KR20210055733A - 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 - Google Patents
핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 Download PDFInfo
- Publication number
- KR20210055733A KR20210055733A KR1020217009863A KR20217009863A KR20210055733A KR 20210055733 A KR20210055733 A KR 20210055733A KR 1020217009863 A KR1020217009863 A KR 1020217009863A KR 20217009863 A KR20217009863 A KR 20217009863A KR 20210055733 A KR20210055733 A KR 20210055733A
- Authority
- KR
- South Korea
- Prior art keywords
- cas9
- terminal fragment
- deaminase
- polynucleotide
- fused
- 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.)
- Ceased
Links
Images
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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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]
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
- C12N15/113—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
-
- 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
-
- 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
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- 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)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04002—Adenine deaminase (3.5.4.2)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
- C07K2319/92—Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
-
- 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]
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862728703P | 2018-09-07 | 2018-09-07 | |
| US62/728,703 | 2018-09-07 | ||
| US201862779404P | 2018-12-13 | 2018-12-13 | |
| US62/779,404 | 2018-12-13 | ||
| PCT/US2019/050111 WO2020051561A1 (en) | 2018-09-07 | 2019-09-07 | Compositions and methods for delivering a nucleobase editing system |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20210055733A true KR20210055733A (ko) | 2021-05-17 |
Family
ID=69722796
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020217009863A Ceased KR20210055733A (ko) | 2018-09-07 | 2019-09-07 | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US12529041B2 (https=) |
| EP (1) | EP3847254A4 (https=) |
| JP (2) | JP7657711B2 (https=) |
| KR (1) | KR20210055733A (https=) |
| CN (1) | CN112969790B (https=) |
| AU (1) | AU2019336245B2 (https=) |
| CA (1) | CA3112011A1 (https=) |
| WO (1) | WO2020051561A1 (https=) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11649442B2 (en) | 2017-09-08 | 2023-05-16 | The Regents Of The University Of California | RNA-guided endonuclease fusion polypeptides and methods of use thereof |
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| AU2020215730A1 (en) | 2019-01-31 | 2021-07-29 | Beam Therapeutics Inc. | Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors |
| CN120174005A (zh) | 2019-02-13 | 2025-06-20 | 比姆医疗股份有限公司 | 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞 |
| 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 |
| JP7759311B2 (ja) | 2019-07-19 | 2025-10-23 | フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー | リコンビナーゼ組成物及び使用方法 |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| CN116209756A (zh) | 2020-03-04 | 2023-06-02 | 旗舰先锋创新Vi有限责任公司 | 调控基因组的方法和组合物 |
| JP2023518395A (ja) | 2020-03-19 | 2023-05-01 | インテリア セラピューティクス,インコーポレイテッド | 指向性ゲノム編集のための方法及び組成物 |
| MX2022012683A (es) * | 2020-04-09 | 2023-01-11 | Verve Therapeutics Inc | Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades. |
| 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 |
| CA3196831A1 (en) | 2020-09-25 | 2022-03-31 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| CA3198671A1 (en) * | 2020-10-14 | 2022-04-21 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| CA3227103A1 (en) | 2021-07-30 | 2023-02-02 | Matthew P. GEMBERLING | Compositions and methods for modulating expression of frataxin (fxn) |
