CA3114109A1 - Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder - Google Patents
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Classifications
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- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C12N15/09—Recombinant DNA-technology
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- 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
- C12N15/86—Viral vectors
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C12N2330/00—Production
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Applications Claiming Priority (3)
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| US201862736847P | 2018-09-26 | 2018-09-26 | |
| US62/736,847 | 2018-09-26 | ||
| PCT/US2019/053268 WO2020069199A1 (en) | 2018-09-26 | 2019-09-26 | Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder |
Publications (1)
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| CA3114109A1 true CA3114109A1 (en) | 2020-04-02 |
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| CA3114109A Pending CA3114109A1 (en) | 2018-09-26 | 2019-09-26 | Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder |
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| WO (1) | WO2020069199A1 (enExample) |
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| MX2022005362A (es) * | 2019-11-04 | 2022-09-23 | Flagship Pioneering Inc | Composiciones de trem para codones con-raros y usos relacionados. |
| EP4158031A1 (en) * | 2020-05-29 | 2023-04-05 | Flagship Pioneering Innovations VI, LLC | Trem compositions and methods relating thereto |
| AU2021281453A1 (en) * | 2020-05-29 | 2022-11-17 | Flagship Pioneering Innovations Vi, Llc. | Trem compositions and methods relating thereto |
| BR112023022805A2 (pt) * | 2021-05-05 | 2024-01-16 | Tevard Biosciences Inc | Métodos e composições para tratamento de um distúrbio mediado por códon de terminação prematura |
| TW202405173A (zh) | 2022-04-18 | 2024-02-01 | 美商維泰克斯製藥公司 | 用於增強aav療法及降低aav向肝臟之趨性的組合物及方法 |
| CN117257958B (zh) * | 2023-11-21 | 2024-02-09 | 四川大学华西医院 | Trps1抑制剂的新用途及用于治疗和/或预防雄激素性秃发的药物 |
| WO2025176166A1 (en) * | 2024-02-21 | 2025-08-28 | Zhuhai Codone Biotechnology Co., Ltd. | Engineered trna expression cassette |
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| JP2002525048A (ja) | 1998-09-17 | 2002-08-13 | スミスクライン・ビーチャム・コーポレイション | Ｒｎａｓｅｐポリペプチド |
| US20040142325A1 (en) | 2001-09-14 | 2004-07-22 | Liat Mintz | Methods and systems for annotating biomolecular sequences |
| JP4406731B2 (ja) | 2005-03-31 | 2010-02-03 | 国立大学法人 岡山大学 | 部位特異的アミノ酸導入法のための新規な直交化ｔＲＮＡ |
| WO2008127900A1 (en) | 2007-04-13 | 2008-10-23 | The Salk Institute For Biological Studies | Methods of genetically encoding unnatural amino acids in eukaryotic cells using orthogonal trna/synthetase pairs |
| US20120283116A1 (en) * | 2009-09-11 | 2012-11-08 | Leppert Mark F | Mutant Sodium Channel Nav1.7 and Methods Related Thereto |
| WO2012006551A2 (en) | 2010-07-08 | 2012-01-12 | The Brigham And Women's Hospital, Inc. | Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules |
| US20130129668A1 (en) * | 2011-09-01 | 2013-05-23 | The Regents Of The University Of California | Diagnosis and treatment of arthritis using epigenetics |
| CA2922005A1 (en) | 2012-09-27 | 2014-04-03 | Population Diagnostics, Inc. | Methods and compositions for screening and treating developmental disorders |
| WO2015048989A1 (en) | 2013-10-02 | 2015-04-09 | Wageningen Universiteit | Systematic optimization of coding sequence for functional protein expression |
| US20180313836A1 (en) | 2015-10-15 | 2018-11-01 | The Scripps Research Institute | BINDING ACTIVITY OF AMINOACYL-tRNA SYNTHETASE IN CHARCOT-MARIE-TOOTH (CMT) NEUROPATHY AND CMT-RELATED NEUROLOGICAL DISEASES |
| KR102604132B1 (ko) * | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| US11859234B2 (en) | 2016-05-06 | 2024-01-02 | The Trustees Of Princeton University | Method of monitoring RNase L activity |
| PL3458074T3 (pl) | 2016-05-16 | 2024-11-12 | Board Of Regents Of The University Of Texas System | KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA |
| WO2018104385A1 (en) | 2016-12-07 | 2018-06-14 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Codon optimization |
| WO2020243560A1 (en) | 2019-05-31 | 2020-12-03 | Flagship Pioneering, Inc. | Uses of trem compositions to modulate trna pools |
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| CO2021005040A2 (es) | 2021-09-20 |
| EP3856905A4 (en) | 2023-06-21 |
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| US20220073933A1 (en) | 2022-03-10 |
| WO2020069199A1 (en) | 2020-04-02 |
| EP3856905A1 (en) | 2021-08-04 |
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