ES2906263T3 - Silenciamiento génico epigénico permanente - Google Patents
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- ES2906263T3 ES2906263T3 ES15794644T ES15794644T ES2906263T3 ES 2906263 T3 ES2906263 T3 ES 2906263T3 ES 15794644 T ES15794644 T ES 15794644T ES 15794644 T ES15794644 T ES 15794644T ES 2906263 T3 ES2906263 T3 ES 2906263T3
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| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| CN107847524A (zh) * | 2015-03-27 | 2018-03-27 | 哈佛学院校长同事会 | 经过修饰的t细胞及其制备和使用方法 |
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| US11970710B2 (en) | 2015-10-13 | 2024-04-30 | Duke University | Genome engineering with Type I CRISPR systems in eukaryotic cells |
| EA201891317A3 (ru) | 2015-11-30 | 2019-04-30 | Дьюк Юниверсити | Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения |
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| BR112019023608A2 (pt) | 2017-05-12 | 2020-05-26 | Crispr Therapeutics Ag | Materiais e métodos para células manipuladas e seus usos em imuno-oncologia |
| US11072782B2 (en) | 2017-07-24 | 2021-07-27 | Albert-Ludwigs-Universitaet Freiburg | Construct for epigenetic modification and its use in the silencing of genes |
| CN107759673B (zh) * | 2017-09-27 | 2021-06-04 | 复旦大学 | 可对hbv的dna进行表观甲基化修饰的蛋白分子及其应用 |
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| US12428639B2 (en) | 2019-03-05 | 2025-09-30 | Ramot At Tel-Aviv University Ltd. | Plant DNA methyltransferases and uses thereof |
| PE20212332A1 (es) | 2019-04-23 | 2021-12-14 | Sangamo Therapeutics Inc | Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos |
| WO2020222176A1 (en) | 2019-04-30 | 2020-11-05 | Crispr Therapeutics Ag | Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19 |
| AU2020352552A1 (en) | 2019-09-23 | 2022-03-17 | Omega Therapeutics, Inc. | Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression |
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| US20210139935A1 (en) * | 2019-11-13 | 2021-05-13 | Crispr Therapeutics Ag | Methods of manufacturing car-t cells |
| WO2022067033A1 (en) * | 2020-09-24 | 2022-03-31 | Flagship Pioneering Innovations V, Inc. | Compositions and methods for inhibiting gene expression |
| MX2023007524A (es) * | 2020-12-22 | 2023-09-19 | Chroma Medicine Inc | Composiciones y metodos para edicion epigenetica. |
| WO2022162247A1 (en) * | 2021-02-01 | 2022-08-04 | Epsilen Bio S.R.L. | Gene silencing |
| KR20230171424A (ko) * | 2021-02-15 | 2023-12-20 | 오스페달레 산 라파엘 에스.알.엘. | 암의 치료를 위한 후성적 침묵 |
| CN115247175B (zh) * | 2021-11-25 | 2025-10-17 | 南京启真基因工程有限公司 | 构建setdb1基因突变的表观遗传失调模型猪核移植供体细胞的基因编辑系统及其应用 |
| EP4486901A1 (en) * | 2022-03-04 | 2025-01-08 | Epigenic Therapeutics Inc. | Compositions and methods of genome editing |
| WO2023250490A1 (en) * | 2022-06-23 | 2023-12-28 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of trac expression |
| CA3260474A1 (en) * | 2022-06-23 | 2023-12-28 | Chroma Medicine Inc | Compositions and methods for epigenetic regulation of b2m expression |
| WO2023250512A1 (en) * | 2022-06-23 | 2023-12-28 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of ciita expression |
| WO2023250148A1 (en) | 2022-06-23 | 2023-12-28 | Chroma Medicine, Inc. | Compositions and methods for epigenetic editing |
| AU2023289696A1 (en) | 2022-06-24 | 2025-01-16 | Tune Therapeutics, Inc. | Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression |
| TW202426641A (zh) | 2022-08-19 | 2024-07-01 | 美商圖恩療法股份有限公司 | 以靶向基因抑制來調節b型肝炎病毒之組合物、系統和方法 |