| US20240252684A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| AU2022343268A1 (en) | 2021-09-08 | 2024-03-28 | Flagship Pioneering Innovations Vi, Llc | Methods and compositions for modulating a genome |
| EP4441074A2 (en) | 2021-12-03 | 2024-10-09 | The Broad Institute, Inc. | Compositions and methods for efficient in vivo delivery |
| WO2023169454A1 (zh) * | 2022-03-08 | 2023-09-14 | 中国科学院遗传与发育生物学研究所 | 腺嘌呤脱氨酶及其在碱基编辑中的用途 |
| CN120303407A (zh) | 2022-05-17 | 2025-07-11 | 恩维洛普治疗有限责任公司 | 用于有效体内递送的组合物和方法 |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| EP4612166A1 (en) * | 2022-11-01 | 2025-09-10 | Memorial Sloan-Kettering Cancer Center | Intein-based sorting system and modular chimeric polypeptides |
| 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) |
| WO2024233941A1 (en) * | 2023-05-11 | 2024-11-14 | Massachusetts Eye And Ear Infirmary | Base editing approaches to treat abca4-associated stargardt disease |
| AU2024279278A1 (en) | 2023-05-31 | 2025-12-18 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025038955A1 (en) * | 2023-08-16 | 2025-02-20 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Systems and methods for improving safety of split intein aav mediated gene therapy |
| CN117568313B (zh) * | 2024-01-15 | 2024-04-26 | 上海贝斯昂科生物科技有限公司 | 基因编辑组合物及其用途 |
| WO2025194124A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | Modified st1cas9 guide nucleic acids |
| WO2025194138A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | St1cas9 compositions and methods for modulating a genome |
| WO2025243196A1 (en) * | 2024-05-21 | 2025-11-27 | Fondazione Istituto Italiano Di Tecnologia | Gene regulation system (tuser) |
| 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 |
Family Cites Families (197)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH0329734A (ja) | 1989-06-20 | 1991-02-07 | Kubota Corp | ラベルプリンタ |
| EP1235914A2 (en) | 1999-11-24 | 2002-09-04 | Joseph Rosenecker | Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells |
| US20040003420A1 (en) | 2000-11-10 | 2004-01-01 | Ralf Kuhn | Modified recombinase |
| US20050222030A1 (en) | 2001-02-21 | 2005-10-06 | Anthony Allison | Modified annexin proteins and methods for preventing thrombosis |
| WO2002068676A2 (en) | 2001-02-27 | 2002-09-06 | University Of Rochester | METHODS AND COMPOSITIONS FOR MODIFYING APOLIPOPROTEIN B mRNA EDITING |
| AU2002330714A1 (en) | 2001-05-30 | 2003-01-02 | Biomedical Center | In silico screening for phenotype-associated expressed sequences |
| US7300829B2 (en) | 2003-06-02 | 2007-11-27 | Applied Materials, Inc. | Low temperature process for TFT fabrication |
| WO2010132092A2 (en) | 2009-05-12 | 2010-11-18 | The Scripps Research Institute | Cytidine deaminase fusions and related methods |
| US20110104787A1 (en) | 2009-11-05 | 2011-05-05 | President And Fellows Of Harvard College | Fusion Peptides That Bind to and Modify Target Nucleic Acid Sequences |
| EP2513296A4 (en) | 2009-12-18 | 2013-05-22 | Univ Leland Stanford Junior | USE OF CYTIDINE DAMINASE ASSOCIATED ACTIVITIES TO PROMOTE DEMETHYLATION AND CELL REPROGRAMMEIRING |
| WO2012004778A1 (en) | 2010-07-09 | 2012-01-12 | Ecole Polytechnique Federale De Lausanne (Epfl) | Method for in-vitro monitoring of neuronal disorders and use thereof |
| KR102096534B1 (ko) | 2011-09-28 | 2020-04-03 | 에라 바이오테크, 에스.에이. | 분할된 인테인 및 그의 이용 |
| CN103088008B (zh) | 2011-10-31 | 2014-08-20 | 中国科学院微生物研究所 | 胞苷脱氨酶及其编码基因和它们的应用 |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| US20150017136A1 (en) | 2013-07-15 | 2015-01-15 | Cellectis | Methods for engineering allogeneic and highly active t cell for immunotherapy |
| WO2013188037A2 (en) | 2012-06-11 | 2013-12-19 | Agilent Technologies, Inc | Method of adaptor-dimer subtraction using a crispr cas6 protein |
| EP3431497B1 (en) | 2012-06-27 | 2022-07-27 | The Trustees of Princeton University | Split inteins, conjugates and uses thereof |
