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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- C12N2750/14111—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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- C12N2750/00011—Details
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- 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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| BR112020010883A2 (pt) | 2017-12-01 | 2020-11-10 | Encoded Therapeutics, Inc. | proteínas de ligação a dna engenheiradas |
| 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 |
| WO2020150832A1 (en) * | 2019-01-24 | 2020-07-30 | Valorisation-Hsj, Limited Partnership | Cell-specific transcriptional regulatory sequences and uses thereof |
| US20220168449A1 (en) * | 2019-04-12 | 2022-06-02 | Encoded Therapeutics, Inc. | Compositions and methods for administration of therapeutics |
| SG11202113048SA (en) | 2019-05-29 | 2021-12-30 | Encoded Therapeutics Inc | Compositions and methods for selective gene regulation |
| 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 |
| IL294781A (en) * | 2020-02-14 | 2022-09-01 | Ultragenyx Pharmaceutical Inc | Gene therapy for treating cdkl5 deficiency disorder |
| BR112022021762A2 (pt) * | 2020-04-27 | 2023-01-17 | Univ Pennsylvania | Composições úteis em tratamento de distúrbio de deficiência de cdkl5 (cdd) |
| AU2022325165A1 (en) * | 2021-08-05 | 2024-01-18 | Insmed Incorporated | Adeno-associated virus particles and methods of use thereof |
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| US20090018031A1 (en) | 2006-12-07 | 2009-01-15 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways tools, and methods |
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| US20130129668A1 (en) * | 2011-09-01 | 2013-05-23 | The Regents Of The University Of California | Diagnosis and treatment of arthritis using epigenetics |
| JP6373763B2 (ja) | 2012-02-17 | 2018-08-22 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| EP2909324B1 (en) | 2012-10-25 | 2020-02-26 | Tocagen Inc. | Retroviral vector with mini-promoter cassette |
| 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 |
| CN110944674B (zh) | 2017-05-19 | 2024-07-19 | 编码治疗公司 | 高活性调控元件 |
| BR112020010883A2 (pt) * | 2017-12-01 | 2020-11-10 | Encoded Therapeutics, Inc. | proteínas de ligação a dna engenheiradas |
| AU2020229382A1 (en) * | 2019-02-28 | 2021-09-16 | 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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| CN110944674B (zh) | 2024-07-19 |
| WO2018213786A1 (en) | 2018-11-22 |
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| US20240376441A1 (en) | 2024-11-14 |
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