| EP4590822A1 (en) * | 2022-09-23 | 2025-07-30 | nChroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2024081879A1 (en) * | 2022-10-14 | 2024-04-18 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of cd247 expression |
| CN120981573A (zh) | 2023-02-17 | 2025-11-18 | 怀特黑德生物医学研究所 | 用于进行表观遗传修饰的组合物和方法 |
| WO2024220857A1 (en) * | 2023-04-20 | 2024-10-24 | Chroma Medicine, Inc. | Fusion proteins for epigenetic regulation |
| WO2024238700A1 (en) * | 2023-05-15 | 2024-11-21 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| IT202300011790A1 (it) | 2023-06-08 | 2024-12-08 | Fond Telethon Ets | Protocolli di manipolazione genica in cellule immunitarie |
| WO2025031446A1 (zh) * | 2023-08-09 | 2025-02-13 | 厦门大学 | 瓜氨酸化setdb1多肽及其在诊断类风湿性关节炎中的应用 |
| WO2025160155A1 (en) | 2024-01-22 | 2025-07-31 | The Broad Institute, Inc. | Epigenetic targeting of prion diseases |
Family Cites Families (56)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5723315A (en) | 1996-08-23 | 1998-03-03 | Genetics Institute, Inc. | Secreted proteins and polynucleotides encoding them |
| WO1998005635A1 (en) | 1996-08-07 | 1998-02-12 | Darwin Discovery Limited | Hydroxamic and carboxylic acid derivatives having mmp and tnf inhibitory activity |
| US6126939A (en) | 1996-09-03 | 2000-10-03 | Yeda Research And Development Co. Ltd. | Anti-inflammatory dipeptide and pharmaceutical composition thereof |
| GB9803351D0 (en) | 1998-02-17 | 1998-04-15 | Oxford Biomedica Ltd | Anti-viral vectors |
| US20020188103A1 (en) | 1998-10-09 | 2002-12-12 | Timothy H. Bestor | Chimeric dna-binding/dna methyltransferase nucleic acid and polypeptide and uses thereof |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| GB0009760D0 (en) | 2000-04-19 | 2000-06-07 | Oxford Biomedica Ltd | Method |
| US7001768B2 (en) * | 2000-04-28 | 2006-02-21 | Sangamo Biosciences, Inc. | Targeted modification of chromatin structure |
| US6919204B2 (en) | 2000-09-29 | 2005-07-19 | Sangamo Biosciences, Inc. | Modulation of gene expression using localization domains |
| 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 |
| US7407776B2 (en) | 2003-09-19 | 2008-08-05 | Sangamo Biosciences, Inc. | Engineered zinc finger proteins for regulation of gene expression |
| US20070192880A1 (en) * | 2003-10-03 | 2007-08-16 | University Of Rochester | Horming response element binding transregulators |
| WO2005100392A2 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| GB0526449D0 (en) | 2005-12-23 | 2006-02-08 | Medical Res Council | Polypeptide targeting |
| KR100756055B1 (ko) | 2006-01-27 | 2007-09-07 | 연세대학교 산학협력단 | 신생혈관 생성을 조절하는 재조합 아데노바이러스 |
| GB0607063D0 (en) | 2006-04-07 | 2006-05-17 | Cellcentric Ltd | Compositions and methods for epigenetic modification of nucleic acid sequences in vivo |
| GB0700374D0 (en) | 2007-01-09 | 2007-02-14 | Oncomethylome Sciences S A | NDRG family methylation markers |
| WO2010108126A2 (en) * | 2009-03-19 | 2010-09-23 | Fate Therapeutics, Inc. | Reprogramming compositions and methods of using the same |
| WO2011016840A2 (en) | 2009-07-28 | 2011-02-10 | Sangamo Biosciences, Inc. | Methods and compositions for treating trinucleotide repeat disorders |
| SI3590949T1 (sl) * | 2010-10-01 | 2022-09-30 | Modernatx, Inc. | Ribonukleinske kisline, ki vsebujejo N1-metil-psevdouracil, in njihove uporabe |
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US8450107B1 (en) | 2011-11-30 | 2013-05-28 | The Broad Institute Inc. | Nucleotide-specific recognition sequences for designer TAL effectors |
| GB201121924D0 (en) * | 2011-12-20 | 2012-02-01 | Fahy Gurteen Labs Ltd | Detection of breast cancer |
| WO2013130807A1 (en) | 2012-02-28 | 2013-09-06 | Sigma-Aldrich Co. Llc | Targeted histone acetylation |
| AU2013225950B2 (en) | 2012-02-29 | 2018-02-15 | Sangamo Therapeutics, Inc. | Methods and compositions for treating huntington's disease |
| JP6329936B2 (ja) * | 2012-03-14 | 2018-05-23 | ソーク インスティテュート フォー バイオロジカル スタディーズ | アデノウイルス腫瘍診断 |
| WO2013141680A1 (en) | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
| US20160046961A1 (en) | 2012-05-25 | 2016-02-18 | Emmanuelle Charpentier | Methods and Compositions for RNA-Directed Target DNA Modification and For RNA-Directed Modulation of Transcription |
| KR101706085B1 (ko) | 2012-10-23 | 2017-02-14 | 주식회사 툴젠 | 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도 |
| EP2928496B1 (en) | 2012-12-06 | 2019-10-09 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
| CA2894668A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells |
| US8993233B2 (en) | 2012-12-12 | 2015-03-31 | The Broad Institute Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| CN105209621B (zh) | 2012-12-12 | 2021-05-25 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| CN105121648B (zh) | 2012-12-12 | 2021-05-07 | 布罗德研究所有限公司 | 用于序列操纵的系统、方法和优化的指导组合物的工程化 |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US9970024B2 (en) | 2012-12-17 | 2018-05-15 | President And Fellows Of Harvard College | RNA-guided human genome engineering |
| EP2971041B1 (en) | 2013-03-15 | 2018-11-28 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
| 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 |
| US9267135B2 (en) | 2013-06-04 | 2016-02-23 | President And Fellows Of Harvard College | RNA-guided transcriptional regulation |
| CA2914519A1 (en) | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| RU2748433C2 (ru) | 2013-07-10 | 2021-05-25 | Президент Энд Фэллоуз Оф Харвард Коллидж | ОРТОГОНАЛЬНЫЕ БЕЛКИ Cas9 ДЛЯ РНК-НАПРАВЛЯЕМОЙ РЕГУЛЯЦИИ И РЕДАКТИРОВАНИЯ ГЕНОВ |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US20150191744A1 (en) | 2013-12-17 | 2015-07-09 | University Of Massachusetts | Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling |
| AU2015234204A1 (en) | 2014-03-20 | 2016-10-06 | Universite Laval | CRISPR-based methods and products for increasing frataxin levels and uses thereof |
| US20170219596A1 (en) | 2014-07-14 | 2017-08-03 | 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 |
| WO2016054106A1 (en) | 2014-09-29 | 2016-04-07 | The Regents Of The University Of California | SCAFFOLD RNAs |
| EP3204032B1 (en) | 2014-10-10 | 2024-03-27 | Massachusetts Eye & Ear Infirmary | Chimeric molecule comprising crispr/cas or cas9 nuclease and at least one guide rna targeting pmca2 gene locus and fused, complexed or linked to an anionic fluorescent protein, and its use in the treatment of deafness or disorders thereof |
| GB201418965D0 (enExample) | 2014-10-24 | 2014-12-10 | Ospedale San Raffaele And Fond Telethon | |
| LT3250691T (lt) | 2015-01-28 | 2023-09-11 | Caribou Biosciences, Inc. | Crispr hibridiniai dnr/rnr polinukleotidai ir naudojimo būdai |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | 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 |
| SG10201913151TA (en) | 2015-09-09 | 2020-03-30 | Seattle Childrens Hospital Dba Seattle Childrens Res Inst | Genetic engineering of macrophages for immunotherapy |
| EP3147363B1 (en) | 2015-09-26 | 2019-10-16 | B.R.A.I.N. Ag | Activation of taste receptor genes in mammalian cells using crispr-cas-9 |
| JP6500293B2 (ja) | 2015-11-25 | 2019-04-17 | 国立大学法人群馬大学 | Ｄｎａメチル化編集用キットおよびｄｎａメチル化編集方法 |
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