| PL3360964T3 (pl) | 2012-12-06 | 2020-03-31 | Sigma-Aldrich Co. Llc | Modyfikacja i regulacja genomu oparta na crispr |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US20140273230A1 (en) | 2013-03-15 | 2014-09-18 | Sigma-Aldrich Co., Llc | Crispr-based genome modification and regulation |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| FI2997141T3 (fi) | 2013-05-13 | 2022-12-15 | CD19-spesifinen kimeerinen antigeenireseptori ja sen käyttöjä | |
| WO2014186686A2 (en) | 2013-05-17 | 2014-11-20 | Two Blades Foundation | Targeted mutagenesis and genome engineering in plants using rna-guided cas nucleases |
| US9267135B2 (en) | 2013-06-04 | 2016-02-23 | President And Fellows Of Harvard College | RNA-guided transcriptional regulation |
| WO2014197748A2 (en) | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| EP3019595A4 (en) | 2013-07-09 | 2016-11-30 | THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS | |
| WO2015021426A1 (en) | 2013-08-09 | 2015-02-12 | Sage Labs, Inc. | A crispr/cas system-based novel fusion protein and its application in genome editing |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| EP3835419A1 (en) | 2013-12-12 | 2021-06-16 | The Regents of The University of California | Methods and compositions for modifying a single stranded target nucleic acid |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| JP7103750B2 (ja) | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用 |
| ES2918501T3 (es) | 2013-12-19 | 2022-07-18 | Novartis Ag | Receptores de antígenos quiméricos de mesotelina humana y usos de los mismos |
| WO2015092024A2 (en) | 2013-12-20 | 2015-06-25 | Cellectis | Method of engineering multi-input signal sensitive t cell for immunotherapy |
| EP3115457B1 (en) | 2014-03-05 | 2019-10-02 | National University Corporation Kobe University | Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same |
| US20170335281A1 (en) | 2014-03-15 | 2017-11-23 | Novartis Ag | Treatment of cancer using chimeric antigen receptor |
| EP3155116A4 (en) | 2014-06-10 | 2017-12-27 | Massachusetts Institute Of Technology | Method for gene editing |
| KR20170032406A (ko) | 2014-07-15 | 2017-03-22 | 주노 쎄러퓨티크스 인코퍼레이티드 | 입양 세포 치료를 위한 조작된 세포 |
| US10077453B2 (en) * | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| US10544201B2 (en) | 2014-07-31 | 2020-01-28 | Cellectis | ROR1 specific multi-chain chimeric antigen receptor |
| EP3186375A4 (en) | 2014-08-28 | 2019-03-13 | North Carolina State University | Novel CAS9 PROTEINS AND CHARACTERISTICS FOR DNA TARGETING AND GENOME EDITING |
| BR112017007765B1 (pt) | 2014-10-14 | 2023-10-03 | Halozyme, Inc | Composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo |
| WO2016061368A1 (en) | 2014-10-15 | 2016-04-21 | The Children's Hospital Of Philadelphia | Compositions and methods for treating b-lymphoid malignancies |
| US10258697B2 (en) | 2014-10-29 | 2019-04-16 | Massachusetts Eye And Ear Infirmary | Efficient delivery of therapeutic molecules in vitro and in vivo |
| PT3216867T (pt) | 2014-11-04 | 2020-07-16 | Univ Kobe Nat Univ Corp | Método para modificar a sequência de genoma para introduzir mutação específica a sequência de adn alvo por reação de remoção de bases, e complexo molecular nele utilizado |
| MA40921A (fr) | 2014-11-05 | 2017-09-12 | Abbvie Stemcentrx Llc | Récepteurs antigéniques chimériques anti-cldn et procédés d'utilisation |
| WO2016075612A1 (en) | 2014-11-12 | 2016-05-19 | Rinat Neuroscience Corp. | Inhibitory chimeric antigen receptors |
| HRP20191873T1 (hr) | 2014-12-12 | 2020-01-24 | Bluebird Bio, Inc. | Kimerni antigenski receptori |
| EP3234134B1 (en) | 2014-12-17 | 2020-05-27 | ProQR Therapeutics II B.V. | Targeted rna editing |
| WO2016112242A1 (en) | 2015-01-08 | 2016-07-14 | President And Fellows Of Harvard College | Split cas9 proteins |
| MA41613A (fr) | 2015-02-23 | 2018-01-02 | Abbvie Stemcentrx Llc | Récepteurs antigéniques chimériques anti-dll3 et procédés d'utilisation desdits récepteurs |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| JP2018509148A (ja) | 2015-03-11 | 2018-04-05 | セレクティスCellectis | 患者における持続性および/または生着を増加させるために同種t細胞を改変する方法 |
