ATE553206T1 - Expression von genen mit überlappenden offenen leserastern in insektenzellen, verfahren und zusammensetzungen dafür - Google Patents

Expression von genen mit überlappenden offenen leserastern in insektenzellen, verfahren und zusammensetzungen dafür

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Publication number
ATE553206T1
ATE553206T1 AT07814439T AT07814439T ATE553206T1 AT E553206 T1 ATE553206 T1 AT E553206T1 AT 07814439 T AT07814439 T AT 07814439T AT 07814439 T AT07814439 T AT 07814439T AT E553206 T1 ATE553206 T1 AT E553206T1
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insect cells
methods
open reading
gene
genes
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AT07814439T
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English (en)
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Haifeng Chen
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Virovek Inc
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/09Recombinant DNA-technology
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    • C12N2750/00011Details
    • C12N2750/00041Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011Parvoviridae
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/103Plasmid DNA for invertebrates
    • C12N2800/105Plasmid DNA for invertebrates for insects
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    • C12N2830/00Vector systems having a special element relevant for transcription
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    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AT07814439T 2006-08-24 2007-08-24 Expression von genen mit überlappenden offenen leserastern in insektenzellen, verfahren und zusammensetzungen dafür ATE553206T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US83976106P 2006-08-24 2006-08-24
PCT/US2007/076799 WO2008024998A2 (en) 2006-08-24 2007-08-24 Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor

Publications (1)

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ATE553206T1 true ATE553206T1 (de) 2012-04-15

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Country Status (7)

Country Link
US (4) US8945918B2 (de)
EP (1) EP2061891B1 (de)
CN (1) CN101522903B (de)
AT (1) ATE553206T1 (de)
DK (1) DK2061891T3 (de)
ES (1) ES2385679T3 (de)
WO (1) WO2008024998A2 (de)

