IL302101A - Reprogrammable iscb nucleases and uses thereof - Google Patents
Reprogrammable iscb nucleases and uses thereofInfo
- Publication number
- IL302101A IL302101A IL302101A IL30210123A IL302101A IL 302101 A IL302101 A IL 302101A IL 302101 A IL302101 A IL 302101A IL 30210123 A IL30210123 A IL 30210123A IL 302101 A IL302101 A IL 302101A
- Authority
- IL
- Israel
- Prior art keywords
- composition
- iscb
- protein
- activity
- target
- Prior art date
Links
- 101710163270 Nuclease Proteins 0.000 title claims 6
- 239000000203 mixture Substances 0.000 claims 55
- 108091033319 polynucleotide Proteins 0.000 claims 46
- 102000040430 polynucleotide Human genes 0.000 claims 46
- 239000002157 polynucleotide Substances 0.000 claims 46
- 108090000623 proteins and genes Proteins 0.000 claims 37
- 230000000694 effects Effects 0.000 claims 35
- 102000004169 proteins and genes Human genes 0.000 claims 31
- 238000000034 method Methods 0.000 claims 17
- 210000004027 cell Anatomy 0.000 claims 14
- 238000003780 insertion Methods 0.000 claims 12
- 230000037431 insertion Effects 0.000 claims 12
- 229920001184 polypeptide Polymers 0.000 claims 11
- 102000004196 processed proteins & peptides Human genes 0.000 claims 11
- 108090000765 processed proteins & peptides Proteins 0.000 claims 11
- 239000013598 vector Substances 0.000 claims 10
- 108091033409 CRISPR Proteins 0.000 claims 8
- 238000010354 CRISPR gene editing Methods 0.000 claims 8
- 102000039446 nucleic acids Human genes 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 8
- 150000007523 nucleic acids Chemical class 0.000 claims 8
- 238000012986 modification Methods 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 6
- 230000035897 transcription Effects 0.000 claims 6
- 238000013518 transcription Methods 0.000 claims 6
- 241000842783 Orna Species 0.000 claims 5
- 150000001413 amino acids Chemical class 0.000 claims 5
- 230000009870 specific binding Effects 0.000 claims 5
- 108020004414 DNA Proteins 0.000 claims 4
- 230000007018 DNA scission Effects 0.000 claims 4
- 230000007022 RNA scission Effects 0.000 claims 4
- 230000004913 activation Effects 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 238000013519 translation Methods 0.000 claims 4
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims 3
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims 3
- 102100034343 Integrase Human genes 0.000 claims 3
- 108010092799 RNA-directed DNA polymerase Proteins 0.000 claims 3
- 230000027455 binding Effects 0.000 claims 3
- 125000006850 spacer group Chemical group 0.000 claims 3
- 108700028369 Alleles Proteins 0.000 claims 2
- 108010077544 Chromatin Proteins 0.000 claims 2
- 108010033040 Histones Proteins 0.000 claims 2
- 101000615488 Homo sapiens Methyl-CpG-binding domain protein 2 Proteins 0.000 claims 2
- 102100021299 Methyl-CpG-binding domain protein 2 Human genes 0.000 claims 2
- 108020004485 Nonsense Codon Proteins 0.000 claims 2
- 102000008579 Transposases Human genes 0.000 claims 2
- 108010020764 Transposases Proteins 0.000 claims 2
- 210000004899 c-terminal region Anatomy 0.000 claims 2
- 210000003483 chromatin Anatomy 0.000 claims 2
- 230000005782 double-strand break Effects 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 108020001507 fusion proteins Proteins 0.000 claims 2
- 102000037865 fusion proteins Human genes 0.000 claims 2
- 230000006801 homologous recombination Effects 0.000 claims 2
- 238000002744 homologous recombination Methods 0.000 claims 2
- 238000012545 processing Methods 0.000 claims 2
- 238000007634 remodeling Methods 0.000 claims 2
- 108091069025 single-strand RNA Proteins 0.000 claims 2
