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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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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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- C12N2830/00—Vector systems having a special element relevant for transcription
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- 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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| US201762508968P | 2017-05-19 | 2017-05-19 | |
| US62/508,968 | 2017-05-19 |
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| TW201903152A TW201903152A (zh) | 2019-01-16 |
| TWI811219B true TWI811219B (zh) | 2023-08-11 |
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| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| CN118956965A (zh) | 2017-05-19 | 2024-11-15 | 编码治疗公司 | 高活性调控元件 |
| JP7401432B2 (ja) | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス， インコーポレイテッド | 操作されたｄｎａ結合タンパク質 |
| 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 |
| WO2020210633A1 (en) * | 2019-04-12 | 2020-10-15 | Encoded Therapeutics, Inc. | Compositions and methods for administration of therapeutics |
| WO2020243651A1 (en) | 2019-05-29 | 2020-12-03 | Encoded Therapeutics, Inc. | Compositions and methods for selective gene regulation |
| CA3147875A1 (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 |
| BR112022015921A2 (pt) * | 2020-02-14 | 2022-10-04 | Ultragenyx Pharmaceutical Inc | Terapia gênica para tratar o transtorno de deficiência de cdkl5 |
| EP4150051A4 (en) * | 2020-04-27 | 2025-05-21 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD) |
| CA3224482A1 (en) * | 2021-08-05 | 2023-02-09 | Brian Kaspar | Adeno-associated virus particles and methods of use thereof |
| EP4602061A1 (en) * | 2022-10-11 | 2025-08-20 | MeiraGTx UK II Limited | Atp7b gene therapy |
| 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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| 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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| US6436703B1 (en) * | 2000-03-31 | 2002-08-20 | Hyseq, Inc. | Nucleic acids and polypeptides |
| 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 |
| KR102057540B1 (ko) * | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| US11065311B2 (en) | 2012-10-25 | 2021-07-20 | Denovo Biopharma Llc | Retroviral vector with mini-promoter cassette |
| JP6865167B2 (ja) * | 2014-12-17 | 2021-04-28 | フンダシオン パラ ラ インベスティガシオン メディカ アプリカダ | ウィルソン病の処置に使用するための核酸構築物及び遺伝子治療ベクター |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| CN118956965A (zh) | 2017-05-19 | 2024-11-15 | 编码治疗公司 | 高活性调控元件 |
| JP7401432B2 (ja) * | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス， インコーポレイテッド | 操作されたｄｎａ結合タンパク質 |
| MA55087A (fr) * | 2019-02-28 | 2022-01-05 | Encoded Therapeutics Inc | Compositions et procédés de traitement de laminopathies |
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Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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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| WO2018213786A1 (en) | 2018-11-22 |
| EP3624858A1 (en) | 2020-03-25 |
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| AU2018269050A1 (en) | 2020-01-16 |
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| TW201903152A (zh) | 2019-01-16 |
| KR20240024326A (ko) | 2024-02-23 |
| JP2024015408A (ja) | 2024-02-01 |
| US12104175B2 (en) | 2024-10-01 |
| MA49153A (fr) | 2021-03-24 |
| BR112019024222A2 (pt) | 2020-08-18 |
| EP3624858A4 (en) | 2021-06-23 |
| CA3063464A1 (en) | 2018-11-22 |
| US20240376441A1 (en) | 2024-11-14 |
| KR20200026814A (ko) | 2020-03-11 |
| JP2022173348A (ja) | 2022-11-18 |
| CN110944674A (zh) | 2020-03-31 |
| US20200231943A1 (en) | 2020-07-23 |
| JP2020520643A (ja) | 2020-07-16 |
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