TW202016310A - 經密碼子最佳化之酸性α葡萄糖苷酶表現盒及其使用方法 - Google Patents
經密碼子最佳化之酸性α葡萄糖苷酶表現盒及其使用方法 Download PDFInfo
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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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| BR112021003399A2 (pt) | 2018-08-24 | 2021-05-18 | Spark Therapeutics, Inc. | sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso |
| EP3996696A1 (en) * | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| JP2023507575A (ja) | 2019-12-20 | 2023-02-24 | コデクシス， インコーポレイテッド | 操作された酸性アルファ－グルコシダーゼバリアント |
| MX2023008362A (es) * | 2021-01-14 | 2023-10-04 | Spark Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de fabry. |
| CR20240137A (es) * | 2021-08-20 | 2024-07-29 | Biocad Joint Stock Co | Método para obtener una cápside de virus adenoasociado modificada |
| CA3229998A1 (en) * | 2021-08-25 | 2023-03-02 | Canbridge Pharmaceuticals, Inc. | Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease |
| AR126877A1 (es) * | 2021-08-25 | 2023-11-22 | Canbridge Pharmaceuticals Inc | PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GLUCOSIDASA ÁCIDA (GAA) Y SU USO PARA TRATAR LA ENFERMEDAD DE POMPE |
| CA3242731A1 (en) * | 2022-02-02 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease |
| CN118667797A (zh) * | 2023-03-14 | 2024-09-20 | 北京据德医药科技有限公司 | 一种重组酸性α-葡萄糖苷酶及其用途 |
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| CN103443273B (zh) | 2011-03-16 | 2016-10-26 | 天野酶制品株式会社 | 修饰型α-葡萄糖苷酶及其用途 |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| CN104411338A (zh) * | 2012-04-02 | 2015-03-11 | 现代治疗公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| NZ701693A (en) * | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| WO2015042064A1 (en) * | 2013-09-17 | 2015-03-26 | Edeniq, Inc. | Improved beta-glucosidase enzymes for increased biomass saccharification |
| AU2014354839B2 (en) * | 2013-11-26 | 2020-06-04 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Adeno-associated virus vectors for treatment of glycogen storage disease |
| US10781459B2 (en) * | 2014-06-20 | 2020-09-22 | University Of Florida Research Foundation, Incorporated | Methods of packaging multiple adeno-associated virus vectors |
| EP3211076A4 (en) | 2014-10-20 | 2018-03-21 | Showa Sangyo Co., Ltd. | Novel alpha-glucosidase |
| WO2018046774A1 (en) * | 2016-09-12 | 2018-03-15 | Genethon | Acid-alpha glucosidase variants and uses thereof |
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| US20250101396A1 (en) | 2025-03-27 |
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| PE20210337A1 (es) | 2021-02-19 |
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