding protein MNT Human genes 0.000 description 1
- 210000001744 T-lymphocyte Anatomy 0.000 description 1
- 239000007853 buffer solution Substances 0.000 description 1
- 210000004671 cell-free system Anatomy 0.000 description 1
- 239000013611 chromosomal DNA Substances 0.000 description 1
- 238000003776 cleavage reaction Methods 0.000 description 1
- 230000000295 complement effect Effects 0.000 description 1
- 230000001276 controlling effect Effects 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 1
- 230000000415 inactivating effect Effects 0.000 description 1
- 230000003834 intracellular effect Effects 0.000 description 1
- 230000006780 non-homologous end joining Effects 0.000 description 1
- 230000007017 scission Effects 0.000 description 1
- 239000007787 solid Substances 0.000 description 1
- 230000002194 synthesizing effect Effects 0.000 description 1
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/IB2021/055958 WO2023275601A1 (en) | 2021-07-02 | 2021-07-02 | A novel rna-programmable system for targeting polynucleotides |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024524514A JP2024524514A (ja) | 2024-07-05 |
| JPWO2023275601A5 true JPWO2023275601A5 (https=) | 2024-07-12 |
| JP2024524514A5 JP2024524514A5 (https=) | 2024-07-12 |
Family
ID=76859656
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024500032A Pending JP2024524514A (ja) | 2021-07-02 | 2021-07-02 | ポリヌクレオチドを標的とする新規ｒｎａプログラム可能システム |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20240368630A1 (https=) |
| EP (1) | EP4363566A1 (https=) |
| JP (1) | JP2024524514A (https=) |
| KR (1) | KR20240027724A (https=) |
| WO (1) | WO2023275601A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116355878B (zh) * | 2023-02-28 | 2024-04-26 | 华中农业大学 | 新型TnpB编程性核酸酶及其应用 |
| CN116355938A (zh) * | 2023-03-01 | 2023-06-30 | 山东大学 | 基于微型RNA引导核酸酶TnpB的基因编辑载体系统及其构建方法和应用 |
| WO2024192291A1 (en) | 2023-03-15 | 2024-09-19 | Renagade Therapeutics Management Inc. | Delivery of gene editing systems and methods of use thereof |
| WO2024199134A1 (en) * | 2023-03-27 | 2024-10-03 | Beijing Astragenomics Technology Co., Ltd. | Isolated nuclease and use thereof |
| AU2024335327A1 (en) | 2023-09-01 | 2026-03-26 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| CN116970590B (zh) * | 2023-09-22 | 2024-01-30 | 北京科芙兰德生物科学有限责任公司 | 小于380个氨基酸的超级迷你型基因编辑器及其应用 |
| WO2025117544A1 (en) * | 2023-11-29 | 2025-06-05 | The Broad Institute, Inc. | Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3012607A1 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzymes and systems |
| EP4159856A1 (en) * | 2015-06-18 | 2023-04-05 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| US12431216B2 (en) * | 2016-08-17 | 2025-09-30 | Broad Institute, Inc. | Methods for identifying class 2 crispr-cas systems |
| US20230040216A1 (en) * | 2019-11-19 | 2023-02-09 | The Broad Institute, Inc. | Retrotransposons and use thereof |
| EP4281567A4 (en) * | 2021-01-25 | 2025-03-05 | The Broad Institute Inc. | REPROGRAMMABLE TNPB POLYPEPTIDES AND THEIR USE |
-
2021
- 2021-07-02 WO PCT/IB2021/055958 patent/WO2023275601A1/en not_active Ceased
- 2021-07-02 US US18/575,173 patent/US20240368630A1/en active Pending
- 2021-07-02 JP JP2024500032A patent/JP2024524514A/ja active Pending
- 2021-07-02 EP EP21740202.3A patent/EP4363566A1/en active Pending
- 2021-07-02 KR KR1020247002706A patent/KR20240027724A/ko active Pending
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