AU2021268634A1 - Compositions, systems, and methods for the generation, identification, and characterization of effector domains for activating and silencing gene expression - Google Patents
Compositions, systems, and methods for the generation, identification, and characterization of effector domains for activating and silencing gene expression Download PDFInfo
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- AU2021268634A1 AU2021268634A1 AU2021268634A AU2021268634A AU2021268634A1 AU 2021268634 A1 AU2021268634 A1 AU 2021268634A1 AU 2021268634 A AU2021268634 A AU 2021268634A AU 2021268634 A AU2021268634 A AU 2021268634A AU 2021268634 A1 AU2021268634 A1 AU 2021268634A1
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- C—CHEMISTRY; METALLURGY
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- 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
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- 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
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1055—Protein x Protein interaction, e.g. two hybrid selection
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- 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
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1065—Preparation or screening of tagged libraries, e.g. tagged microorganisms by STM-mutagenesis, tagged polynucleotides, gene tags
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- 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
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1086—Preparation or screening of expression libraries, e.g. reporter assays
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
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- 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]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- 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]
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- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Bioinformatics & Computational Biology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Analytical Chemistry (AREA)
- Immunology (AREA)
- Medicinal Chemistry (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Detergent Compositions (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063019706P | 2020-05-04 | 2020-05-04 | |
| US63/019,706 | 2020-05-04 | ||
| US202063074793P | 2020-09-04 | 2020-09-04 | |
| US63/074,793 | 2020-09-04 | ||
| PCT/US2021/030643 WO2021226077A2 (en) | 2020-05-04 | 2021-05-04 | Compositions, systems, and methods for the generation, identification, and characterization of effector domains for activating and silencing gene expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2021268634A1 true AU2021268634A1 (en) | 2022-11-24 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2021268634A Pending AU2021268634A1 (en) | 2020-05-04 | 2021-05-04 | Compositions, systems, and methods for the generation, identification, and characterization of effector domains for activating and silencing gene expression |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20240401032A1 (https=) |
| EP (2) | EP4644573A3 (https=) |
| JP (1) | JP2023524733A (https=) |
| KR (1) | KR20230005984A (https=) |
| CN (1) | CN116234902A (https=) |
| AU (1) | AU2021268634A1 (https=) |
| CA (1) | CA3176046A1 (https=) |
| ES (1) | ES3036984T3 (https=) |
| WO (1) | WO2021226077A2 (https=) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
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| WO2020252455A1 (en) | 2019-06-13 | 2020-12-17 | The General Hospital Corporation | Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells |
| CA3189601A1 (en) | 2020-07-24 | 2022-01-27 | The General Hospital Corporation | Enhanced virus-like particles and methods of use thereof for delivery to cells |
| US20240252684A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| CA3227103A1 (en) | 2021-07-30 | 2023-02-02 | Matthew P. GEMBERLING | Compositions and methods for modulating expression of frataxin (fxn) |
| GB2625500B (en) | 2021-09-21 | 2026-04-01 | Scribe Therapeutics Inc | Engineered CasX repressor systems |
| US20250154503A1 (en) | 2022-01-14 | 2025-05-15 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression |
| US20250134999A1 (en) | 2022-01-14 | 2025-05-01 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation |
| IL316722A (en) | 2022-05-01 | 2024-12-01 | Chroma Medicine Inc | Compositions and methods for epigenetic regulation of PCSK9 expression |
| JP2025524455A (ja) | 2022-06-23 | 2025-07-30 | エヌクロマ・バイオ,インコーポレーテッド | Trac発現のエピジェネティック調節のための組成物及び方法 |
| EP4544058A1 (en) | 2022-06-23 | 2025-04-30 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of ciita expression |
| US20250387518A1 (en) | 2022-06-23 | 2025-12-25 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of b2m expression |
| IL317874A (en) | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| KR20250077608A (ko) | 2022-08-19 | 2025-05-30 | 튠 쎄라퓨틱스, 인코포레이티드 | 표적 유전자 억제를 통한 b형 간염 바이러스 조절용 조성물, 시스템 및 방법 |
| WO2024064642A2 (en) | 2022-09-19 | 2024-03-28 | Tune Therapeutics, Inc. | Compositions, systems, and methods for modulating t cell function |