| CN106011104B (zh) * | 2015-05-21 | 2019-09-27 | 清华大学 | 利用拆分Cas系统进行基因编辑和表达调控方法 |
| WO2016196388A1 (en) | 2015-05-29 | 2016-12-08 | Juno Therapeutics, Inc. | Composition and methods for regulating inhibitory interactions in genetically engineered cells |
| WO2016205759A1 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| JP7044373B2 (ja) | 2015-07-15 | 2022-03-30 | ラトガース,ザ ステート ユニバーシティ オブ ニュージャージー | ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途 |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| US11008555B2 (en) | 2015-09-17 | 2021-05-18 | The Regents Of The University Of California | Variant Cas9 polypeptides comprising internal insertions |
| AU2016323985B2 (en) | 2015-09-17 | 2022-12-15 | Novartis Ag | CAR T cell therapies with enhanced efficacy |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| BR112018008442A2 (pt) | 2015-11-05 | 2018-11-06 | Juno Therapeutics Inc | receptores quiméricos contendo domínios induto-res de traf e composições e métodos relacionados |
| US11020429B2 (en) | 2015-11-05 | 2021-06-01 | Juno Therapeutics, Inc. | Vectors and genetically engineered immune cells expressing metabolic pathway modulators and uses in adoptive cell therapy |
| BR112018008971A2 (pt) | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| CN109715801B (zh) | 2015-12-01 | 2022-11-01 | 克里斯普治疗股份公司 | 用于治疗α1抗胰蛋白酶缺乏的材料和方法 |
| PT3408292T (pt) | 2016-01-29 | 2023-07-19 | Univ Princeton | Inteínas divididas com excecional atividade de splicing |
| US11512311B2 (en) | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
| LT3436077T (lt) | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| EP3443002A4 (en) | 2016-04-14 | 2019-12-04 | Bluebird Bio, Inc. | SYSTEMS FOR RECOVERED CHIMERIC ANTIGEN RECEPTORS |
| KR20260004568A (ko) | 2016-04-19 | 2026-01-08 | 더 브로드 인스티튜트, 인코퍼레이티드 | 신규한 crispr 효소 및 시스템 |
| AU2017257274B2 (en) | 2016-04-19 | 2023-07-13 | Massachusetts Institute Of Technology | Novel CRISPR enzymes and systems |
| WO2018020323A2 (en) | 2016-07-25 | 2018-02-01 | Crispr Therapeutics Ag | Materials and methods for treatment of fatty acid disorders |
| KR102546839B1 (ko) | 2016-08-03 | 2023-06-23 | 워싱턴 유니버시티 | 키메라 항원 수용체를 이용한 t 세포 악성종양의 치료를 위한 car-t 세포의 유전자 편집 |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| EP4485466A3 (en) | 2016-08-17 | 2025-04-02 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| IL315358A (en) | 2016-08-18 | 2024-11-01 | Univ California | CRISPR-CAS genome engineering using a modular AAV delivery system |
| CN110352244B (zh) | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| WO2018085690A1 (en) | 2016-11-04 | 2018-05-11 | Bluebird Bio, Inc. | Anti-bcma car t cell compositions |
| EP3538561A4 (en) | 2016-11-11 | 2020-10-21 | The Regents of The University of California | RNA-GUIDED VARIANT POLYPEPTIDES AND THEIR METHODS OF USE |
| CN107043779B (zh) | 2016-12-01 | 2020-05-12 | 中国农业科学院作物科学研究所 | 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用 |
| SG10202106058WA (en) | 2016-12-08 | 2021-07-29 | Intellia Therapeutics Inc | Modified guide rnas |
| TWI851534B (zh) | 2016-12-22 | 2024-08-11 | 美商英特利亞醫療公司 | 用於治療α-1抗胰蛋白酶缺乏症之組合物及方法 |
| KR20230125856A (ko) | 2016-12-23 | 2023-08-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Pcsk9의 유전자 편집 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3565608A4 (en) | 2017-01-05 | 2020-12-16 | Rutgers, The State University of New Jersey | TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES |
| WO2018152197A1 (en) | 2017-02-15 | 2018-08-23 | Massachusetts Institute Of Technology | Dna writers, molecular recorders and uses thereof |
| RU2019130504A (ru) | 2017-02-28 | 2021-03-30 | Вор Байофарма, Инк. | Композиции и способы ингибирования линиеспецифических белков |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| CN106916852B (zh) | 2017-04-13 | 2020-12-04 | 上海科技大学 | 一种碱基编辑系统及其构建和应用方法 |
