JP6929791B2 - エピゲノム編集のための組成物および方法 - Google Patents
エピゲノム編集のための組成物および方法 Download PDFInfo
- Publication number
- JP6929791B2 JP6929791B2 JP2017560481A JP2017560481A JP6929791B2 JP 6929791 B2 JP6929791 B2 JP 6929791B2 JP 2017560481 A JP2017560481 A JP 2017560481A JP 2017560481 A JP2017560481 A JP 2017560481A JP 6929791 B2 JP6929791 B2 JP 6929791B2
- Authority
- JP
- Japan
- Prior art keywords
- dcas9
- gene
- target
- core
- base pairs
- 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.)
- Active
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
- 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
- C12N15/11—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
- C12N15/63—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
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal 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
- C12N9/10—Transferases (2.)
- C12N9/1025—Acyltransferases (2.3)
- C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y203/00—Acyltransferases (2.3)
- C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
- C12Y203/01048—Histone acetyltransferase (2.3.1.48)
-
- 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]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
- Bakery Products And Manufacturing Methods Therefor (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021102533A JP7553110B2 (ja) | 2015-02-09 | 2021-06-21 | エピゲノム編集のための組成物および方法 |
| JP2024061564A JP2024083479A (ja) | 2015-02-09 | 2024-04-05 | エピゲノム編集のための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562113569P | 2015-02-09 | 2015-02-09 | |
| US62/113,569 | 2015-02-09 | ||
| PCT/US2016/017221 WO2016130600A2 (en) | 2015-02-09 | 2016-02-09 | Compositions and methods for epigenome editing |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021102533A Division JP7553110B2 (ja) | 2015-02-09 | 2021-06-21 | エピゲノム編集のための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018505219A JP2018505219A (ja) | 2018-02-22 |
| JP2018505219A5 JP2018505219A5 (enExample) | 2019-03-28 |
| JP6929791B2 true JP6929791B2 (ja) | 2021-09-01 |
Family
ID=56615565
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017560481A Active JP6929791B2 (ja) | 2015-02-09 | 2016-02-09 | エピゲノム編集のための組成物および方法 |
| JP2021102533A Active JP7553110B2 (ja) | 2015-02-09 | 2021-06-21 | エピゲノム編集のための組成物および方法 |
| JP2024061564A Pending JP2024083479A (ja) | 2015-02-09 | 2024-04-05 | エピゲノム編集のための組成物および方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021102533A Active JP7553110B2 (ja) | 2015-02-09 | 2021-06-21 | エピゲノム編集のための組成物および方法 |
| JP2024061564A Pending JP2024083479A (ja) | 2015-02-09 | 2024-04-05 | エピゲノム編集のための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (4) | US10676726B2 (enExample) |
| EP (1) | EP3256487A4 (enExample) |
| JP (3) | JP6929791B2 (enExample) |
| WO (1) | WO2016130600A2 (enExample) |
Families Citing this family (93)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| KR102874079B1 (ko) | 2013-03-15 | 2025-10-22 | 더 제너럴 하스피탈 코포레이션 | Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용 |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| GB201418965D0 (enExample) | 2014-10-24 | 2014-12-10 | Ospedale San Raffaele And Fond Telethon | |
| EP3256487A4 (en) | 2015-02-09 | 2018-07-18 | Duke University | Compositions and methods for epigenome editing |
| WO2017015637A1 (en) | 2015-07-22 | 2017-01-26 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
| JP6905755B2 (ja) | 2015-08-25 | 2021-07-21 | デューク ユニバーシティ | Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法 |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| MX2018005377A (es) | 2015-11-30 | 2018-11-09 | Univ Duke | Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso. |
| WO2017180915A2 (en) | 2016-04-13 | 2017-10-19 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| WO2018002762A1 (en) * | 2016-06-29 | 2018-01-04 | Crispr Therapeutics Ag | Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders |
| EP3487523B1 (en) | 2016-07-19 | 2023-09-06 | Duke University | Therapeutic applications of cpf1-based genome editing |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CN109804066A (zh) | 2016-08-09 | 2019-05-24 | 哈佛大学的校长及成员们 | 可编程cas9-重组酶融合蛋白及其用途 |
| WO2018031762A1 (en) * | 2016-08-10 | 2018-02-15 | Duke University | Compositions, systems and methods for programming immune cell function through targeted gene regulation |
| WO2018035495A1 (en) | 2016-08-19 | 2018-02-22 | Whitehead Institute For Biomedical Research | Methods of editing dna methylation |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| US20200149039A1 (en) | 2016-12-12 | 2020-05-14 | Whitehead Institute For Biomedical Research | Regulation of transcription through ctcf loop anchors |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| FI3565891T3 (fi) | 2017-01-09 | 2023-07-04 | Whitehead Inst Biomedical Res | Menetelmiä geeniekspression muuttamiseksi häiritsemällä säätelysilmukoita muodostavia transkriptiotekijämultimeerejä |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| US11771703B2 (en) | 2017-03-17 | 2023-10-03 | The Johns Hopkins University | Targeted epigenetic therapy against distal regulatory element of TGFβ2 expression |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| 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 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| US20210017544A1 (en) * | 2017-12-22 | 2021-01-21 | Novozymes A/S | Counter-Selection by Inhibition of Conditionally Essential Genes |
| CN112334577B (zh) | 2018-04-19 | 2023-10-17 | 加利福尼亚大学董事会 | 用于基因编辑的组合物和方法 |
| CN112166194A (zh) * | 2018-04-23 | 2021-01-01 | 杜克大学 | 通过dna甲基化的靶向编辑下调snca的表达 |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020160125A1 (en) * | 2019-01-29 | 2020-08-06 | Flagship Pioneering Innovations V, Inc. | Compositions comprising an endonuclease and methods for purifying an endonuclease |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP4017544A4 (en) * | 2019-08-19 | 2024-04-03 | Duke University | Skeletal myoblast progenitor cell lineage specification by crispr/cas9-based transcriptional activators |
| US20220409744A1 (en) * | 2019-09-23 | 2022-12-29 | Vigeneron Gmbh | Method of transactivating a homologous gene of a gene of interest and an in vitro method of diagnosing a disease |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| JP2023525304A (ja) | 2020-05-08 | 2023-06-15 | ザ ブロード インスティテュート，インコーポレーテッド | 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物 |
| WO2022008557A2 (en) * | 2020-07-08 | 2022-01-13 | UCB Biopharma SRL | Modulation of cftr expression |
| CN112481262B (zh) * | 2020-12-04 | 2022-08-19 | 中国农业科学院农业基因组研究所 | 一种基于CRISPR/Cas9基因编辑技术分析增强子细胞生物学功能的方法 |
| WO2022140577A2 (en) * | 2020-12-22 | 2022-06-30 | Chroma Medicine, Inc. | Compositions and methods for epigenetic editing |
| CA3227105A1 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| US20240254483A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
