ATE409750T1 - Verbesserter adeno-assoziierter virus (aav)- vektor für gentherapie - Google Patents

Verbesserter adeno-assoziierter virus (aav)- vektor für gentherapie

Info

Publication number
ATE409750T1
ATE409750T1 AT04762994T AT04762994T ATE409750T1 AT E409750 T1 ATE409750 T1 AT E409750T1 AT 04762994 T AT04762994 T AT 04762994T AT 04762994 T AT04762994 T AT 04762994T AT E409750 T1 ATE409750 T1 AT E409750T1
Authority
AT
Austria
Prior art keywords
gene therapy
aav
vector
associated virus
adeno
Prior art date
Application number
AT04762994T
Other languages
English (en)
Inventor
Juergen Kleinschmidt
Oliver Mueller
Original Assignee
Mueller Dr Oliver
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Mueller Dr Oliver filed Critical Mueller Dr Oliver
Application granted granted Critical
Publication of ATE409750T1 publication Critical patent/ATE409750T1/de

Links

Classifications

    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AT04762994T 2003-06-11 2004-06-09 Verbesserter adeno-assoziierter virus (aav)- vektor für gentherapie ATE409750T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP03013169A EP1486567A1 (de) 2003-06-11 2003-06-11 Verbesserter Adeno-assoziierter Virus (AAV)-Vektor für Gentherapie

Publications (1)

Publication Number Publication Date
ATE409750T1 true ATE409750T1 (de) 2008-10-15

Family

ID=33185871

Family Applications (1)

Application Number Title Priority Date Filing Date
AT04762994T ATE409750T1 (de) 2003-06-11 2004-06-09 Verbesserter adeno-assoziierter virus (aav)- vektor für gentherapie

Country Status (5)

Country Link
US (1) US7629322B2 (de)
EP (2) EP1486567A1 (de)
AT (1) ATE409750T1 (de)
DE (1) DE602004016871D1 (de)
WO (1) WO2004111248A2 (de)