| IL319704A (en) | 2022-09-23 | 2025-05-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of HBV gene expression |
| WO2024081879A1 (en) | 2022-10-14 | 2024-04-18 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of cd247 expression |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| WO2024163683A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist) |
| AU2024217832A1 (en) * | 2023-02-10 | 2025-10-02 | Integrate Bioscience Llc | Personalized crispr profiling for cancer |
| WO2024238700A1 (en) | 2023-05-15 | 2024-11-21 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2025029840A1 (en) | 2023-07-31 | 2025-02-06 | Tune Therapeutics, Inc. | Compositions and methods for multiplexed activation and repression of t cell gene expression |
| CN121909284A (zh) | 2023-07-31 | 2026-04-21 | 图恩疗法股份有限公司 | 用于调节il-2基因表达的组合物和方法 |
| WO2025038494A1 (en) | 2023-08-11 | 2025-02-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation |
| WO2025059073A1 (en) | 2023-09-11 | 2025-03-20 | Tune Therapeutics, Inc. | Epigenetic editing methods and systems for differentiating stem cells |
| CN117143257B (zh) * | 2023-10-31 | 2024-02-09 | 深圳市帝迈生物技术有限公司 | Trim28-krab-znf10二元复合物、制备方法及用于前列腺癌筛查的试剂盒 |
| WO2025260068A1 (en) | 2024-06-14 | 2025-12-18 | Tune Therapeutics, Inc. | Lipid nanoparticle formulation for delivery of nucleic acids to cells |
| WO2026015647A1 (en) | 2024-07-09 | 2026-01-15 | Tune Therapeutics, Inc. | Compositions, systems, and methods for cell differentiation using targeted gene activation of dll4 and/or vcam1 |
| WO2026064753A1 (en) | 2024-09-23 | 2026-03-26 | Tune Therapeutics, Inc. | Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing |
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| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US20030180777A1 (en) * | 2002-03-12 | 2003-09-25 | Victor Bartsevich | Rapid identification of transcriptional regulatory domains |
| US20060134670A1 (en) * | 2004-11-19 | 2006-06-22 | Fabrice Piu | Enabling tools to identify ligands for hormone nuclear receptors |
| WO2010033647A2 (en) * | 2008-09-16 | 2010-03-25 | Stanford University | Cysteine labeling system and method of use thereof |
| CN101892196B (zh) * | 2010-07-14 | 2012-10-10 | 北京大学 | 一种细胞分选磁珠及合成方法以及其在细胞分选中的应用 |
| EP2825561A4 (en) * | 2012-03-15 | 2016-03-09 | Permeon Biolog Inc | CELL PENETRATING COMPOSITIONS FOR THE DISPOSAL OF INTRA-CELLULAR ANTIBODIES AND ANTIBODY-RELATED UNITS AND METHODS OF USE |
| KR101355176B1 (ko) * | 2012-03-22 | 2014-01-27 | 한국과학기술원 | 혈중종양세포 진단용 조성물 및 이를 이용한 혈중종양세포 검출방법 |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| EP2700713B1 (en) * | 2012-08-21 | 2016-07-13 | Miltenyi Biotec GmbH | Screening and enrichment system for protein expression in eukaryotic cells using a tricistronic expression cassette |
| CA2954791C (en) * | 2014-07-14 | 2025-11-18 | The Regents Of The University Of California | CRISPR/CAS TRANSCRIPTIONAL MODULATION |
| IL260532B2 (en) * | 2016-01-11 | 2023-12-01 | Univ Leland Stanford Junior | Chimeric proteins- containing systems and uses thereof in regulating gene expression |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
| EP3404022B1 (en) * | 2017-05-19 | 2020-10-07 | Paris Sciences et Lettres - Quartier Latin | Membrane-impermeant fluorogenic chromophores |
| US11629342B2 (en) * | 2017-10-17 | 2023-04-18 | President And Fellows Of Harvard College | Cas9-based transcription modulation systems |
| WO2019089803A1 (en) * | 2017-10-31 | 2019-05-09 | The Broad Institute, Inc. | Methods and compositions for studying cell evolution |
| TWI908706B (zh) * | 2018-03-07 | 2025-12-21 | 美商波西達治療公司 | CARTyrin組成物和使用方法 |
| WO2019226588A1 (en) * | 2018-05-23 | 2019-11-28 | University Of Florida Research Foundation | Paramagnetic immunobeads for the isolation of human adipose-derived stem cells |
| US20210079404A9 (en) * | 2018-09-25 | 2021-03-18 | Massachusetts Institute Of Technology | Engineered dcas9 with reduced toxicity and its use in genetic circuits |
-
2021
- 2021-05-04 US US17/997,228 patent/US20240401032A1/en active Pending
- 2021-05-04 ES ES21800824T patent/ES3036984T3/es active Active
- 2021-05-04 JP JP2022567096A patent/JP2023524733A/ja active Pending
- 2021-05-04 EP EP25185379.2A patent/EP4644573A3/en active Pending
- 2021-05-04 KR KR1020227042321A patent/KR20230005984A/ko active Pending
- 2021-05-04 CN CN202180047231.6A patent/CN116234902A/zh active Pending
- 2021-05-04 WO PCT/US2021/030643 patent/WO2021226077A2/en not_active Ceased
- 2021-05-04 AU AU2021268634A patent/AU2021268634A1/en active Pending
- 2021-05-04 EP EP21800824.1A patent/EP4146801B1/en active Active
- 2021-05-04 CA CA3176046A patent/CA3176046A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230005984A (ko) | 2023-01-10 |
| JP2023524733A (ja) | 2023-06-13 |
| EP4644573A2 (en) | 2025-11-05 |
| EP4146801A4 (en) | 2024-06-26 |
| WO2021226077A2 (en) | 2021-11-11 |
| US20240401032A1 (en) | 2024-12-05 |
| ES3036984T3 (en) | 2025-09-26 |
| EP4146801A2 (en) | 2023-03-15 |
| CA3176046A1 (en) | 2021-11-11 |
| EP4146801B1 (en) | 2025-07-02 |
| WO2021226077A3 (en) | 2022-02-03 |
| EP4644573A3 (en) | 2026-01-14 |
| CN116234902A (zh) | 2023-06-06 |
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