| JP7707832B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707836B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707824B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707831B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707823B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707825B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707833B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707828B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707826B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707821B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707835B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707822B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707829B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707820B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707834B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| JP7707827B2 (ja) * | 2021-10-06 | 2025-07-15 | 株式会社三洋物産 | 遊技機 |
| EP4463548A1 (en) | 2022-01-14 | 2024-11-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation |
| EP4463549A2 (en) | 2022-01-14 | 2024-11-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression |
| CN114507689B (zh) * | 2022-01-27 | 2024-01-16 | 山东大学 | 一种提高真核生物基因编辑效率的方法及其应用 |
| EP4273242A1 (en) * | 2022-05-03 | 2023-11-08 | ETH Zurich | Crispr-based modification of human hbd gene |
| WO2023250511A2 (en) | 2022-06-24 | 2023-12-28 | Tune Therapeutics, Inc. | Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics Inc | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| CA3264214A1 (en) | 2022-08-19 | 2024-02-22 | Tune Therapeutics Inc | Compositions, systems and methods of regulating the hepatitis B virus by targeted gene suppression |
| CN120239746A (zh) | 2022-09-19 | 2025-07-01 | 图恩疗法股份有限公司 | 用于调节t细胞功能的组合物、系统和方法 |
| WO2024086805A2 (en) * | 2022-10-21 | 2024-04-25 | Watchmaker Genomics, Inc. | Methods and compositions for sequencing library normalization |
| 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) |
| WO2024168154A2 (en) * | 2023-02-09 | 2024-08-15 | William Marsh Rice University | Engineered lysine acyltransferases and methods of use thereof |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2025029835A1 (en) | 2023-07-31 | 2025-02-06 | Tune Therapeutics, Inc. | Compositions and methods for modulating il-2 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 |
| 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 |
| CN118291415B (zh) * | 2024-03-14 | 2024-11-19 | 内蒙古大学 | 一种CRISPR::Cr-P300M巴豆酰化修饰编辑工具及其应用 |
Family Cites Families (428)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US4469863A (en) | 1980-11-12 | 1984-09-04 | Ts O Paul O P | Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof |
| US4554101A (en) | 1981-01-09 | 1985-11-19 | New York Blood Center, Inc. | Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity |
| US5023243A (en) | 1981-10-23 | 1991-06-11 | Molecular Biosystems, Inc. | Oligonucleotide therapeutic agent and method of making same |
| US4476301A (en) | 1982-04-29 | 1984-10-09 | Centre National De La Recherche Scientifique | Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon |
| JPS5927900A (ja) | 1982-08-09 | 1984-02-14 | Wakunaga Seiyaku Kk | 固定化オリゴヌクレオチド |
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| FR2540122B1 (fr) | 1983-01-27 | 1985-11-29 | Centre Nat Rech Scient | Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application |
| US4605735A (en) | 1983-02-14 | 1986-08-12 | Wakunaga Seiyaku Kabushiki Kaisha | Oligonucleotide derivatives |
| US4948882A (en) | 1983-02-22 | 1990-08-14 | Syngene, Inc. | Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis |
| US4824941A (en) | 1983-03-10 | 1989-04-25 | Julian Gordon | Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems |
| US4587044A (en) | 1983-09-01 | 1986-05-06 | The Johns Hopkins University | Linkage of proteins to nucleic acids |
| US5118802A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside |
| US5550111A (en) | 1984-07-11 | 1996-08-27 | Temple University-Of The Commonwealth System Of Higher Education | Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof |
| US5258506A (en) | 1984-10-16 | 1993-11-02 | Chiron Corporation | Photolabile reagents for incorporation into oligonucleotide chains |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| US5430136A (en) | 1984-10-16 | 1995-07-04 | Chiron Corporation | Oligonucleotides having selectably cleavable and/or abasic sites |
| US4828979A (en) | 1984-11-08 | 1989-05-09 | Life Technologies, Inc. | Nucleotide analogs for nucleic acid labeling and detection |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5405938A (en) | 1989-12-20 | 1995-04-11 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5235033A (en) | 1985-03-15 | 1993-08-10 | Anti-Gene Development Group | Alpha-morpholino ribonucleoside derivatives and polymers thereof |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US4762779A (en) | 1985-06-13 | 1988-08-09 | Amgen Inc. | Compositions and methods for functionalizing nucleic acids |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
| US5317098A (en) | 1986-03-17 | 1994-05-31 | Hiroaki Shizuya | Non-radioisotope tagging of fragments |
| JPS638396A (ja) | 1986-06-30 | 1988-01-14 | Wakunaga Pharmaceut Co Ltd | ポリ標識化オリゴヌクレオチド誘導体 |
| EP0260032B1 (en) | 1986-09-08 | 1994-01-26 | Ajinomoto Co., Inc. | Compounds for the cleavage at a specific position of RNA, oligomers employed for the formation of said compounds, and starting materials for the synthesis of said oligomers |
| US5219740A (en) | 1987-02-13 | 1993-06-15 | Fred Hutchinson Cancer Research Center | Retroviral gene transfer into diploid fibroblasts for gene therapy |
| US5264423A (en) | 1987-03-25 | 1993-11-23 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
| US5276019A (en) | 1987-03-25 | 1994-01-04 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
| US4904582A (en) | 1987-06-11 | 1990-02-27 | Synthetic Genetics | Novel amphiphilic nucleic acid conjugates |
| DE3851889T2 (de) | 1987-06-24 | 1995-04-13 | Florey Howard Inst | Nukleosid-derivate. |
| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5188897A (en) | 1987-10-22 | 1993-02-23 | Temple University Of The Commonwealth System Of Higher Education | Encapsulated 2',5'-phosphorothioate oligoadenylates |
| US4924624A (en) | 1987-10-22 | 1990-05-15 | Temple University-Of The Commonwealth System Of Higher Education | 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof |
| US5525465A (en) | 1987-10-28 | 1996-06-11 | Howard Florey Institute Of Experimental Physiology And Medicine | Oligonucleotide-polyamide conjugates and methods of production and applications of the same |
| DE3738460A1 (de) | 1987-11-12 | 1989-05-24 | Max Planck Gesellschaft | Modifizierte oligonukleotide |
| JP3019994B2 (ja) | 1987-11-30 | 2000-03-15 | ユニバーシティ オブ アイオワ リサーチ ファウンデーション | 新規なオリゴデオキシヌクレオチド、それを使用した標的遺伝子の発現をブロックする方法、及び新規なオリゴデオキシヌクレオチド並びにそれを使用した標的遺伝子の発現を阻止する方法 |
| US5403711A (en) | 1987-11-30 | 1995-04-04 | University Of Iowa Research Foundation | Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved |
| US5082830A (en) | 1988-02-26 | 1992-01-21 | Enzo Biochem, Inc. | End labeled nucleotide probe |
| WO1989009221A1 (en) | 1988-03-25 | 1989-10-05 | University Of Virginia Alumni Patents Foundation | Oligonucleotide n-alkylphosphoramidates |
| US5278302A (en) | 1988-05-26 | 1994-01-11 | University Patents, Inc. | Polynucleotide phosphorodithioates |
| US5109124A (en) | 1988-06-01 | 1992-04-28 | Biogen, Inc. | Nucleic acid probe linked to a label having a terminal cysteine |
| US5216141A (en) | 1988-06-06 | 1993-06-01 | Benner Steven A | Oligonucleotide analogs containing sulfur linkages |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5262536A (en) | 1988-09-15 | 1993-11-16 | E. I. Du Pont De Nemours And Company | Reagents for the preparation of 5'-tagged oligonucleotides |