- MTCFGRXMJLQNBG-REOHCLBHSA-N (2S)-2-Amino-3-hydroxypropansäure Chemical compound OC[C@H](N)C(O)=O MTCFGRXMJLQNBG-REOHCLBHSA-N 0.000 claims 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims 1
- 102000055025 Adenosine deaminases Human genes 0.000 claims 1
- 108091023037 Aptamer Proteins 0.000 claims 1
- 102100026846 Cytidine deaminase Human genes 0.000 claims 1
- 108010031325 Cytidine deaminase Proteins 0.000 claims 1
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 claims 1
- 102000018120 Recombinases Human genes 0.000 claims 1
- 108010091086 Recombinases Proteins 0.000 claims 1
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Chemical Or Physical Treatment Of Fibers (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Detergent Compositions (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063105191P | 2020-10-23 | 2020-10-23 | |
| US202063105177P | 2020-10-23 | 2020-10-23 | |
| US202163156857P | 2021-03-04 | 2021-03-04 | |
| US202163195659P | 2021-06-01 | 2021-06-01 | |
| US202163235583P | 2021-08-20 | 2021-08-20 | |
| PCT/US2021/056361 WO2022087494A1 (en) | 2020-10-23 | 2021-10-22 | Reprogrammable iscb nucleases and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL302101A true IL302101A (en) | 2023-06-01 |
Family
ID=81289497
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL302101A IL302101A (en) | 2020-10-23 | 2021-10-22 | Reprogrammable iscb nucleases and uses thereof |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230392131A1 (enExample) |
| EP (1) | EP4203993A4 (enExample) |
| JP (1) | JP2023546671A (enExample) |
| KR (1) | KR20230111189A (enExample) |
| AU (1) | AU2021364399A1 (enExample) |
| CA (1) | CA3193961A1 (enExample) |
| IL (1) | IL302101A (enExample) |
| WO (1) | WO2022087494A1 (enExample) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10913941B2 (en) | 2019-02-14 | 2021-02-09 | Metagenomi Ip Technologies, Llc | Enzymes with RuvC domains |
| US20230040216A1 (en) * | 2019-11-19 | 2023-02-09 | The Broad Institute, Inc. | Retrotransposons and use thereof |
| CA3177051A1 (en) | 2020-03-31 | 2021-10-07 | Metagenomi, Inc. | Class ii, type ii crispr systems |
| EP4225928A4 (en) * | 2020-10-09 | 2025-04-30 | The Broad Institute, Inc. | GENETIC MODIFICATION USING A HELITRON |
| CN119452085A (zh) | 2021-08-27 | 2025-02-14 | 宏基因组学公司 | 具有ruvc结构域的酶 |
| WO2023097228A1 (en) * | 2021-11-23 | 2023-06-01 | The Broad Institute, Inc. | Reprogrammable iscb nucleases and uses thereof |
| KR20240110603A (ko) | 2021-11-24 | 2024-07-15 | 메타지노미, 인크. | 엔도뉴클레아제 시스템 |
| US20250283116A1 (en) * | 2022-05-06 | 2025-09-11 | Cornell University | Use of iscb in genome editing |
| WO2023230483A2 (en) * | 2022-05-23 | 2023-11-30 | The Broad Institute, Inc. | Engineered chimeric iscb polypeptides and uses thereof |
| JP2025525583A (ja) | 2022-07-19 | 2025-08-05 | ランパート バイオサイエンス インコーポレイテッド | 非免疫原性環状非ウイルス性ｄｎａベクター |
| WO2024038168A1 (en) * | 2022-08-19 | 2024-02-22 | UCB Biopharma SRL | Novel rna-guided nucleases and nucleic acid targeting systems comprising such |
| WO2024083135A1 (en) * | 2022-10-17 | 2024-04-25 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Iscb polypeptides and uses thereof |
| WO2024094084A1 (en) * | 2022-11-01 | 2024-05-10 | Huidagene Therapeutics Co., Ltd. | Iscb polypeptides and uses thereof |
| CN118620868A (zh) * | 2023-03-07 | 2024-09-10 | 上海科技大学 | IscBn-ωRNA编辑系统及其应用 |
| WO2024192141A1 (en) | 2023-03-13 | 2024-09-19 | Dana-Farber Cancer Institute, Inc. | Treatment of cancers having a drug-resistant mesenchymal cell state |
| WO2024192291A1 (en) | 2023-03-15 | 2024-09-19 | Renagade Therapeutics Management Inc. | Delivery of gene editing systems and methods of use thereof |