Families Citing this family (136)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4969002B2 (ja) 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
JP2006521825A (ja) 2003-03-31 2006-09-28 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ導入を高めるための化合物および方法
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP3085389A1 (de) 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Methode um die funktion eines aav vektors zu erhöhen
CN101495624A (zh) * 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
WO2007127464A2 (en) * 2006-04-28 2007-11-08 University Of Iowa Research Foundation Methods and compounds to modulate parvovirus transduction of mammalian cells or to alter virus infection, method to identify a viral receptor or co-receptor
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
JP2010526528A (ja) 2007-05-03 2010-08-05 メディツィニーチェ ウニベルシタット インスブルック 補体因子ｈ由来ショートコンセンサスリピート−抗体構築物
EP2315833B8 (de) 2008-05-20 2015-05-27 Eos Neuroscience, Inc. Vektoren zur freisetzung lichtempfindlicher proteine und anwendungsverfahren dafür
EP2687609B1 (de) 2008-11-10 2017-01-04 The United States of America, as represented by The Secretary, Department of Health and Human Services Behandlung von soliden Tumoren
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CN107012171B (zh) 2011-04-22 2021-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
KR20140041505A (ko) 2011-04-29 2014-04-04 셀렉타 바이오사이언시즈, 인크. 조절 b 세포 유도를 위한 관용원성 합성 나노운반체
JP6342886B2 (ja) 2012-04-18 2018-06-13 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カプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
WO2013159036A1 (en) * 2012-04-19 2013-10-24 Oregon Health & Science University Adeno associated virus plasmids and vectors
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2872183B1 (de) 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-vermittelte gentherapie zur x-chromosomalen netzhautdegeneration
ES2741502T3 (es) 2013-02-08 2020-02-11 Univ Pennsylvania Cápside de AAV8 modificada para la transferencia génica para terapias de retina
BR112015027291A8 (pt) 2013-05-03 2019-12-24 Selecta Biosciences Inc uso de um imunossupressor que têm uma vida eficaz farmacodinâmica especificada e composição compreendendo o referido imunossupressor
KR102298579B1 (ko) * 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
US10801070B2 (en) 2013-11-25 2020-10-13 The Broad Institute, Inc. Compositions and methods for diagnosing, evaluating and treating cancer
US11725237B2 (en) 2013-12-05 2023-08-15 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
NZ721908A (en) 2013-12-20 2022-12-23 Massachusetts Gen Hospital Combination therapy with neoantigen vaccine
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
ES2768763T3 (es) * 2014-03-04 2020-06-23 Univ Florida Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
EP3800191B1 (de) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Zusammensetzungen und verfahren zur verbesserten genexpression in zapfenzellen
DK3628334T5 (da) 2014-03-21 2024-09-02 Genzyme Corp Genterapi til behandling af retinitis pigmentosa
EP3137497B2 (de) 2014-05-02 2025-03-05 Genzyme Corporation Aav-vektoren zur retinalen und cns-gentherapie
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016037164A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US10975442B2 (en) 2014-12-19 2021-04-13 Massachusetts Institute Of Technology Molecular biomarkers for cancer immunotherapy
EP4636401A2 (de) 2014-12-19 2025-10-22 The Broad Institute, Inc. Verfahren zur profilierung des t-zell-rezeptor-repertoirs
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
JP7095994B2 (ja) 2015-03-02 2022-07-05 アドヴェラム バイオテクノロジーズ， インコーポレイテッド ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
ES2860712T3 (es) 2015-03-24 2021-10-05 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
RU2021132397A (ru) 2015-05-16 2022-02-24 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
WO2017106202A2 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
CA3019423A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Novel aav8 mutant capsids and compositions containing same
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
KR102799528B1 (ko) 2016-07-08 2025-04-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
EP4700118A3 (de) 2016-07-29 2026-04-22 The Regents of the University of California Adenoassoziierte virusvirionen mit veränderlichem kapsid und verfahren zur verwendung davon
SG10201913002QA (en) 2016-08-15 2020-03-30 Genzyme Corp Methods for detecting aav
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018129268A1 (en) 2017-01-07 2018-07-12 Selecta Biosciences, Inc. Patterned dosing of immunosuppressants coupled to synthetic nanocarriers
WO2018132747A1 (en) 2017-01-13 2018-07-19 University Of Iowa Research Foundation Bocaparvovirus small noncoding rna and uses thereof
CA3114549A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
KR20230093072A (ko) 2017-03-01 2023-06-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 안구 장애에 대한 유전자 치료
KR102753928B1 (ko) 2017-03-11 2025-01-14 셀렉타 바이오사이언시즈, 인크. 항염증제, 및 면역억제제를 포함하는 합성 나노담체를 사용한 조합 치료와 관련된 방법 및 조성물
BR112019019813A2 (pt) 2017-03-30 2020-04-22 The University Of Queensland moléculas quiméricas e usos das mesmas
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
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
BR112019023303A2 (pt) 2017-05-11 2020-06-16 The Trustees Of The University Of Pennsylvania Terapia de gene para lipofuscinoses ceroides neuronais