| US5512439A (en) | 1988-11-21 | 1996-04-30 | Dynal As | Oligonucleotide-linked magnetic particles and uses thereof |
| US5599923A (en) | 1989-03-06 | 1997-02-04 | Board Of Regents, University Of Tx | Texaphyrin metal complexes having improved functionalization |
| US5457183A (en) | 1989-03-06 | 1995-10-10 | Board Of Regents, The University Of Texas System | Hydroxylated texaphyrins |
| US5391723A (en) | 1989-05-31 | 1995-02-21 | Neorx Corporation | Oligonucleotide conjugates |
| US5256775A (en) | 1989-06-05 | 1993-10-26 | Gilead Sciences, Inc. | Exonuclease-resistant oligonucleotides |
| US4958013A (en) | 1989-06-06 | 1990-09-18 | Northwestern University | Cholesteryl modified oligonucleotides |
| US5451463A (en) | 1989-08-28 | 1995-09-19 | Clontech Laboratories, Inc. | Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US5254469A (en) | 1989-09-12 | 1993-10-19 | Eastman Kodak Company | Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures |
| US5399676A (en) | 1989-10-23 | 1995-03-21 | Gilead Sciences | Oligonucleotides with inverted polarity |
| US5264564A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences | Oligonucleotide analogs with novel linkages |
| US5264562A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences, Inc. | Oligonucleotide analogs with novel linkages |
| US5292873A (en) | 1989-11-29 | 1994-03-08 | The Research Foundation Of State University Of New York | Nucleic acids labeled with naphthoquinone probe |
| US5177198A (en) | 1989-11-30 | 1993-01-05 | University Of N.C. At Chapel Hill | Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates |
| US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
| US5486603A (en) | 1990-01-08 | 1996-01-23 | Gilead Sciences, Inc. | Oligonucleotide having enhanced binding affinity |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5623065A (en) | 1990-08-13 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Gapped 2' modified oligonucleotides |
| US5578718A (en) | 1990-01-11 | 1996-11-26 | Isis Pharmaceuticals, Inc. | Thiol-derivatized nucleosides |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
| US5220007A (en) | 1990-02-15 | 1993-06-15 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of RNA and production of encoded polypeptides |
| US5214136A (en) | 1990-02-20 | 1993-05-25 | Gilead Sciences, Inc. | Anthraquinone-derivatives oligonucleotides |
| AU7579991A (en) | 1990-02-20 | 1991-09-18 | Gilead Sciences, Inc. | Pseudonucleosides and pseudonucleotides and their polymers |
| US5321131A (en) | 1990-03-08 | 1994-06-14 | Hybridon, Inc. | Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling |
| US5470967A (en) | 1990-04-10 | 1995-11-28 | The Dupont Merck Pharmaceutical Company | Oligonucleotide analogs with sulfamate linkages |
| EP0455905B1 (en) | 1990-05-11 | 1998-06-17 | Microprobe Corporation | Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides |
| GB9011454D0 (en) | 1990-05-22 | 1990-07-11 | Medical Res Council | Polynucleotide amplification |
| WO1992000387A1 (en) | 1990-06-29 | 1992-01-09 | The Regents Of The University Of Michigan | Neurofibromatosis gene |
| US5688941A (en) | 1990-07-27 | 1997-11-18 | Isis Pharmaceuticals, Inc. | Methods of making conjugated 4' desmethyl nucleoside analog compounds |
| CA2088258C (en) | 1990-07-27 | 2004-09-14 | Phillip Dan Cook | Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression |
| US5489677A (en) | 1990-07-27 | 1996-02-06 | Isis Pharmaceuticals, Inc. | Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms |
| US5623070A (en) | 1990-07-27 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| US5541307A (en) | 1990-07-27 | 1996-07-30 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs and solid phase synthesis thereof |
| US5218105A (en) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5618704A (en) | 1990-07-27 | 1997-04-08 | Isis Pharmacueticals, Inc. | Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5138045A (en) | 1990-07-27 | 1992-08-11 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5677437A (en) | 1990-07-27 | 1997-10-14 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
| US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| US5245022A (en) | 1990-08-03 | 1993-09-14 | Sterling Drug, Inc. | Exonuclease resistant terminally substituted oligonucleotides |
| KR100211552B1 (ko) | 1990-08-03 | 1999-08-02 | 디. 꼬쉬 | 유전자 발현 억제용 화합물 및 방법 |
| US5177196A (en) | 1990-08-16 | 1993-01-05 | Microprobe Corporation | Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof |
| US5512667A (en) | 1990-08-28 | 1996-04-30 | Reed; Michael W. | Trifunctional intermediates for preparing 3'-tailed oligonucleotides |
| US5214134A (en) | 1990-09-12 | 1993-05-25 | Sterling Winthrop Inc. | Process of linking nucleosides with a siloxane bridge |
| US5561225A (en) | 1990-09-19 | 1996-10-01 | Southern Research Institute | Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages |
| JPH06505704A (ja) | 1990-09-20 | 1994-06-30 | ギリアド サイエンシズ，インコーポレイテッド | 改変ヌクレオシド間結合 |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| KR930702373A (ko) | 1990-11-08 | 1993-09-08 | 안토니 제이. 페이네 | 합성 올리고누클레오티드에 대한 다중 리포터(Reporter)그룹의 첨합 |
| US5510473A (en) | 1990-11-09 | 1996-04-23 | The United States Of American As Represented By The Secretary Of Health And Human Services | Cloning of the recA gene from thermus aquaticus YT-1 |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| US5371241A (en) | 1991-07-19 | 1994-12-06 | Pharmacia P-L Biochemicals Inc. | Fluorescein labelled phosphoramidites |
| US5571799A (en) | 1991-08-12 | 1996-11-05 | Basco, Ltd. | (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response |
| ATE239484T1 (de) | 1991-10-24 | 2003-05-15 | Isis Pharmaceuticals Inc | Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| US5700922A (en) | 1991-12-24 | 1997-12-23 | Isis Pharmaceuticals, Inc. | PNA-DNA-PNA chimeric macromolecules |
| US5595726A (en) | 1992-01-21 | 1997-01-21 | Pharmacyclics, Inc. | Chromophore probe for detection of nucleic acid |
| US5565552A (en) | 1992-01-21 | 1996-10-15 | Pharmacyclics, Inc. | Method of expanded porphyrin-oligonucleotide conjugate synthesis |
| GB9206016D0 (en) | 1992-03-19 | 1992-04-29 | Sandoz Ltd | Improvements in or relating to organic compounds |
| US5633360A (en) | 1992-04-14 | 1997-05-27 | Gilead Sciences, Inc. | Oligonucleotide analogs capable of passive cell membrane permeation |
| US5434257A (en) | 1992-06-01 | 1995-07-18 | Gilead Sciences, Inc. | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| AU4528493A (en) | 1992-06-04 | 1994-01-04 | Regents Of The University Of California, The | In vivo gene therapy with intron-free sequence of interest |
| US5272250A (en) | 1992-07-10 | 1993-12-21 | Spielvogel Bernard F | Boronated phosphoramidate compounds |
| US5652355A (en) | 1992-07-23 | 1997-07-29 | Worcester Foundation For Experimental Biology | Hybrid oligonucleotide phosphorothioates |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5574142A (en) | 1992-12-15 | 1996-11-12 | Microprobe Corporation | Peptide linkers for improved oligonucleotide delivery |
| PT681483E (pt) | 1993-01-26 | 2005-11-30 | Wyeth Corp | Composicoes e metodos para distribuicao de material genetico |
| US5593972A (en) | 1993-01-26 | 1997-01-14 | The Wistar Institute | Genetic immunization |