Families Citing this family (91)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011512156A (ja) * 2008-02-19 2011-04-21 アムステルダム モレキュラー セラピューティクス ビー.ブイ. 昆虫細胞におけるパルボウイルスrep及びcapタンパク質の発現の最適化
US20110319478A1 (en) 2010-06-04 2011-12-29 Medtronic, Inc. Compositions to Treat Cardiac Pacing Conditions
EP2788489B1 (de) 2011-12-08 2017-03-15 Virovek, Inc. Vektoren mit toxischen genen, verfahren und verwendungen dafür
CN104363961B (zh) 2012-02-21 2017-10-03 斯坦福大学托管董事会 用于治疗盆底神经源性病症的组合物和方法
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
CN105142529A (zh) 2012-11-21 2015-12-09 电路治疗公司 用于光遗传疗法的系统和方法
JP6594854B2 (ja) 2013-03-15 2019-10-23 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 行動状態の光遺伝学的制御方法
JP6549559B2 (ja) 2013-04-29 2019-07-24 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 標的細胞における活動電位の光遺伝学的調節のための装置、システム及び方法
JP6621747B2 (ja) 2013-08-14 2019-12-18 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 疼痛を制御するための組成物及び方法
DK3089760T3 (da) 2013-12-31 2020-03-16 Proteonic Biotechnology Ip B V Konstrukt og sekvens til forbedret genekspression
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
EP3800191B1 (de) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Zusammensetzungen und verfahren zur verbesserten genexpression in zapfenzellen
BR112016019940A2 (pt) 2014-03-21 2017-10-24 Univ Leland Stanford Junior edição de genoma sem nucleases
CN106459138A (zh) 2014-03-28 2017-02-22 斯坦福大学托管董事会 工程化的光激活阴离子通道蛋白及其使用方法
EP3151866B1 (de) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimäre kapside
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California アデノ随伴ウイルス変異体及びその使用方法
JP7097070B2 (ja) 2015-10-01 2022-07-07 ゴレイニ インコーポレイテッド クロライドチャネルの標的発現及びその使用方法
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
EP3384034B1 (de) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Neue rekombinante kapside des adeno-associated virus mit erhöhtem tropismus für menschliche skelett- muskulatur
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
JP6947739B2 (ja) 2016-02-16 2021-10-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド
JP7496667B2 (ja) 2016-04-21 2024-06-07 ビロベク,インコーポレイテッド 昆虫細胞中でのaav生成、方法およびその組成物
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (de) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7764129B2 (ja) 2017-07-10 2025-11-05 ユニキュアー アイピー ビー.ブイ. ヒトにおけるaav遺伝子療法のための手段及び方法
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US20200231986A1 (en) 2017-09-29 2020-07-23 Massachusetts Eye And Ear Infirmary Production of adeno-associated viruses in insect cells
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
SI3740222T1 (sl) 2018-01-17 2023-11-30 Meiragtx Uk Ii Limited Modificiran kapsidni protein raav za gensko terapijo
EP3774853A1 (de) 2018-03-30 2021-02-17 The Board of Trustees of the Leland Stanford Junior University Neuartige kapside des rekombinanten adeno-assoziierten virus mit erhöhtem menschlichem pankreatischem tropismus
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
WO2019237375A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 一种将hdmx基因定点整合至mcf-7细胞的方法及其应用
EP3833766A1 (de) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nukleinsäuremoleküle und verwendungen davon zur nichtviralen gentherapie
EP3856762A1 (de) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin-expressionskonstrukte mit gentechnisch veränderten promotoren und verfahren zu deren verwendung
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
WO2020072844A1 (en) * 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020160508A1 (en) 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
CN113728108A (zh) 2019-02-15 2021-11-30 桑格摩生物治疗股份有限公司 用于生产重组aav的组合物和方法
EP3947700A4 (de) 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-assoziiertes virus mit manipuliertem kapsid
CA3136004A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP7664848B2 (ja) 2019-04-10 2025-04-18 プリベイル セラピューティクス,インコーポレーテッド リソソーム障害のための遺伝子療法
MX2022000551A (es) 2019-07-15 2022-05-18 Meiragtx Uk Ii Ltd Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica.
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
JP7598920B2 (ja) 2019-07-23 2024-12-12 ヤンセン バイオテツク,インコーポレーテツド 生物製造のための合成遺伝子要素
EP4069856A1 (de) 2019-12-04 2022-10-12 Sangamo Therapeutics, Inc. Neuartige zusammensetzungen und verfahren zur herstellung von rekombinantem aav
CN111004803B (zh) * 2019-12-31 2023-05-16 中山大学 褐飞虱NlStit基因作为靶点在制备防治褐飞虱药物中的应用
EP4127135A1 (de) 2020-04-02 2023-02-08 uniQure biopharma B.V. Duale bifunktionelle vektoren zur aav-herstellung
EP4127146A1 (de) 2020-04-02 2023-02-08 uniQure biopharma B.V. Neue zelllinie
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
JP2023537903A (ja) 2020-08-10 2023-09-06 プリベイル セラピューティクス,インコーポレーテッド リソソーム障害に対する遺伝子療法
AU2021325891A1 (en) 2020-08-10 2023-04-06 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
BR112023006305A2 (pt) 2020-10-09 2023-05-09 Tenaya Therapeutics Inc Métodos e composições de terapia gênica com placofilina-2
CN112852882A (zh) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法
US20240124850A1 (en) 2021-03-03 2024-04-18 Shape Therapeutics Inc. Auxotrophic Cells for Virus Production and Compositions and Methods of Making
WO2022207899A1 (en) 2021-04-02 2022-10-06 Uniqure Biopharma B.V. Methods for producing single insect cell clones
AU2022286647A1 (en) 2021-06-02 2023-11-30 Uniqure Biopharma B.V. Insect cell production of parvoviral vectors with modified capsid proteins
JP2024521537A (ja) * 2021-06-23 2024-05-31 フロンテーラ セラピューティクス,インク. 血友病bを処置するための組成物および方法
EP4392434A1 (de) 2021-08-26 2024-07-03 uniQure biopharma B.V. Durch insektenzellen produzierte hochwirksame aav-vektoren mit zns-tropismus
CN113897396B (zh) * 2021-09-18 2022-08-30 劲帆生物医药科技(武汉)有限公司 一种用于在昆虫细胞中表达包含重叠开放阅读框的基因的表达盒及其应用
EP4426716A1 (de) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
CN114150021B (zh) 2021-11-26 2022-10-28 劲帆生物医药科技(武汉)有限公司 一种包含重叠开放阅读框的基因的表达盒及其在昆虫细胞中的应用
JP2024545923A (ja) 2021-12-30 2024-12-13 リーゲル セラピューティクス,インコーポレイティッド ナトリウム電位依存性チャネルアルファサブユニット1の発現を調節するための組成物、及びその使用
WO2023201207A1 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CN119403933A (zh) 2022-06-21 2025-02-07 里格尔医疗股份有限公司 遗传调控元件及其用途
WO2024047587A1 (en) 2022-08-31 2024-03-07 Regel Therapeutics, Inc. Cas-phi compositions and methods of use
EP4598940A1 (de) 2022-10-04 2025-08-13 Eli Lilly and Company Gentherapie für trem2-assoziierte erkrankungen und störungen
AU2023375569A1 (en) 2022-11-11 2025-05-29 Eli Lilly And Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
AU2024258816A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Generation of adeno-associated virus capsid libraries for insect cells
WO2024249172A1 (en) 2023-05-26 2024-12-05 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 2 and uses thereof
WO2025025493A1 (zh) * 2023-08-03 2025-02-06 劲帆生物医药科技(武汉)有限公司 一种含有重叠开放阅读框的表达盒及应用
CN117070518A (zh) * 2023-08-03 2023-11-17 劲帆生物医药科技(武汉)有限公司 一种用于表达包含重叠开放阅读框的基因的表达盒及应用
US12416019B2 (en) 2023-08-03 2025-09-16 Genevoyager (WUHAN) CO., LTD. Expression cassette containing overlapping open reading frames and application
WO2025083591A2 (en) 2023-10-17 2025-04-24 Regel Therapeutics, Inc. Genetic regulatory elements for cardiac tissue expression and uses thereof
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions
WO2026043765A1 (en) 2024-08-21 2026-02-26 Eli Lilly And Company Engineered regulatory splice elements for gene therapies

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
JP2001506133A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション 組換えaavベクターの産生における使用のための、aavスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1124976A1 (de) 1998-10-27 2001-08-22 Crucell Holland B.V. Verbesserte produktion von aav-vektoren
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
WO2003042361A2 (en) * 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1588166A4 (de) * 2003-01-09 2007-06-27 Macrogenics Inc Doppelexpressionsvektorsystem für die antikörperexpression in bakteriellen und säugetierzellen
CN1778812A (zh) * 2004-11-24 2006-05-31 国家海洋环境监测中心 重组杆状病毒表达的诺瓦克样病毒衣壳蛋白及其制备方法
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells

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WO2008024998A3 (en) 2008-11-06
US8945918B2 (en) 2015-02-03
EP2061891A2 (de) 2009-05-27
US20140329313A1 (en) 2014-11-06
US20090203071A1 (en) 2009-08-13
US9879282B2 (en) 2018-01-30
DK2061891T3 (da) 2012-07-23
WO2008024998A2 (en) 2008-02-28
ES2385679T3 (es) 2012-07-30
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US9879279B2 (en) 2018-01-30
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