| WO2024197185A1 (en) | 2023-03-21 | 2024-09-26 | The Broad Institute, Inc. | Methods and compositions for dissecting organelle physiology |
| WO2024206825A2 (en) * | 2023-03-31 | 2024-10-03 | Aera Therapeutics, Inc. | Modified iscb nucleases, modified guide rnas, and uses thereof |
| WO2024220715A2 (en) * | 2023-04-19 | 2024-10-24 | Mammoth Biosciences, Inc. | Effector proteins and uses thereof |
| WO2024226838A2 (en) | 2023-04-25 | 2024-10-31 | The Brigham And Women's Hospital, Inc. | Treatment of autoimmune diseases having a pathogenic t cell state |
| WO2025007044A1 (en) * | 2023-06-28 | 2025-01-02 | The University Of Chicago | Methods and compositions comprising iscb variants |
| WO2025049788A1 (en) | 2023-08-29 | 2025-03-06 | The Broad Institute, Inc. | Optical genetic screens of intracellular and intercellular transcriptional circuits with perturb-fish |
| EP4771154A2 (en) | 2023-09-01 | 2026-07-08 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025096916A1 (en) | 2023-11-03 | 2025-05-08 | The Broad Institute, Inc. | Multi-site editing in living cells |
| WO2025100980A1 (ko) * | 2023-11-08 | 2025-05-15 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| WO2025101679A1 (en) * | 2023-11-10 | 2025-05-15 | Mammoth Biosciences, Inc. | Effector proteins and uses thereof |
| 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 |
| WO2025129158A1 (en) | 2023-12-15 | 2025-06-19 | The Broad Institute, Inc. | Engineered arc delivery vesicles and uses thereof |
| WO2025149083A1 (en) * | 2024-01-11 | 2025-07-17 | Huidagene Therapeutics Co., Ltd. | Iscb polypeptides and uses thereof |
| WO2025155923A1 (en) | 2024-01-17 | 2025-07-24 | The Broad Institute, Inc. | Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor |
| WO2025160155A1 (en) | 2024-01-22 | 2025-07-31 | The Broad Institute, Inc. | Epigenetic targeting of prion diseases |
| 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 |
| WO2025217174A1 (en) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv |
| WO2025217163A2 (en) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Novel aav capsids binding to human cd59 |
| WO2026107393A2 (en) | 2024-11-14 | 2026-05-21 | The Broad Institute, Inc. | Aav capsid modifications that reduce immunogenicity |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018035250A1 (en) * | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Methods for identifying class 2 crispr-cas systems |
| US12534714B2 (en) * | 2019-03-18 | 2026-01-27 | The Broad Institute, Inc. | Type VII CRISPR proteins and systems |
| WO2020191241A1 (en) * | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
-
2021
- 2021-10-22 EP EP21884039.5A patent/EP4203993A4/en active Pending
- 2021-10-22 AU AU2021364399A patent/AU2021364399A1/en active Pending
- 2021-10-22 KR KR1020237015731A patent/KR20230111189A/ko active Pending
- 2021-10-22 IL IL302101A patent/IL302101A/en unknown
- 2021-10-22 US US18/032,868 patent/US20230392131A1/en active Pending
- 2021-10-22 WO PCT/US2021/056361 patent/WO2022087494A1/en not_active Ceased
- 2021-10-22 JP JP2023524521A patent/JP2023546671A/ja active Pending
- 2021-10-22 CA CA3193961A patent/CA3193961A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2021364399A1 (en) | 2023-05-11 |
| JP2023546671A (ja) | 2023-11-07 |
| KR20230111189A (ko) | 2023-07-25 |
| EP4203993A1 (en) | 2023-07-05 |
| CA3193961A1 (en) | 2022-04-28 |
| AU2021364399A9 (en) | 2023-07-06 |
| WO2022087494A1 (en) | 2022-04-28 |
| US20230392131A1 (en) | 2023-12-07 |
| EP4203993A4 (en) | 2024-10-23 |
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