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
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- 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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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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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- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- C—CHEMISTRY; METALLURGY
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- C12N2830/00—Vector systems having a special element relevant for transcription
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
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| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| AU2018269050A1 (en) | 2017-05-19 | 2020-01-16 | Encoded Therapeutics, Inc. | High activity regulatory elements |
| EP3717505A4 (en) | 2017-12-01 | 2021-12-01 | Encoded Therapeutics, Inc. | GENENICALLY MODIFIED DNA-BINDING PROTEINS |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| MX2021008739A (es) * | 2019-01-24 | 2021-10-13 | Valorisation Hsj Lp | Secuencias reguladoras de la transcripción específicas de células y usos de estas. |
| EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
| PL3976798T3 (pl) | 2019-05-29 | 2026-04-07 | Encoded Therapeutics, Inc. | Kompozycje i sposoby selektywnej regulacji genów |
| JP7759311B2 (ja) | 2019-07-19 | 2025-10-23 | フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー | リコンビナーゼ組成物及び使用方法 |
| 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 |
| JP2023514204A (ja) * | 2020-02-14 | 2023-04-05 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | Ｃｄｋｌ５欠損障害を処置するための遺伝子治療 |
| KR20230003569A (ko) * | 2020-04-27 | 2023-01-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물 |
| US20250041450A1 (en) * | 2021-08-05 | 2025-02-06 | Insmed Incorporated | Adeno-associated virus particles and methods of use thereof |
| JP2025533994A (ja) * | 2022-10-11 | 2025-10-09 | メイラグティーエックス ユーケー アイアイ リミティド | Ａｔｐ７ｂ遺伝子療法 |
| 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 |
| CN117947035A (zh) * | 2024-03-25 | 2024-04-30 | 上海凌医生物科技有限公司 | 一种增强基因表达的启动子 |
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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 |
| WO2008073303A2 (en) * | 2006-12-07 | 2008-06-19 | 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 |
| MX367100B (es) * | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| ES2786051T3 (es) | 2012-10-25 | 2020-10-08 | Tocagen Inc | Vector retroviral con casete de mini-promotor |
| US11147887B2 (en) * | 2014-12-17 | 2021-10-19 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| AU2018269050A1 (en) | 2017-05-19 | 2020-01-16 | Encoded Therapeutics, Inc. | High activity regulatory elements |
| EP3717505A4 (en) * | 2017-12-01 | 2021-12-01 | Encoded Therapeutics, Inc. | GENENICALLY MODIFIED DNA-BINDING PROTEINS |
| SG11202109362XA (en) * | 2019-02-28 | 2021-09-29 | Encoded Therapeutics Inc | Compositions and methods for treating 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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| CA3063464A1 (en) | 2018-11-22 |
| MA49153A (fr) | 2021-03-24 |
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| TW201903152A (zh) | 2019-01-16 |
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| CN110944674B (zh) | 2024-07-19 |
| US20240376441A1 (en) | 2024-11-14 |
| AU2018269050A1 (en) | 2020-01-16 |
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