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3645052A4 (de) 2017-06-30 2021-07-28 Regents of the University of California Adenoassoziierte virus-virionen mit kapsidvarianten und verfahren zur verwendung davon
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
EP3687561A4 (de) 2017-09-01 2021-06-09 The Australian National University Immunregulatorische moleküle und deren verwendung
PE20201264A1 (es) 2017-09-20 2020-11-19 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas
EP3461836A1 (de) 2017-09-28 2019-04-03 Universität zu Köln Mutierte kapsid proteine von adeno-assoziierten viren zur chemischen kopplung von liganden, nanopartikeln oder medikamenten über thioetherbindung und methode zu deren herstellung
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
MX2020003838A (es) 2017-10-13 2020-08-06 Selecta Biosciences Inc Metodos y composiciones para atenuar las respuestas de inmunoglobulina m del vector de transferencia antiviral.
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (de) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose (als)
ES2946747T3 (es) 2017-11-27 2023-07-25 4D Molecular Therapeutics Inc Variantes de cápsides de virus adenoasociado y su utilización para inhibir la angiogénesis
BR112020014404A2 (pt) 2018-01-17 2020-12-29 Adrenas Therapeutics, Inc. Terapia gênica de vírus adenoassociado para deficiência de 21-hidroxilase
KR20200139679A (ko) 2018-02-26 2020-12-14 안톨알엑스, 인크. 면역관용성 리포솜 및 그의 사용 방법
US12404312B2 (en) 2018-03-05 2025-09-02 Massachusetts Eye And Ear Infirmary Engineered VEGF variants for retinal neuroprotection, promotion of axon growth and axon regeneration
US12338267B2 (en) 2018-05-15 2025-06-24 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
EP3794126A1 (de) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von morbus parkinson
EP3823676A1 (de) 2018-07-16 2021-05-26 Selecta Biosciences, Inc. Verfahren und zusammensetzungen von mma-konstrukten und vektoren
JP2021531282A (ja) 2018-07-16 2021-11-18 セレクタ バイオサイエンシーズ インコーポレーテッドSelecta Biosciences, Inc. Ｏｔｃコンストラクトおよびベクターの方法および組成物
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
JP7637060B2 (ja) 2019-03-04 2025-02-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ａｋｔ経路を標的とする神経保護遺伝子療法
BR112021021566A2 (pt) 2019-04-28 2022-04-19 Selecta Biosciences Inc Métodos para tratamento de indivíduos com imunidade preexistente a vetores de transferência viral
MX2021013266A (es) 2019-04-29 2021-11-17 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen.
EP3736330A1 (de) 2019-05-08 2020-11-11 European Molecular Biology Laboratory Partikel mit modifiziertem adeno-assoziiertem virus (aav) zur gentherapie
BR112021023594A2 (pt) 2019-05-28 2022-02-08 Selecta Biosciences Inc Métodos e composições para resposta imune de vetor de transferência antiviral atenuada
EP3980784A1 (de) 2019-06-04 2022-04-13 Selecta Biosciences, Inc. Formulierungen und dosen von pegylierter uricase
WO2021000024A1 (en) * 2019-07-04 2021-01-07 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
GB2606844B (en) 2019-09-18 2025-08-06 Aldevron Llc Synthetic DNA vectors and methods of use
EP4045093A4 (de) 2019-10-14 2024-02-07 Duke University Zusammensetzungen mit neuen prokaryotischen natriumkanälen und zugehörige verfahren
US12617821B2 (en) 2019-11-08 2026-05-05 President And Fellows Of Harvard College; Engineered viral capsid polypeptides and uses thereof
EP4055030A4 (de) 2019-11-08 2024-05-22 President And Fellows Of Harvard College Virale kapsidpolypeptide
WO2021207500A2 (en) * 2020-04-08 2021-10-14 President And Fellows Of Harvard College TGFβ THERAPY FOR OCULAR AND NEURODEGENERATIVE DISEASES
TW202221127A (zh) * 2020-08-14 2022-06-01 賓州大學委員會 新穎aav衣殼及含有其之組成物
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
CA3191543A1 (en) * 2020-09-04 2022-03-10 Lei Cao Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
WO2022076556A2 (en) 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)
CA3196499A1 (en) 2020-10-29 2022-05-05 James M. Wilson Aav capsids and compositions containing same
AU2022206197A1 (en) 2021-01-05 2023-07-13 Selecta Biosciences, Inc. Viral vector dosing protocols
EP4284335A1 (de) 2021-02-01 2023-12-06 RegenxBio Inc. Gentherapie für neuronale ceroid-lipofuscinosen
AR125143A1 (es) 2021-03-19 2023-06-14 Adrenas Therapeutics Inc Terapias génicas para la deficiencia de la 21-hidroxilasa
AR125467A1 (es) 2021-04-27 2023-07-19 Univ Pennsylvania Cápsides de virus adenoasociados derivados de porcinos y usos de los estos
EP4415746A1 (de) 2021-10-12 2024-08-21 Bridgebio Gene Therapy LLC Verfahren und zusammensetzungen zur behandlung von leukodystrophien
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
JP2024539646A (ja) 2021-10-12 2024-10-29 カーティザン セラピューティクス，インコーポレーテッド ウイルスベクター投薬プロトコル
WO2023086615A1 (en) 2021-11-14 2023-05-19 Selecta Biosciences, Inc. Multiple dosing with viral vectors
WO2023156530A1 (en) 2022-02-17 2023-08-24 Lysogene Gene therapy for neurodegenerative diseases
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
CA3264659A1 (en) 2022-08-11 2024-02-15 Cartesian Therapeutics, Inc. COMPOSITIONS AND PROCESSES ASSOCIATED WITH IMMUNOGLOBULIN PROTEASES AND FUSION THEREOF
CN117402222B (zh) * 2023-11-17 2024-03-29 广州派真生物技术有限公司 腺相关病毒突变体及其应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
ES2467156T3 (es) * 2001-12-21 2014-06-12 Medigene Ag Una biblioteca de genes estructurales modificados o de partículas modificadas de cápside útiles para la identificación de clones virales con tropismo celular deseado
US8802080B2 (en) * 2002-05-01 2014-08-12 University Of Florida Research Foundation, Inc. Raav expression systems for genetic modification of specific capsid proteins