| US5476925A (en) | 1993-02-01 | 1995-12-19 | Northwestern University | Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups |
| GB9304618D0 (en) | 1993-03-06 | 1993-04-21 | Ciba Geigy Ag | Chemical compounds |
| WO1994022891A1 (en) | 1993-03-31 | 1994-10-13 | Sterling Winthrop Inc. | Oligonucleotides with amide linkages replacing phosphodiester linkages |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| KR100386337B1 (ko) | 1993-12-09 | 2004-03-24 | 토마스 제퍼슨 대학교 | 진핵세포에서부위-특이적돌연변이를위한화합물과그방법 |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5625050A (en) | 1994-03-31 | 1997-04-29 | Amgen Inc. | Modified oligonucleotides and intermediates useful in nucleic acid therapeutics |
| US5658784A (en) * | 1994-04-14 | 1997-08-19 | Dana-Farber Cancer Institute, Inc. | Nucleic acid encoding transcription factor p300 and uses of p300 |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5597696A (en) | 1994-07-18 | 1997-01-28 | Becton Dickinson And Company | Covalent cyanine dye oligonucleotide conjugates |
| US5580731A (en) | 1994-08-25 | 1996-12-03 | Chiron Corporation | N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith |
| US5962428A (en) | 1995-03-30 | 1999-10-05 | Apollon, Inc. | Compositions and methods for delivery of genetic material |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US5652356A (en) | 1995-08-17 | 1997-07-29 | Hybridon, Inc. | Inverted chimeric and hybrid oligonucleotides |
| DE19608753C1 (de) | 1996-03-06 | 1997-06-26 | Medigene Gmbh | Transduktionssystem und seine Verwendung |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| CA2263784A1 (en) | 1998-03-23 | 1999-09-23 | Megabios Corporation | Dual-tagged proteins and their uses |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| US6734291B2 (en) | 1999-03-24 | 2004-05-11 | Exiqon A/S | Synthesis of [2.2.1]bicyclo nucleosides |
| PT1178999E (pt) | 1999-05-04 | 2007-06-26 | Santaris Pharma As | Análogos de l-ribo-lna |
| US20040192593A1 (en) | 1999-07-26 | 2004-09-30 | Baylor College Of Medicine | Protease resistant ti-growth hormone releasing hormone |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| US6287860B1 (en) | 2000-01-20 | 2001-09-11 | Isis Pharmaceuticals, Inc. | Antisense inhibition of MEKK2 expression |
| AU5391401A (en) | 2000-04-28 | 2001-11-12 | Sangamo Biosciences Inc | Targeted modification of chromatin structure |
| WO2001083783A2 (en) | 2000-04-28 | 2001-11-08 | Genzyme Corporation | In vivo loading of mhc |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| US7879540B1 (en) | 2000-08-24 | 2011-02-01 | Promega Corporation | Synthetic nucleic acid molecule compositions and methods of preparation |
| JP2005500061A (ja) | 2001-08-20 | 2005-01-06 | ザ スクリップス リサーチ インスティテュート | Ｃｎｎについての亜鉛フィンガー結合ドメイン |
| WO2003020739A2 (en) | 2001-09-04 | 2003-03-13 | Exiqon A/S | Novel lna compositions and uses thereof |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| AU2003219847A1 (en) | 2002-02-21 | 2003-09-09 | The Wistar Institute Of Anatomy And Biology | Methods and compositions for reversibly controlling expression of target genes in cells |
| US7449561B1 (en) | 2002-02-26 | 2008-11-11 | City Of Hope | Alterations in the dystrophin gene associated with sporadic dilated cardiomyopathy |
| US7074596B2 (en) | 2002-03-25 | 2006-07-11 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse mRNA cap analogues |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US7901708B2 (en) | 2002-06-28 | 2011-03-08 | Protiva Biotherapeutics, Inc. | Liposomal apparatus and manufacturing methods |
| US20040175727A1 (en) | 2002-11-04 | 2004-09-09 | Advisys, Inc. | Synthetic muscle promoters with activities exceeding naturally occurring regulatory sequences in cardiac cells |
| US20070185042A1 (en) | 2003-08-08 | 2007-08-09 | The Brigham And Women's Hospital, Inc. President And Fellows Of Harvard College | Sirna based methods for treating alzheimer's disease |
| US7407776B2 (en) | 2003-09-19 | 2008-08-05 | Sangamo Biosciences, Inc. | Engineered zinc finger proteins for regulation of gene expression |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US20070192880A1 (en) | 2003-10-03 | 2007-08-16 | University Of Rochester | Horming response element binding transregulators |
| US8507277B2 (en) | 2003-10-24 | 2013-08-13 | Gencia Corporation | Nonviral vectors for delivering polynucleotides |
| JP4764426B2 (ja) | 2004-06-07 | 2011-09-07 | プロチバ バイオセラピューティクス インコーポレイティッド | カチオン性脂質および使用方法 |
| AU2005252273B2 (en) | 2004-06-07 | 2011-04-28 | Arbutus Biopharma Corporation | Lipid encapsulated interfering RNA |
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| US7728118B2 (en) | 2004-09-17 | 2010-06-01 | Promega Corporation | Synthetic nucleic acid molecule compositions and methods of preparation |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| JP5639338B2 (ja) | 2005-07-27 | 2014-12-10 | プロチバ バイオセラピューティクス インコーポレイティッド | リポソームの製造システムおよび製造方法 |
| BRPI0614716A2 (pt) | 2005-08-05 | 2011-04-12 | Univ Michigan State | método para produção de um produto quìmico a partir da via c4 de a. succinogenes |
| US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| HUE043492T2 (hu) | 2005-08-23 | 2019-08-28 | Univ Pennsylvania | Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| KR20090031938A (ko) | 2006-07-05 | 2009-03-30 | 더 스크립스 리서치 인스티튜트 | 방향성 진화로 촉매 작용을 최적화시킨 키메라 징크 핑거 리컴비나제 |
| EP2097538A4 (en) | 2006-12-07 | 2011-11-30 | Switchgear Genomics | TRANSCRIPTION REAGULATION ELEMENTS OF BIOLOGICAL PATHS, TOOLS AND METHODS |
| US8198244B2 (en) | 2006-12-07 | 2012-06-12 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Regulation of skin characteristics by DICKKOPF1 (DKK1) |
| WO2008086807A2 (en) | 2007-01-19 | 2008-07-24 | Exiqon A/S | Mediated cellular delivery of lna oligonucleotides |
| US20100261175A1 (en) | 2007-06-15 | 2010-10-14 | Exiqon A/S | Use of short oligonucleotides for reagent redundancy experiments in rna functional analysis |
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| CA3065577C (en) | 2008-05-13 | 2022-05-31 | Phaserx, Inc. | Diblock copolymers and polynucleotide complexes thereof for delivery into cells |
| US20100076057A1 (en) | 2008-09-23 | 2010-03-25 | Northwestern University | TARGET DNA INTERFERENCE WITH crRNA |
| WO2010042877A1 (en) | 2008-10-09 | 2010-04-15 | Tekmira Pharmaceuticals Corporation | Improved amino lipids and methods for the delivery of nucleic acids |
| EP2998402A1 (en) | 2008-10-17 | 2016-03-23 | Joule Unlimited Technologies, Inc. | Ethanol production by microorganisms |
| TR201902952T4 (tr) | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
| JP5766611B2 (ja) | 2008-11-06 | 2015-08-19 | ユニヴァーシティ オブ ワシントン | マルチブロック共重合体 |
| MX353900B (es) | 2008-11-07 | 2018-02-01 | Massachusetts Inst Technology | Lipidoides de aminoalcohol y usos de los mismos. |
| WO2010075424A2 (en) | 2008-12-22 | 2010-07-01 | The Regents Of University Of California | Compositions and methods for downregulating prokaryotic genes |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| WO2010108126A2 (en) | 2009-03-19 | 2010-09-23 | Fate Therapeutics, Inc. | Reprogramming compositions and methods of using the same |
| KR20230098713A (ko) | 2009-06-10 | 2023-07-04 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| EP2281579A1 (en) | 2009-08-05 | 2011-02-09 | BioNTech AG | Vaccine composition comprising 5'-Cap modified RNA |
| WO2011036640A2 (en) | 2009-09-24 | 2011-03-31 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
| AU2010312240B2 (en) | 2009-10-31 | 2016-12-15 | Genesis Technologies Limited | Methods for reprogramming cells and uses thereof |
| CA2783351C (en) | 2009-12-10 | 2021-09-07 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| BR112012020257A8 (pt) | 2010-02-11 | 2018-02-14 | Recombinetics Inc | métodos e aparelhos para produzir artiodátilos transgênicos |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| SG10201502270TA (en) | 2010-03-29 | 2015-05-28 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130145487A1 (en) | 2010-05-12 | 2013-06-06 | Cellectis | Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof |
| CA2798988C (en) | 2010-05-17 | 2020-03-10 | Sangamo Biosciences, Inc. | Tal-effector (tale) dna-binding polypeptides and uses thereof |
| IT1400425B1 (it) | 2010-06-08 | 2013-05-31 | Amsterdam Molecular Therapeutics Bv | Modified snrnas for use in therapy. |
| EP2517731A1 (en) | 2011-04-07 | 2012-10-31 | Ludwig-Maximilians-Universität München | Method of activating a target gene in a cell |
| CN103534417B (zh) | 2011-05-12 | 2016-03-23 | 日立建机株式会社 | 工程机械 |
| US20140206753A1 (en) | 2011-06-08 | 2014-07-24 | Shire Human Genetic Therapies, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| EP2729567B1 (en) | 2011-07-08 | 2016-10-05 | Cellectis | Method for increasing the efficiency of double-strand break-induced mutagenssis |
| HK1200871A1 (en) | 2011-11-16 | 2015-08-14 | Sangamo Therapeutics, Inc. | Modified dna-binding proteins and uses thereof |
| US8450107B1 (en) | 2011-11-30 | 2013-05-28 | The Broad Institute Inc. | Nucleotide-specific recognition sequences for designer TAL effectors |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| EP2834357B1 (en) | 2012-04-04 | 2017-12-27 | Life Technologies Corporation | Tal-effector assembly platform, customized services, kits and assays |
| US9738879B2 (en) | 2012-04-27 | 2017-08-22 | Duke University | Genetic correction of mutated genes |
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| FI3597749T3 (fi) | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| US9890364B2 (en) | 2012-05-29 | 2018-02-13 | The General Hospital Corporation | TAL-Tet1 fusion proteins and methods of use thereof |
| WO2013182683A1 (en) | 2012-06-08 | 2013-12-12 | Ethris Gmbh | Pulmonary delivery of messenger rna |
| EP2877213B1 (en) | 2012-07-25 | 2020-12-02 | The Broad Institute, Inc. | Inducible dna binding proteins and genome perturbation tools and applications thereof |
| US9567581B2 (en) | 2012-08-07 | 2017-02-14 | The General Hospital Corporation | Selective reactivation of genes on the inactive X chromosome |
| EP2906602B1 (en) | 2012-10-12 | 2019-01-16 | The General Hospital Corporation | Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins |
| KR101656236B1 (ko) | 2012-10-23 | 2016-09-12 | 주식회사 툴젠 | 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도 |
| BR122019025681B1 (pt) | 2012-11-01 | 2023-04-18 | Factor Bioscience Inc | Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula |
| WO2014081855A1 (en) | 2012-11-20 | 2014-05-30 | Universite De Montreal | Methods and compositions for muscular dystrophies |
| PT3363902T (pt) | 2012-12-06 | 2019-12-19 | Sigma Aldrich Co Llc | Modificação e regulação de genoma baseadas em crispr |
| WO2014093479A1 (en) | 2012-12-11 | 2014-06-19 | Montana State University | Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| KR20150105956A (ko) | 2012-12-12 | 2015-09-18 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화 |
| PT2784162E (pt) | 2012-12-12 | 2015-08-27 | Broad Inst Inc | Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| EP2931898B1 (en) | 2012-12-12 | 2016-03-09 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| EP4282970A3 (en) | 2012-12-17 | 2024-01-17 | President and Fellows of Harvard College | Rna-guided human genome engineering |
| NZ732507A (en) | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| KR102874079B1 (ko) * | 2013-03-15 | 2025-10-22 | 더 제너럴 하스피탈 코포레이션 | Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용 |
| EP2973735B1 (en) | 2013-03-15 | 2020-02-12 | Edward J. Britt | Energy conversion device and method for making and using same |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| WO2014172470A2 (en) * | 2013-04-16 | 2014-10-23 | Whitehead Institute For Biomedical Research | Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal |
| AU2014265331B2 (en) | 2013-05-15 | 2019-12-05 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| DK3004337T3 (da) | 2013-05-29 | 2017-11-13 | Cellectis | Fremgangsmåde til konstruktion af T-celler til immunoterapi ved brug af RNA-guidet Cas nuklease-system |
| RU2690935C2 (ru) | 2013-06-04 | 2019-06-06 | Президент Энд Фэллоуз Оф Харвард Коллидж | Направляемая рнк регуляция транскрипции |
| US20140356956A1 (en) | 2013-06-04 | 2014-12-04 | President And Fellows Of Harvard College | RNA-Guided Transcriptional Regulation |
| JP7085716B2 (ja) | 2013-06-05 | 2022-06-17 | デューク ユニバーシティ | Ｒｎａガイド遺伝子編集及び遺伝子調節 |
| JP6738729B2 (ja) | 2013-06-17 | 2020-08-12 | ザ・ブロード・インスティテュート・インコーポレイテッド | 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化 |
| EP3011035B1 (en) | 2013-06-17 | 2020-05-13 | The Broad Institute, Inc. | Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences |
| CN107995927B (zh) | 2013-06-17 | 2021-07-30 | 布罗德研究所有限公司 | 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途 |
| US10011850B2 (en) * | 2013-06-21 | 2018-07-03 | The General Hospital Corporation | Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing |
| DK3019619T3 (da) | 2013-07-11 | 2021-10-11 | Modernatx Inc | Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder |
| US10421957B2 (en) | 2013-07-29 | 2019-09-24 | Agilent Technologies, Inc. | DNA assembly using an RNA-programmable nickase |
| WO2015017519A1 (en) | 2013-07-30 | 2015-02-05 | Phaserx, Inc. | Block copolymers and their conjugates or complexes with oligonucleotides |
| 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 |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| US10265347B2 (en) | 2013-08-29 | 2019-04-23 | Norimasa Miura | Biomolecular group related to cell anti-aging |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US10822606B2 (en) | 2013-09-27 | 2020-11-03 | The Regents Of The University Of California | Optimized small guide RNAs and methods of use |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| BR112016013201B1 (pt) | 2013-12-12 | 2023-01-31 | The Broad Institute, Inc. | Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular |
| EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| SG10201804973TA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
| EP3079726B1 (en) | 2013-12-12 | 2018-12-05 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| JP2017503485A (ja) | 2013-12-12 | 2017-02-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 遺伝子産物の発現、構造情報、及び誘導性モジュラーｃａｓ酵素を変更するためのｃｒｉｓｐｒ−ｃａｓ系並びに方法 |
| EP3540051B1 (en) | 2013-12-12 | 2022-08-17 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders. |
| WO2015114059A1 (en) | 2014-01-29 | 2015-08-06 | Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) | Trans-differentiation of differentiated cells |
| KR20160130392A (ko) | 2014-02-18 | 2016-11-11 | 듀크 유니버시티 | 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법 |
| EP3553176A1 (en) | 2014-03-10 | 2019-10-16 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| CN106460003A (zh) | 2014-04-08 | 2017-02-22 | 北卡罗来纳州立大学 | 用于使用crispr相关基因rna引导阻遏转录的方法和组合物 |
| WO2015161276A2 (en) | 2014-04-18 | 2015-10-22 | Editas Medicine, Inc. | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
| SG10202002486QA (en) | 2014-06-16 | 2020-04-29 | Univ Johns Hopkins | Compositions and methods for the expression of crispr guide rnas using the h1 promoter |
| EP3169702A4 (en) | 2014-07-14 | 2018-04-18 | The Regents of The University of California | A protein tagging system for in vivo single molecule imaging and control of gene transcription |
| US11254933B2 (en) | 2014-07-14 | 2022-02-22 | The Regents Of The University Of California | CRISPR/Cas transcriptional modulation |
| US9932566B2 (en) | 2014-08-07 | 2018-04-03 | Agilent Technologies, Inc. | CIS-blocked guide RNA |
| EP3180034B1 (en) | 2014-08-11 | 2022-04-20 | The Board of Regents of The University of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
| WO2016049258A2 (en) | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| GB201418965D0 (enExample) | 2014-10-24 | 2014-12-10 | Ospedale San Raffaele And Fond Telethon | |
| MA40880A (fr) | 2014-10-30 | 2017-09-05 | Temple Univ Of The Commonwealth | Éradication guidée par l'arn du virus jc humain et d'autres polyomavirus |
| WO2016081924A1 (en) | 2014-11-20 | 2016-05-26 | Duke University | Compositions, systems and methods for cell therapy |
| CA3176380A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
| US10190106B2 (en) * | 2014-12-22 | 2019-01-29 | Univesity Of Massachusetts | Cas9-DNA targeting unit chimeras |
| WO2016114972A1 (en) | 2015-01-12 | 2016-07-21 | The Regents Of The University Of California | Heterodimeric cas9 and methods of use thereof |
| ES2880473T5 (es) | 2015-01-30 | 2024-05-09 | Univ California | Suministro de proteínas en células hematopoyéticas primarias |
| EP3256487A4 (en) | 2015-02-09 | 2018-07-18 | Duke University | Compositions and methods for epigenome editing |
| KR102598856B1 (ko) | 2015-03-03 | 2023-11-07 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| US20160281166A1 (en) | 2015-03-23 | 2016-09-29 | Parabase Genomics, Inc. | Methods and systems for screening diseases in subjects |
| EP3748004A1 (en) | 2015-04-01 | 2020-12-09 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
| JP2018512876A (ja) | 2015-04-22 | 2018-05-24 | ミナ セラピューティクス リミテッド | ｓａＲＮＡ組成物および使用方法 |
| JP2018522249A (ja) | 2015-04-24 | 2018-08-09 | エディタス・メディシン、インコーポレイテッド | Ｃａｓ９分子／ガイドｒｎａ分子複合体の評価 |
| WO2016187717A1 (en) | 2015-05-26 | 2016-12-01 | Exerkine Corporation | Exosomes useful for genome editing |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| CN109536474A (zh) | 2015-06-18 | 2019-03-29 | 布罗德研究所有限公司 | 降低脱靶效应的crispr酶突变 |
| WO2017015637A1 (en) | 2015-07-22 | 2017-01-26 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
| BR112018001400B1 (pt) | 2015-07-24 | 2021-11-09 | Basf Se | Compostos, composição, uso do composto, método para o combate de fungos fitopatogênicos e semente |
| US20200123533A1 (en) | 2015-07-31 | 2020-04-23 | The Trustees Of Columbia University In The City Of New York | High-throughput strategy for dissecting mammalian genetic interactions |
| JP6905755B2 (ja) | 2015-08-25 | 2021-07-21 | デューク ユニバーシティ | Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法 |
| US11091780B2 (en) | 2015-09-18 | 2021-08-17 | The Regents Of The University Of California | Methods for autocatalytic genome editing and neutralizing autocatalytic genome editing and compositions thereof |
| WO2017049407A1 (en) | 2015-09-23 | 2017-03-30 | UNIVERSITé LAVAL | Modification of the dystrophin gene and uses thereof |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| ES3040945T3 (en) | 2015-10-28 | 2025-11-06 | Vertex Pharma | Materials and methods for treatment of duchenne muscular dystrophy |
| EP3368689B1 (en) | 2015-10-28 | 2020-06-17 | The Broad Institute, Inc. | Composition for modulating immune responses by use of immune cell gene signature |
| EP3374501B1 (en) | 2015-11-11 | 2023-07-12 | Lonza Ltd | Crispr-associated (cas) proteins with reduced immunogenicity |
| US11306308B2 (en) | 2015-11-13 | 2022-04-19 | Massachusetts Institute Of Technology | High-throughput CRISPR-based library screening |
| MX2018005377A (es) | 2015-11-30 | 2018-11-09 | Univ Duke | Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso. |
| NZ743983A (en) | 2016-01-11 | 2025-08-29 | Univ Leland Stanford Junior | Chimeric proteins and methods of regulating gene expression |
| EP3199632A1 (en) | 2016-01-26 | 2017-08-02 | ACIB GmbH | Temperature-inducible crispr/cas system |
| JP6998313B2 (ja) | 2016-02-11 | 2022-02-04 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 細胞のゲノムにおける変異ジストロフィン遺伝子を修飾する方法及び組成物 |
| WO2017165859A1 (en) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| WO2017180915A2 (en) | 2016-04-13 | 2017-10-19 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| HUE066442T2 (hu) | 2016-04-15 | 2024-08-28 | Res Institute At Nationwide | ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése |
| CN109661470A (zh) | 2016-04-15 | 2019-04-19 | 宾夕法尼亚州大学信托人 | 新型aav8突变衣壳和含有其的组合物 |
| ES2957660T3 (es) | 2016-05-05 | 2024-01-23 | Univ Duke | Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne |
| US11278550B2 (en) | 2016-05-17 | 2022-03-22 | Duke University | Compositions and methods for the treatment of Prader-Willi syndrome |
| US20170362635A1 (en) | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
| US11427838B2 (en) | 2016-06-29 | 2022-08-30 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders |
| KR20190024977A (ko) | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| MX2019000262A (es) | 2016-07-05 | 2019-05-27 | Univ Johns Hopkins | Composiciones basadas en crispr/cas9 y metodos para el tratamiento de degeneraciones retinianas. |
| US11674158B2 (en) | 2016-07-15 | 2023-06-13 | Salk Institute For Biological Studies | Methods and compositions for genome editing in non-dividing cells |
| US11566243B2 (en) | 2016-07-18 | 2023-01-31 | Jaan Biotherapeutics Llc | Compositions and methods for treatment of cardiac diseases |
| US20190235624A1 (en) | 2016-07-19 | 2019-08-01 | Supereye, Inc. | Systems and methods for predictive visual rendering |
| EP3487523B1 (en) | 2016-07-19 | 2023-09-06 | Duke University | Therapeutic applications of cpf1-based genome editing |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| WO2018031762A1 (en) | 2016-08-10 | 2018-02-15 | Duke University | Compositions, systems and methods for programming immune cell function through targeted gene regulation |
| US11352647B2 (en) | 2016-08-17 | 2022-06-07 | The Broad Institute, Inc. | Crispr enzymes and systems |
| WO2018035495A1 (en) | 2016-08-19 | 2018-02-22 | Whitehead Institute For Biomedical Research | Methods of editing dna methylation |
| AU2017313616B2 (en) | 2016-08-19 | 2022-12-08 | Institute For Basic Science | Artificially engineered angiogenesis regulatory system |
| US20190185850A1 (en) | 2016-08-20 | 2019-06-20 | Avellino Lab Usa, Inc. | Single guide rna/crispr/cas9 systems, and methods of use thereof |
| WO2018053632A1 (en) | 2016-09-23 | 2018-03-29 | UNIVERSITé LAVAL | Methods of modifying the dystrophin gene and restoring dystrophin expression and uses thereof |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| WO2018081504A1 (en) | 2016-10-28 | 2018-05-03 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus |
| CA3044531A1 (en) | 2016-11-28 | 2018-05-31 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing |
| US20200056206A1 (en) | 2016-12-01 | 2020-02-20 | UNIVERSITé LAVAL | Crispr-based treatment of friedreich ataxia |
| AU2017370730A1 (en) | 2016-12-08 | 2019-06-27 | The Board Of Regents Of The University Of Texas System | DMD reporter models containing humanized duschene muscular dystrophy mutations |
| JOP20190166A1 (ar) | 2017-01-05 | 2019-07-02 | Univ Texas | استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي |
| AU2018218280B2 (en) | 2017-02-07 | 2024-10-17 | The Regents Of The University Of California | Gene therapy for haploinsufficiency |
| JP2018143239A (ja) | 2017-03-01 | 2018-09-20 | エリクサジェン，エルエルシー． | 多能性幹細胞を所望の細胞型へ効率的に分化する方法 |
| EP3589330A4 (en) | 2017-03-03 | 2021-01-06 | Flagship Pioneering Innovations V, Inc. | DNA MODIFICATION METHODS AND SYSTEMS |
| US10687520B2 (en) | 2017-03-07 | 2020-06-23 | The Board Of Regents Of The University Of Texas System | Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44 |
| WO2018162702A1 (en) | 2017-03-10 | 2018-09-13 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Nuclease fusions for enhancing genome editing by homology-directed transgene integration |
| US11541130B2 (en) | 2017-03-23 | 2023-01-03 | DNARx | Systems and methods for nucleic acid expression in vivo |
| KR102758434B1 (ko) | 2017-03-30 | 2025-01-21 | 고쿠리츠 다이가쿠 호진 교토 다이가쿠 | 게놈 편집에 의한 엑손 스키핑 유도 방법 |
| CN110799645B (zh) | 2017-04-12 | 2024-08-02 | 博德研究所 | 新型vi型crispr直系同源物和系统 |
| US20180305719A1 (en) | 2017-04-19 | 2018-10-25 | The Board Of Trustees Of The University Of Illinois | Vectors For Integration Of DNA Into Genomes And Methods For Altering Gene Expression And Interrogating Gene Function |
| JP7398279B2 (ja) | 2017-05-10 | 2023-12-14 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | Ｃｒｉｓｐｒ／ｃａｓ９核送達による細胞ｒｎａの狙いを定めた編集 |
| EP3420811A1 (en) | 2017-06-29 | 2019-01-02 | Paris Sciences et Lettres - Quartier Latin | Non-human model for neurofibromatosis type 1 |
| EP3650540B1 (en) | 2017-07-07 | 2025-04-16 | Toolgen Incorporated | Target-specific crispr mutant |
| US11279765B2 (en) | 2017-07-08 | 2022-03-22 | The General Hospital Corporation | Compositions and methods to improve anti-angiogenic therapy and immunotherapy |
| WO2019023291A2 (en) | 2017-07-25 | 2019-01-31 | Dana-Farber Cancer Institute, Inc. | COMPOSITIONS AND METHODS FOR PRODUCTION AND DECODING OF GUIDE RNA LIBRARIES AND USES THEREOF |
| CA3106738C (en) | 2017-07-25 | 2025-02-11 | Shanghai Inst Biological Sciences Cas | METHOD FOR MODULATING RNA SPLICING BY INDUCING A BASE MUTATION AT A SPLICE SITE OR A BASE SUBSTITUTION IN A POLYPYRIMIDIN REGION |
| EP3441461A1 (en) | 2017-08-11 | 2019-02-13 | Baylor College of Medicine | Cd1d-restricted nkt cells as a platform for off-the-shelf cancer immunotherapy |
| WO2019036599A1 (en) | 2017-08-18 | 2019-02-21 | The Board Of Regents Of The University Of Texas System | EXON DELETION CORRECTION OF MUTATIONS OF DUCHENNE MUSCLE DYSTROPHY IN ACTINE DYSTROPHINE BINDING DOMAIN 1 Using a GENOME CRISPR EDITION |
| TW201919655A (zh) | 2017-08-31 | 2019-06-01 | 美商薩羅塔治療公司 | 治療肌肉萎縮症的方法 |
| EP3687548A4 (en) | 2017-09-29 | 2021-05-26 | The Regents of the University of California | MULTI-ARM POLYROTAXANE PLATFORM FOR PROTECTED NUCLEIC ACID ADMINISTRATION |
| US11987790B2 (en) | 2017-10-17 | 2024-05-21 | Massachusetts Institute Of Technology | Methods for high-resolution genome-wide functional dissection of transcriptional regulatory regions |
| IL321714A (en) | 2017-10-19 | 2025-08-01 | CureVac SE | Novel artificial nucleic acid molecules |
| HRP20250322T1 (hr) | 2017-10-23 | 2025-06-06 | Stoke Therapeutics, Inc. | Protusmisleni oligomeri, namijenjeni liječenju stanja i bolesti uzrokovanih raspadanjem rna posredovanim besmislenim mutacijama |
| WO2019092507A2 (en) | 2017-11-09 | 2019-05-16 | Crispr Therapeutics Ag | Crispr/cas systems for treatment of dmd |
| CA3084810A1 (en) | 2017-12-08 | 2019-06-13 | University Of Connecticut | Compositions and methods for treating disorders of genomic imprinting |
| WO2019118902A2 (en) | 2017-12-15 | 2019-06-20 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods for inhibiting t cell exhaustion |
| EP3728604A2 (en) | 2017-12-22 | 2020-10-28 | KWS SAAT SE & Co. KGaA | Targeted transcriptional regulation using synthetic transcription factors |
| KR102098915B1 (ko) | 2017-12-22 | 2020-04-09 | (주)지플러스 생명과학 | 키메라 게놈 조작 분자 및 방법 |
| CN111527204B (zh) | 2017-12-28 | 2023-09-01 | 国立大学法人京都大学 | 靶基因改造用组合物 |
| EP3735462A1 (en) | 2018-01-05 | 2020-11-11 | The Board of Regents of The University of Texas System | Therapeutic crispr/cas9 compositions and methods of use |
| WO2019144061A1 (en) | 2018-01-19 | 2019-07-25 | Duke University | Genome engineering with crispr-cas systems in eukaryotes |
| US12084676B2 (en) | 2018-02-23 | 2024-09-10 | Pioneer Hi-Bred International, Inc. | Cas9 orthologs |
| WO2019204750A1 (en) | 2018-04-20 | 2019-10-24 | Cellino Biotech, Inc. | Directed cell fate specification and targeted maturation |
| US20210363546A1 (en) | 2018-05-03 | 2021-11-25 | President And Fellows Of Harvard College | In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells |
| CA3101477A1 (en) | 2018-05-30 | 2019-12-05 | M2X2 Therapeutics, Inc. | Cell therapy |
| WO2020018918A1 (en) | 2018-07-19 | 2020-01-23 | The Board Of Trustees Of The University Of Illinois | Methods for exon skipping and gene knockout using base editors |
| EP3861130A4 (en) | 2018-10-02 | 2022-08-03 | Sangamo Therapeutics, Inc. | GENETIC MODULATORS |
| WO2020132226A1 (en) | 2018-12-20 | 2020-06-25 | Ohio State Innovation Foundation | Compositions and methods for reprogramming diseased musculoskeletal cells |
| BR102019009665A2 (pt) | 2018-12-21 | 2022-02-08 | Jacques P. Tremblay | Modificação da proteína precursora beta amiloide (app) através da edição de base usando o sistema crispr/cas9 |
| WO2020163396A1 (en) | 2019-02-04 | 2020-08-13 | The General Hospital Corporation | Adenine dna base editor variants with reduced off-target rna editing |
| AU2020223060B2 (en) | 2019-02-13 | 2023-04-13 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| JP2022526669A (ja) | 2019-04-12 | 2022-05-25 | デューク ユニバーシティ | ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物 |
| EP3930766A4 (en) | 2019-04-14 | 2023-02-22 | Duke University | CRISPR/CAS-BASED GENE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION |
| US20220184229A1 (en) | 2019-04-14 | 2022-06-16 | Duke University | Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy |
| JP2022537384A (ja) | 2019-06-20 | 2022-08-25 | セルジーン コーポレーション | ベネトクラクス、ギルテリチニブ、ミドスタウリン、または白血病もしくは骨髄異形成症候群を治療するための他の化合物との組み合わせにおけるアザシチジン |
| EP4010485A4 (en) | 2019-08-08 | 2023-08-23 | Duke University | HIGH-THROUGHPUT SCREENING PLATFORM FOR NEXT-GENERATION GENE THERAPY VECTOR ENGINEERING |
| EP4017544A4 (en) | 2019-08-19 | 2024-04-03 | Duke University | Skeletal myoblast progenitor cell lineage specification by crispr/cas9-based transcriptional activators |
| CN114555805A (zh) | 2019-08-19 | 2022-05-27 | 杜克大学 | 用于鉴定细胞类型命运特化的调控物的组合物和方法 |