| US11591589B2 (en) | 2017-04-21 | 2023-02-28 | The General Hospital Corporation | Variants of Cpf1 (Cas12a) with altered PAM specificity |
| US11866697B2 (en) | 2017-05-18 | 2024-01-09 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| WO2018213726A1 (en) | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| EP3630849A4 (en) | 2017-05-25 | 2021-01-13 | The General Hospital Corporation | BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES |
| CA3065946A1 (en) | 2017-06-05 | 2018-12-13 | Research Institute At Nationwide Children's Hospital | Enhanced modified viral capsid proteins |
| WO2019005886A1 (en) | 2017-06-26 | 2019-01-03 | The Broad Institute, Inc. | CRISPR / CAS-CYTIDINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED EDITING OF NUCLEIC ACIDS |
| KR102761791B1 (ko) | 2017-06-26 | 2025-02-05 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법 |
| US10392616B2 (en) | 2017-06-30 | 2019-08-27 | Arbor Biotechnologies, Inc. | CRISPR RNA targeting enzymes and systems and uses thereof |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| CA3073448A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| WO2019071274A1 (en) | 2017-10-06 | 2019-04-11 | Oregon Health & Science University | Compositions and methods for editing rna |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| CN109957569B (zh) | 2017-12-22 | 2022-10-25 | 苏州齐禾生科生物科技有限公司 | 基于cpf1蛋白的碱基编辑系统和方法 |
| US20210363518A1 (en) | 2018-03-19 | 2021-11-25 | University Of Massachusetts | Modified guide rnas for crispr genome editing |
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| KR20250133482A (ko) | 2018-05-11 | 2025-09-05 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법 |
| CN119570787A (zh) | 2018-05-11 | 2025-03-07 | 比姆医疗股份有限公司 | 使用可编程碱基编辑器系统遏止病原性突变的方法 |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| CA3108281A1 (en) | 2018-08-03 | 2020-02-06 | Beam Therapeutics Inc. | Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence |
| EP3841203A4 (en) | 2018-08-23 | 2022-11-02 | The Broad Institute Inc. | CAS9 VARIANTS WITH NON-CANONICAL PAM SPECIFICITIES AND THEIR USES |
| US11530245B2 (en) | 2018-08-31 | 2022-12-20 | Washington University | Split intein mediated polymerization and production of mussel foot adhesive protein materials |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| CN109295186B (zh) | 2018-09-30 | 2023-10-03 | 中山大学 | 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用 |
| IL282362B2 (en) | 2018-10-15 | 2025-10-01 | Fond Telethon Ets | Intein proteins and their uses |
| EP3886869A4 (en) | 2018-11-28 | 2022-07-06 | Forty Seven, Inc. | GENETICALLY MODIFIED ABLATION-RESISTANT HSPCS |
| EP3887521A1 (en) | 2018-11-29 | 2021-10-06 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| US11667682B2 (en) | 2018-12-13 | 2023-06-06 | Washington University | Split intein mediated protein polymerization for microbial production of materials |
| WO2020146627A1 (en) | 2019-01-10 | 2020-07-16 | California Institute Of Technology | A synthetic system for tunable thresholding of protein signals |
| WO2020160517A1 (en) | 2019-01-31 | 2020-08-06 | Beam Therapeutics Inc. | Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence |
| AU2020215730A1 (en) | 2019-01-31 | 2021-07-29 | Beam Therapeutics Inc. | Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors |
| US11946040B2 (en) | 2019-02-04 | 2024-04-02 | The General Hospital Corporation | Adenine DNA base editor variants with reduced off-target RNA editing |
| CN120174005A (zh) | 2019-02-13 | 2025-06-20 | 比姆医疗股份有限公司 | 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞 |
| US20220098593A1 (en) | 2019-02-13 | 2022-03-31 | Beam Therapeutics Inc. | Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease |
| JP7693552B2 (ja) | 2019-02-13 | 2025-06-17 | ビーム セラピューティクス インク. | アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法 |