Also Published As

Publication number Publication date
EP1664314A2 (de) 2006-06-07
US7629322B2 (en) 2009-12-08
EP1664314B1 (de) 2008-10-01
WO2004111248A3 (en) 2005-07-07
DE602004016871D1 (de) 2008-11-13
US20070036757A1 (en) 2007-02-15
EP1486567A1 (de) 2004-12-15
WO2004111248A2 (en) 2004-12-23

Similar Documents

Publication Publication Date Title
DE602004016871D1 (de) Für gentherapie
CY2021024I2 (el) Ομαδες, αλληλουχιες, φορεις αδενο-σχετιζομενου ιου (aav) που περιεχουν αυτον, και οι χρησεις τους
WO2001011034A3 (en) Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
WO2004027019A3 (en) Improved raav expression systems for genetic modification of specific capsid proteins
IL221602A (en) Virus sequences belonging to the rh10 adenoid type and recombinant viruses containing them
CY1119279T1 (el) Ιοσωματια aav με μειωμενη ανοσοαντιδραστικοτητα και χρησεις αυτων
DE60233955D1 (de) Spezifische bindungsmittel von human angiopoietin-2
WO2002053703A3 (en) Aav2 vectors and methods
WO2007120542A3 (en) Aav capsid library and aav capsid proteins
EP2105449A3 (de) Antagonistische selektive Bindemittel eines Osteoprotegerin-bindenden Proteins
ZA202202242B (en) Isolated modified vp1 capsid protein of aav5
IL199126A (en) Promoters for expression in modified vaccinia virus ankara (mva) and vectors comprising said promoters.
EP1546343A4 (de) Produktion von rekombinantem aav mit hohem titer
AU2002325683A1 (en) Recombinant vector derived from adeno-associated virus for gene therapy
WO2005049850A3 (en) Compositions and methods for systemic nucleic acid sequence delivery
DE69840948D1 (de) Invertierte terminale repeats von aav, die für die transkription aktiviert sind, zur verwendung in rekombinanten aav-vektoren
EA200600042A1 (ru) Способы образования дисульфидных связей и гликозилирования белков и реагенты, применяемые в этих способах
WO2023194796A3 (en) Recombinant aav capsid proteins
WO2002002148A3 (en) Dual recombinant gene therapy compositions and methods of use
DE60229229D1 (de) Teilweises laden von klassendateien in virtuelle maschinen
DE60017924D1 (de) Rekombinante, adenovirale vektoren und ihre verwendung zur behandlung von leberzirrhose
WO2004087886A3 (en) Modified adenoviral e1a constructs and methods of use thereof
DE60038955D1 (de) AN DER OBERFLÄCHE VON KARZINOMZELLEN LOKALISIERTES COLLIGIN/HsP47
WO2023242633A3 (en) Recombinant aav capsid proteins
WO2024064782A3 (en) Adeno-associated virus capsids

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties