CA2876860A1 - Supercoiled minivectors as a tool for dna repair, alteration and replacement - Google Patents
Supercoiled minivectors as a tool for dna repair, alteration and replacement Download PDFInfo
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- CA2876860A1 CA2876860A1 CA2876860A CA2876860A CA2876860A1 CA 2876860 A1 CA2876860 A1 CA 2876860A1 CA 2876860 A CA2876860 A CA 2876860A CA 2876860 A CA2876860 A CA 2876860A CA 2876860 A1 CA2876860 A1 CA 2876860A1
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- minivector
- dna sequence
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- nucleic acid
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Classifications
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- C—CHEMISTRY; METALLURGY
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- 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
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| PCT/US2013/043433 WO2013181440A1 (en) | 2012-05-30 | 2013-05-30 | Supercoiled minivectors as a tool for dna repair, alteration and replacement |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| GB2537000C (en) | 2012-05-25 | 2019-10-09 | Univ California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| CA2891347C (en) | 2012-12-06 | 2018-02-27 | Sigma-Aldrich Co. Llc | Crispr-based genome modification and regulation |
| AU2014235794A1 (en) | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| AU2014262867B2 (en) | 2013-05-10 | 2019-12-05 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| ES2844174T3 (es) | 2013-09-18 | 2021-07-21 | Kymab Ltd | Métodos, células y organismos |
| CN111218447B (zh) | 2013-11-07 | 2024-10-11 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| RU2703416C2 (ru) * | 2014-03-28 | 2019-10-16 | Эпосенс Лтд. | Соединения для транс-мембранной доставки молекул |
| US11318206B2 (en) | 2014-03-28 | 2022-05-03 | Aposense Ltd | Compounds and methods for trans-membrane delivery of molecules |
| NZ739448A (en) | 2014-04-10 | 2019-10-25 | Seattle Children’S Hospital Dba Seattle Children’S Res Institute | Transgene genetic tags and methods of use |
| EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US11071289B2 (en) | 2014-08-14 | 2021-07-27 | Biocytogen Boston Corp | DNA knock-in system |
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
| CN105821072A (zh) * | 2015-01-23 | 2016-08-03 | 深圳华大基因研究院 | 用于DNA组装的CRISPR-Cas9系统及DNA组装方法 |
| WO2017027291A1 (en) | 2015-08-07 | 2017-02-16 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Bispecific car t-cells for solid tumor targeting |
| US11667911B2 (en) | 2015-09-24 | 2023-06-06 | Editas Medicine, Inc. | Use of exonucleases to improve CRISPR/CAS-mediated genome editing |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | Harvard College | Nucleobase editors and uses thereof |
| WO2017147278A1 (en) | 2016-02-25 | 2017-08-31 | The Children's Medical Center Corporation | Customized class switch of immunoglobulin genes in lymphoma and hybridoma by crispr/cas9 technology |
| EP3219803A1 (en) * | 2016-03-15 | 2017-09-20 | Max-Delbrück-Centrum für Molekulare Medizin | Enhanced sleeping beauty transposons, kits and methods of transposition |
| WO2017165826A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| EP4047092B1 (en) | 2016-04-13 | 2025-07-30 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
| KR20190012229A (ko) | 2016-06-01 | 2019-02-08 | 케이더블유에스 사아트 에스이 | 유전자 조작을 위한 하이브리드 핵산 서열 |
| US10892034B2 (en) | 2016-07-01 | 2021-01-12 | Microsoft Technology Licensing, Llc | Use of homology direct repair to record timing of a molecular event |
| US11359234B2 (en) | 2016-07-01 | 2022-06-14 | Microsoft Technology Licensing, Llc | Barcoding sequences for identification of gene expression |
| CN109477130B (zh) | 2016-07-01 | 2022-08-30 | 微软技术许可有限责任公司 | 通过迭代dna编辑的存储 |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| US11873325B2 (en) | 2016-10-10 | 2024-01-16 | Limagrain Europe | Nucleic acid encoding Sm1 resistance to orange wheat blossom midge and method of use |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| CN109906030B (zh) | 2016-11-04 | 2022-03-18 | 安健基因公司 | 用于产生仅重链抗体的经基因修饰的非人动物和方法 |