| EP4031147A4 (en) | 2019-09-20 | 2024-10-02 | The UAB Research Foundation | Methods of treatment of neurofibromatosis type 1 (nf1) and nf1-mediated conditions and compositions for use in such methods |
| US20220364124A1 (en) | 2019-10-02 | 2022-11-17 | Duke University | Epigenetic modulation of genomic targets to control expression of pws-associated genes |
| WO2021113536A1 (en) | 2019-12-03 | 2021-06-10 | Duke University | Systems and methods for lipid nanoparticle delivery of gene editing machinery |
| EP4126224A4 (en) | 2020-04-27 | 2024-07-03 | Duke University | HIGH-THROUGHPUT SCREENING METHODS TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION |
| EP4125350A4 (en) | 2020-04-27 | 2024-04-03 | Duke University | Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1) |
| EP4125349A4 (en) | 2020-04-27 | 2024-07-10 | Duke University | GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS |
| US20230257723A1 (en) | 2020-04-27 | 2023-08-17 | Duke University | Crispr/cas9 therapies for correcting duchenne muscular dystrophy by targeted genomic integration |
| CA3182672A1 (en) | 2020-05-08 | 2021-11-11 | Duke University | Chromatin remodelers to enhance targeted gene activation |
| EP4200406A1 (en) | 2020-08-21 | 2023-06-28 | Julius-Maximilians-Universität Würzburg | Modified lymphocytes |
| EP4232152A4 (en) | 2020-10-21 | 2025-04-23 | Duke University | Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy |
| WO2022103935A1 (en) | 2020-11-11 | 2022-05-19 | The Trustees Of Columbia University In The City Of New York | Multiplex epigenome editing |
| US20240026352A1 (en) | 2020-11-13 | 2024-01-25 | Duke University | Targeted gene regulation of human immune cells with crispr-cas systems |
| WO2022133062A1 (en) | 2020-12-16 | 2022-06-23 | Epicrispr Biotechnologies, Inc. | Systems and methods for engineering characteristics of a cell |
| WO2022187288A2 (en) | 2021-03-01 | 2022-09-09 | Duke University | Systems and methods for genome-wide annotation of gene regulatory elements linked to cell fitness |
| CA3227105A1 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| US20240254483A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
| EP4463548A1 (en) | 2022-01-14 | 2024-11-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation |
| EP4463549A2 (en) | 2022-01-14 | 2024-11-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression |
| US20240058425A1 (en) | 2022-03-08 | 2024-02-22 | Duke University | Systems and methods for genome-wide annotation of gene regulatory elements linked to cell fitness |
| WO2023250511A2 (en) | 2022-06-24 | 2023-12-28 | Tune Therapeutics, Inc. | Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics Inc | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| WO2024040253A1 (en) | 2022-08-18 | 2024-02-22 | Duke University | Epigenetic modulation of genomic targets to control expression of pws-associated genes |
| CA3264214A1 (en) | 2022-08-19 | 2024-02-22 | Tune Therapeutics Inc | Compositions, systems and methods of regulating the hepatitis B virus by targeted gene suppression |
| CN120239746A (zh) | 2022-09-19 | 2025-07-01 | 图恩疗法股份有限公司 | 用于调节t细胞功能的组合物、系统和方法 |
| WO2024081937A2 (en) | 2022-10-13 | 2024-04-18 | Duke University | Cas12a fusion proteins and methods of using same |
-
2016
- 2016-02-09 EP EP16749752.8A patent/EP3256487A4/en active Pending
- 2016-02-09 WO PCT/US2016/017221 patent/WO2016130600A2/en not_active Ceased
- 2016-02-09 US US15/549,842 patent/US10676726B2/en active Active
- 2016-02-09 JP JP2017560481A patent/JP6929791B2/ja active Active
-
2020
- 2020-05-01 US US16/865,151 patent/US11155796B2/en active Active
-
2021
- 2021-06-21 JP JP2021102533A patent/JP7553110B2/ja active Active
- 2021-09-10 US US17/471,935 patent/US12215366B2/en active Active
-
2024
- 2024-04-05 JP JP2024061564A patent/JP2024083479A/ja active Pending
- 2024-11-19 US US18/952,981 patent/US20250257341A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2016130600A2 (en) | 2016-08-18 |
| EP3256487A2 (en) | 2017-12-20 |
| WO2016130600A9 (en) | 2016-12-22 |
| US20220098561A1 (en) | 2022-03-31 |
| WO2016130600A3 (en) | 2016-11-03 |
| US11155796B2 (en) | 2021-10-26 |
| US20250257341A1 (en) | 2025-08-14 |
| EP3256487A4 (en) | 2018-07-18 |
| US10676726B2 (en) | 2020-06-09 |
| JP2021164463A (ja) | 2021-10-14 |
| JP2024083479A (ja) | 2024-06-21 |
| JP7553110B2 (ja) | 2024-09-18 |
| JP2018505219A (ja) | 2018-02-22 |
| US20200385695A1 (en) | 2020-12-10 |
| US20180023064A1 (en) | 2018-01-25 |
| US12215366B2 (en) | 2025-02-04 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6929791B2 (ja) | エピゲノム編集のための組成物および方法 | |
| Amabile et al. | Inheritable silencing of endogenous genes by hit-and-run targeted epigenetic editing | |
| Bernstein et al. | TALE-mediated epigenetic suppression of CDKN2A increases replication in human fibroblasts | |
| EP3325018B1 (en) | High-throughput screening of regulatory element function with epigenome editing technologies | |
| JP6905755B2 (ja) | Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法 | |
| US20220305141A1 (en) | Skeletal myoblast progenitor cell lineage specification by crispr/cas9-based transcriptional activators | |
| JP2023522788A (ja) | 標的化されたゲノム組込みによってデュシェンヌ型筋ジストロフィーを矯正するためのｃｒｉｓｐｒ／ｃａｓ９療法 | |
| US20230416747A1 (en) | Safe harbor loci | |
| JP2021522188A (ja) | 内因性遺伝子活性化を用いる多重腫瘍ワクチン接種のための組成物および方法 | |
| US11072782B2 (en) | Construct for epigenetic modification and its use in the silencing of genes | |
| US11439692B2 (en) | Method of treating diseases associated with MYD88 pathways using CRISPR-GNDM system | |
| Ma et al. | Reactivation of γ-globin expression using a minicircle DNA system to treat β-thalassemia | |
| JP2024506645A (ja) | 転写調節遺伝子の編集による疾患の治癒 | |
| JP7672006B2 (ja) | 制御性ｔ細胞を改変するための組成物および方法 | |
| CN117062912A (zh) | 用于基于crispr的转录抑制的融合蛋白 | |
| US20190307845A1 (en) | USE OF SYNTHETIC HISTONE-BINDING REGULATOR PROTEIN PcTF TO ACTIVATE SUPPRESSION OF CANCER CELL GROWTH | |
| US20250387514A1 (en) | Compositions and methods for epigenetic regulation of trac expression | |
| WO2025119306A1 (zh) | 优化工程化t细胞的碱基编辑系统及其应用 | |
| EA050294B1 (ru) | Безопасный локус | |
| CA3209126A1 (en) | Small molecule-regulated cell signaling expression system | |
| Curinga et al. | 332. Directed Integration of Insulated Lentiviral Vectors To the Heterochromatin Towards Safer Gene Transfer To Stem Cells | |
| Zhang et al. | 411. Ubiquitous Chromatin Opening Element (UCOE)-Driven Transgenes within Lentiviral Vectors Are Not Subject to DNA Methylation Mediated Silencing | |
| Sajib et al. | 330. Genetic Modification To Achieve Targeting of Adenoviral Vectors To Malignant Cells of Lymphocyte Origin |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20190212 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20190212 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20200406 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20200706 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20201221 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20210322 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20210520 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20210621 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20210712 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20210811 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 6929791 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |