TWI811219B - 高活性調控元件 - Google Patents
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- TWI811219B TWI811219B TW107117128A TW107117128A TWI811219B TW I811219 B TWI811219 B TW I811219B TW 107117128 A TW107117128 A TW 107117128A TW 107117128 A TW107117128 A TW 107117128A TW I811219 B TWI811219 B TW I811219B
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- C12N2750/14011—Parvoviridae
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- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
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- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
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| IL274926B2 (en) | 2017-12-01 | 2026-04-01 | Encoded Therapeutics Inc | Pyrazolopyrimidines with activity against respiratory syncytial virus |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| EP3914719A4 (en) | 2019-01-24 | 2023-04-05 | Valorisation-HSJ, Limited Partnership | Cell-specific transcriptional regulatory sequences and uses thereof |
| MA55625A (fr) * | 2019-04-12 | 2022-02-16 | Encoded Therapeutics Inc | Compositions et méthodes d'administration de produits thérapeutiques |
| WO2020243651A1 (en) | 2019-05-29 | 2020-12-03 | Encoded Therapeutics, Inc. | Compositions and methods for selective gene regulation |
| WO2021016075A1 (en) | 2019-07-19 | 2021-01-28 | Flagship Pioneering Innovations Vi, Llc | Recombinase compositions and methods of use |
| CN113088519B (zh) * | 2019-12-23 | 2024-02-02 | 苏州华毅乐健生物科技有限公司 | 一种分离的核酸分子及其应用 |
| MX2022009982A (es) * | 2020-02-14 | 2022-09-12 | Ultragenyx Pharmaceutical Inc | Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5. |
| IL297635A (en) * | 2020-04-27 | 2022-12-01 | Univ Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| JP2024527742A (ja) * | 2021-08-05 | 2024-07-26 | インスメッド インコーポレイテッド | アデノ随伴ウイルス粒子及びその使用方法 |
| KR20250102134A (ko) * | 2022-10-11 | 2025-07-04 | 메이라지티엑스 유케이 Ii 리미티드 | Atp7b 유전자 요법 |
| CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
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| EP1783645A1 (en) * | 2005-11-08 | 2007-05-09 | Actigenics | Methods for the identification of microRNA and their applications in research and human health |
| US20090018031A1 (en) * | 2006-12-07 | 2009-01-15 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways tools, and methods |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| WO2013033627A2 (en) * | 2011-09-01 | 2013-03-07 | The Regents Of The University Of California | Diagnosis and treatment of arthritis using epigenetics |
| AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| EP3623478A1 (en) | 2012-10-25 | 2020-03-18 | Tocagen Inc. | Retroviral vector with mini-promoter cassette |
| ES2876412T3 (es) * | 2014-12-17 | 2021-11-12 | Fundacion Para La Investig Medica Aplicada | Construcciones de ácido nucleico y vectores de terapia génica para su uso en el tratamiento de la enfermedad de Wilson |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| CA3063464A1 (en) | 2017-05-19 | 2018-11-22 | Encoded Therapeutics, Inc. | High activity regulatory elements |
| IL274926B2 (en) * | 2017-12-01 | 2026-04-01 | Encoded Therapeutics Inc | Pyrazolopyrimidines with activity against respiratory syncytial virus |
| MA55087A (fr) * | 2019-02-28 | 2022-01-05 | Encoded Therapeutics Inc | Compositions et procédés de traitement de laminopathies |
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| WO2001036620A2 (en) * | 1999-11-16 | 2001-05-25 | Genzyme Corporation | Vectors and transgenies with regulatory elements for gene delivery to the liver |
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| JP2022173348A (ja) | 2022-11-18 |
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| BR112019024222A2 (pt) | 2020-08-18 |
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| CN118956965A (zh) | 2024-11-15 |
| US20240376441A1 (en) | 2024-11-14 |
| KR102636351B1 (ko) | 2024-02-15 |
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