| CN110291200B (zh) | 2016-12-12 | 2024-05-14 | 西雅图儿童医院（Dba西雅图儿童研究所） | 对哺乳动物细胞中转基因表达的药剂配体诱导具有增大的灵敏度的嵌合转录因子变异体 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| KR20190104392A (ko) | 2017-01-09 | 2019-09-09 | 아포센스 엘티디. | 분자의 막투과 전달을 위한 화합물 및 방법 |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| US20180305701A1 (en) | 2017-03-14 | 2018-10-25 | Baylor College Of Medicine | Targeting minivectors to specific tissue using shape |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| EP3692156A1 (en) | 2017-10-05 | 2020-08-12 | Biogemma | Improved yield in plants by overexpressing a trehalose-6 phosphate synthase |
| AU2018352592C1 (en) | 2017-10-16 | 2025-09-25 | Beam Therapeutics, Inc. | Uses of adenosine base editors |
| WO2019086510A1 (en) | 2017-10-31 | 2019-05-09 | Vilmorin & Cie | Wheat comprising male fertility restorer alleles |
| US20200048716A1 (en) | 2017-11-03 | 2020-02-13 | Twister Biotech, Inc | Using minivectors to treat ovarian cancer |
| MX2020005228A (es) * | 2017-11-21 | 2020-08-24 | Monsanto Technology Llc | Plantas modificadas con rasgos mejorados. |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| IL275773B2 (en) | 2018-01-01 | 2025-07-01 | Aposense Ltd | Compounds and methods for trans-membrane delivery of molecules |
| GB201805865D0 (en) | 2018-04-09 | 2018-05-23 | Innes John Centre | Genes |
| MA52709A (fr) | 2018-05-23 | 2021-03-31 | Modernatx Inc | Administration d'adn |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | 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 |
| US12241076B2 (en) | 2019-02-06 | 2025-03-04 | Vilmorin & Cie | Gene responsible for cytoplasmic male sterility |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3145863A1 (en) | 2019-07-05 | 2021-01-14 | Limagrain Europe | Method for increasing yield in plants |
| FR3099178A1 (fr) | 2019-07-24 | 2021-01-29 | Soltis | Tournesol à teneur élevée en acide oléique et procédé d’obtention |
| JP2022549138A (ja) | 2019-09-18 | 2022-11-24 | インターガラクティック セラピューティクス インコーポレイテッド | 合成ｄｎａベクターおよびその使用法 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CN112779266A (zh) | 2019-11-06 | 2021-05-11 | 青岛清原化合物有限公司 | 在生物体内创制新基因的方法及应用 |
| WO2021202832A1 (en) * | 2020-04-03 | 2021-10-07 | Progenitor Life Sciences | Targeting tapasin and tap complex to improve cellular immune-compatibility |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| JP2023527875A (ja) | 2020-06-01 | 2023-06-30 | モダーナティエックス・インコーポレイテッド | フェニルアラニンヒドロキシラーゼバリアント及びその使用 |
| EP4337177A1 (en) | 2021-05-11 | 2024-03-20 | Modernatx, Inc. | Non-viral delivery of dna for prolonged polypeptide expression in vivo |
| US20240093226A1 (en) | 2021-09-10 | 2024-03-21 | Baylor College Of Medicine | Ultrapure minivectors for gene therapy |
| US20240100189A1 (en) | 2021-09-10 | 2024-03-28 | Twister Biotech, Inc | Using minivectors to treat idiopathic pulmonary fibrosis |
| US20230190955A1 (en) | 2021-10-21 | 2023-06-22 | Baylor College Of Medicine | Treatment of liver cancer or liver fibrosis |
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| US20100076057A1 (en) * | 2008-09-23 | 2010-03-25 | Northwestern University | TARGET DNA INTERFERENCE WITH crRNA |
| CA2777835A1 (en) * | 2009-10-16 | 2011-04-21 | Baylor College Of Medicine | Supercoiled minicircle dna for gene therapy applications |
| WO2012064970A1 (en) * | 2010-11-12 | 2012-05-18 | The Board Of Trustees Of The Leland Stanford Junior University | Site-directed integration of transgenes in mammals |
| WO2013158309A2 (en) * | 2012-04-18 | 2013-10-24 | The Board Of Trustees Of The Leland Stanford Junior University | Non-disruptive gene targeting |
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| US20140056868A1 (en) | 2014-02-27 |
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| KR20150027756A (ko) | 2015-03-12 |
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