| CA3128755C (en) | 2019-02-13 | 2024-06-04 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| KR20210126680A (ko) | 2019-02-13 | 2021-10-20 | 빔 테라퓨틱스, 인크. | 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법 |
| US20230140953A1 (en) | 2019-02-13 | 2023-05-11 | Beam Therapeutics Inc. | Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease |
| WO2020168088A1 (en) | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| KR102129377B1 (ko) | 2019-02-21 | 2020-07-02 | 성균관대학교산학협력단 | 특정 특성이 발현된 단백질을 암호화하는 플라스미드를 선별하는 유도 플라스미드 디스플레이 시스템 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3139324A1 (en) | 2019-05-10 | 2020-11-19 | Beam Therapeutics Inc. | Compositions and methods for treating hepatitis b |
| US20220249697A1 (en) | 2019-05-20 | 2022-08-11 | The Broad Institute, Inc. | Aav delivery of nucleobase editors |
| AU2020279751A1 (en) | 2019-05-21 | 2021-12-02 | Beam Therapeutics Inc. | Methods of editing a single nucleotide polymorphism using programmable base editor systems |
| 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 |
| JP7759311B2 (ja) | 2019-07-19 | 2025-10-23 | フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー | リコンビナーゼ組成物及び使用方法 |
| WO2021022043A2 (en) | 2019-07-30 | 2021-02-04 | Pairwise Plants Services, Inc. | Morphogenic regulators and methods of using the same |
| KR102258713B1 (ko) | 2019-07-31 | 2021-05-31 | 한양대학교 산학협력단 | 사이토신 염기교정용 조성물 및 이의 용도 |
| JP7781742B2 (ja) | 2019-08-29 | 2025-12-08 | ビーム セラピューティクス インク. | 転写または発現を可能にするように変異を編集するための組成物および方法 |
| WO2021042062A2 (en) | 2019-08-30 | 2021-03-04 | Joung J Keith | Combinatorial adenine and cytosine dna base editors |
| AU2020344547A1 (en) | 2019-09-09 | 2022-03-24 | Beam Therapeutics Inc. | Novel nucleobase editors and methods of using same |
| AU2020466994A1 (en) | 2019-09-17 | 2022-04-21 | Rutgers, The State University Of New Jersey | Highly efficient DNA base editors mediated by RNA-aptamer recruitment for targeted genome modification and uses thereof |
| KR20210035752A (ko) | 2019-09-24 | 2021-04-01 | 성균관대학교산학협력단 | 항-바이러스 활성이 향상된 나노천공자 |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| WO2021081264A1 (en) | 2019-10-24 | 2021-04-29 | Pairwise Plants Services, Inc. | Optimized crispr-cas nucleases and base editors and methods of use thereof |
| EP4051788A4 (en) | 2019-10-30 | 2023-12-06 | Pairwise Plants Services, Inc. | TYPE V CRISPR CAS BASE EDITORS AND METHODS OF USE THEREOF |
| CA3162499A1 (en) | 2019-11-22 | 2021-05-27 | Flagship Pioneering Innovations Vi, Llc | Recombinase compositions and methods of use |
| US20230086199A1 (en) | 2019-11-26 | 2023-03-23 | The Broad Institute, Inc. | Systems and methods for evaluating cas9-independent off-target editing of nucleic acids |
| GB201918693D0 (en) | 2019-12-18 | 2020-01-29 | Univ Southampton | Peptide |
| EP4100519A2 (en) | 2020-02-05 | 2022-12-14 | The Broad Institute, Inc. | Adenine base editors and uses thereof |
| KR20230053735A (ko) | 2020-03-04 | 2023-04-21 | 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 | 게놈의 조정을 위한 개선된 방법 및 조성물 |
| BR112022017713A2 (pt) | 2020-03-04 | 2022-11-16 | Flagship Pioneering Innovations Vi Llc | Métodos e composições para modular um genoma |
| CN116209756A (zh) | 2020-03-04 | 2023-06-02 | 旗舰先锋创新Vi有限责任公司 | 调控基因组的方法和组合物 |
| CA3174553A1 (en) | 2020-03-05 | 2021-09-10 | Flagship Pioneering Innovations Vi, Llc | Host defense suppressing methods and compositions for modulating a genome |
| JP2023518395A (ja) | 2020-03-19 | 2023-05-01 | インテリア セラピューティクス,インコーポレイテッド | 指向性ゲノム編集のための方法及び組成物 |
| EP3885440A1 (en) | 2020-03-26 | 2021-09-29 | Splicebio, S.L. | Split inteins and their uses |
| EP4132951A4 (en) | 2020-04-09 | 2025-06-11 | Verve Therapeutics, Inc. | CHEMICALLY MODIFIED GENE EDITING GUIDE RNAS WITH CAS12B |
| WO2021209574A1 (en) | 2020-04-15 | 2021-10-21 | Fondazione Telethon | Constructs comprising inteins |
| EP4143315A1 (en) | 2020-04-28 | 2023-03-08 | The Broad Institute Inc. | <smallcaps/>? ? ?ush2a? ? ? ? ?targeted base editing of thegene |
| WO2022008935A1 (en) | 2020-07-10 | 2022-01-13 | Horizon Discovery Limited | Method for producing genetically modified cells |
| US20240247242A1 (en) | 2020-07-15 | 2024-07-25 | LifeEDIT Therapeutics, Inc. | Uracil stabilizing proteins and active fragments and variants thereof and methods of use |
| 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 |
| JP2023540797A (ja) | 2020-09-11 | 2023-09-26 | メタゲノミ,インコーポレイテッド | 塩基編集酵素 |
| MX2023002848A (es) | 2020-09-11 | 2023-03-22 | Lifeedit Therapeutics Inc | Enzimas modificadoras del acido desoxirribonucleico (adn) y fragmentos activos y sus variantes y metodos de uso. |
| CA3198671A1 (en) | 2020-10-14 | 2022-04-21 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| IT202000028688A1 (it) | 2020-11-27 | 2022-05-27 | Consiglio Nazionale Ricerche | Varianti della citidina deaminasi per l’editazione di basi |
| CA3207144A1 (en) | 2021-01-05 | 2022-07-14 | Horizon Discovery Limited | Method for producing genetically modified cells |
| WO2022150372A1 (en) | 2021-01-05 | 2022-07-14 | Horizon Discovery Ltd. | Guide rna designs and complexes for type v cas systems |
| JP2024502348A (ja) | 2021-01-05 | 2024-01-18 | ホライズン・ディスカバリー・リミテッド | Tracr不含 V型Casシステム用のガイドRNAデザインおよび複合体 |
| EP4274897A4 (en) | 2021-01-08 | 2025-03-05 | The General Hospital Corporation | GENOMEDITING APPROACHES TO THE TREATMENT OF SPINAL MUSCLE ATROPHY |
| CA3208612A1 (en) | 2021-02-19 | 2022-08-25 | Beam Therapeutics Inc. | Recombinant rabies viruses for gene therapy |
| EP4313118A4 (en) | 2021-03-26 | 2025-06-18 | Beam Therapeutics Inc. | ADENOSINE DEAMINASE VARIANTS AND THEIR USES |
| CN113813398B (zh) | 2021-04-23 | 2023-11-07 | 天津医科大学 | 一种向表面富含神经节苷脂的细胞递送重组蛋白质的方法 |
| US20240327859A1 (en) | 2021-07-02 | 2024-10-03 | University Of Maryland, College Park | Cytidine deaminases and methods of genome editing using the same |
| CA3225808A1 (en) | 2021-07-16 | 2023-01-19 | David R. Liu | Context-specific adenine base editors and uses thereof |
| CN113480613B (zh) | 2021-08-04 | 2022-04-15 | 江南大学 | 一种自剪切蛋白标签、改造方法及其应用 |
| CN113735941B (zh) | 2021-08-13 | 2024-01-30 | 江南大学 | 一种蛋白纯化方法及其应用 |
| US20240352439A1 (en) | 2021-09-03 | 2024-10-24 | The University Of Chicago | Polypeptides and methods for modifying nucleic acids |
| IL311607A (en) | 2021-09-22 | 2024-05-01 | Verve Therapeutics Inc | PCSK9 or ANGPTL3 gene editing and preparations and methods for using them to treat the disease |
| US20230105319A1 (en) | 2021-09-24 | 2023-04-06 | Crispr Therapeutics Ag | PRODRUG INCORPORATED sgRNA SYNTHESIS |
| WO2023125814A1 (zh) | 2021-12-29 | 2023-07-06 | 华东师范大学 | 腺嘌呤脱氨酶及其应用 |
| CN119013310A (zh) | 2022-02-17 | 2024-11-22 | 正序(上海)生物科技有限公司 | 具有改善的编辑精确度的突变的胞嘧啶脱氨酶 |
| CN114634923B (zh) | 2022-04-07 | 2024-02-23 | 尧唐(上海)生物科技有限公司 | 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途 |
| EP4532704A2 (en) | 2022-05-26 | 2025-04-09 | UCB Biopharma SRL | Novel nucleic acid-editing proteins |
| EP4540378A1 (en) | 2022-06-20 | 2025-04-23 | CRISPR Therapeutics AG | Base editing proteins and uses thereof |
| AU2023288909A1 (en) | 2022-06-23 | 2025-01-09 | Basf Agricultural Solutions Us Llc | Diversifying base editing |
| WO2024040083A1 (en) | 2022-08-16 | 2024-02-22 | The Broad Institute, Inc. | Evolved cytosine deaminases and methods of editing dna using same |
| KR20250075667A (ko) | 2022-09-23 | 2025-05-28 | 기초과학연구원 | 신규한 아데닌 탈아미노효소 변이체 및 이를 사용한 염기 교정 방법 |
| EP4594486A2 (en) | 2022-09-26 | 2025-08-06 | Beam Therapeutics Inc. | Synthetic polypeptides and uses thereof |
| WO2024179426A2 (en) | 2023-02-28 | 2024-09-06 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Deaminases for use in base editing |
| EP4688814A1 (en) | 2023-04-27 | 2026-02-11 | University of Massachusetts | Cas-embedded cytidine deaminase ribonucleoprotein complexes having improved base editing specificity and efficiency |
| CN121079412A (zh) | 2023-04-28 | 2025-12-05 | 比姆医疗股份有限公司 | 经修饰的指导rna |
| AU2024303631A1 (en) | 2023-06-15 | 2025-12-11 | Beam Therapeutics Inc. | Methods and compositions for treating alpha-1 antitrypsin deficiency |
| CN117965505A (zh) | 2023-06-28 | 2024-05-03 | 微光基因(苏州)有限公司 | 工程化的腺苷脱氨酶及碱基编辑器 |
-
2019
- 2019-09-07 EP EP19857653.0A patent/EP3847254A4/en active Pending
- 2019-09-07 AU AU2019336245A patent/AU2019336245B2/en active Active
- 2019-09-07 KR KR1020217009863A patent/KR20210055733A/ko not_active Ceased
- 2019-09-07 US US17/273,209 patent/US12529041B2/en active Active
- 2019-09-07 JP JP2021512514A patent/JP7657711B2/ja active Active
- 2019-09-07 WO PCT/US2019/050111 patent/WO2020051561A1/en not_active Ceased
- 2019-09-07 CN CN201980073055.6A patent/CN112969790B/zh active Active
- 2019-09-07 CA CA3112011A patent/CA3112011A1/en active Pending
-
2024
- 2024-11-01 JP JP2024192680A patent/JP2025028857A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US12529041B2 (en) | 2026-01-20 |
| AU2019336245A1 (en) | 2021-04-01 |
| JP2022500017A (ja) | 2022-01-04 |
| CN112969790A (zh) | 2021-06-15 |
| AU2019336245B2 (en) | 2026-02-26 |
| JP2025028857A (ja) | 2025-03-05 |
| CN112969790B (zh) | 2024-07-05 |
| JP7657711B2 (ja) | 2025-04-07 |
| EP3847254A4 (en) | 2022-08-10 |
| CA3112011A1 (en) | 2020-03-12 |
| WO2020051561A1 (en) | 2020-03-12 |
| US20210301274A1 (en) | 2021-09-30 |
| EP3847254A1 (en) | 2021-07-14 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| KR20210055733A (ko) | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 | |
| JP7646552B2 (ja) | 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ | |
| KR102852347B1 (ko) | 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법 | |
| CN114040970B (zh) | 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法,包括遗传性疾病的治疗 | |
| US12454694B2 (en) | Compositions and methods for improving base editing | |
| AU2019282149B2 (en) | RNA-guided nucleases and active fragments and variants thereof and methods of use | |
| JP7679311B2 (ja) | プログラム可能塩基エディターシステムを用いた一塩基多型編集法 | |
| CA3108281A1 (en) | Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence | |
| CN116497067A (zh) | 治疗血红素病变的组合物和方法 | |
| KR20250107288A (ko) | 아데노신 염기 편집제의 용도 | |
| WO2020168088A1 (en) | Compositions and methods for treating glycogen storage disease type 1a | |
| KR20240004276A (ko) | 아데노신 데아미나아제 변이체 및 이의 용도 | |
| CA3208612A1 (en) | Recombinant rabies viruses for gene therapy | |
| KR20240012377A (ko) | 염기 편집기의 자기-불활성화용 조성물 및 방법 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
St.27 status event code: A-0-1-A10-A15-nap-PA0105 |
|
| PG1501 | Laying open of application |
St.27 status event code: A-1-1-Q10-Q12-nap-PG1501 |
|
| A201 | Request for examination | ||
| E13-X000 | Pre-grant limitation requested |
St.27 status event code: A-2-3-E10-E13-lim-X000 |
|
| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
|
| P13-X000 | Application amended |
St.27 status event code: A-2-2-P10-P13-nap-X000 |
|
| PA0201 | Request for examination |
St.27 status event code: A-1-2-D10-D11-exm-PA0201 |
|
| R18-X000 | Changes to party contact information recorded |
St.27 status event code: A-3-3-R10-R18-oth-X000 |
|
| E902 | Notification of reason for refusal | ||
| PE0902 | Notice of grounds for rejection |
St.27 status event code: A-1-2-D10-D21-exm-PE0902 |
|
| E13 | Pre-grant limitation requested |
Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| E13-X000 | Pre-grant limitation requested |
St.27 status event code: A-2-3-E10-E13-lim-X000 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
|
| B15 | Application refused following examination |
Free format text: ST27 STATUS EVENT CODE: N-2-6-B10-B15-EXM-PE0601 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| PE0601 | Decision on rejection of patent |
St.27 status event code: N-2-6-B10-B15-exm-PE0601 |