BR9914643A - Vetores adeno-associados para expressão dofator viii por células alvo - Google Patents
Vetores adeno-associados para expressão dofator viii por células alvoInfo
- Publication number
- BR9914643A BR9914643A BR9914643-6A BR9914643A BR9914643A BR 9914643 A BR9914643 A BR 9914643A BR 9914643 A BR9914643 A BR 9914643A BR 9914643 A BR9914643 A BR 9914643A
- Authority
- BR
- Brazil
- Prior art keywords
- vectors
- adeno
- target cells
- present
- associated vectors
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 4
- 239000013607 AAV vector Substances 0.000 abstract 1
- 108010054218 Factor VIII Proteins 0.000 abstract 1
- 102000001690 Factor VIII Human genes 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 229960000301 factor viii Drugs 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 210000005260 human cell Anatomy 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US10499498P | 1998-10-20 | 1998-10-20 | |
| US12597499P | 1999-03-24 | 1999-03-24 | |
| US09/364,862 US6221349B1 (en) | 1998-10-20 | 1999-07-30 | Adeno-associated vectors for expression of factor VIII by target cells |
| PCT/US1999/024495 WO2000023116A1 (en) | 1998-10-20 | 1999-10-19 | Adeno-associated vectors for expression of factor viii by target cells |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR9914643A true BR9914643A (pt) | 2001-12-11 |
Family
ID=27379851
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR9914643-6A BR9914643A (pt) | 1998-10-20 | 1999-10-19 | Vetores adeno-associados para expressão dofator viii por células alvo |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US6221349B1 (https=) |
| EP (1) | EP1124583A4 (https=) |
| JP (1) | JP2002527493A (https=) |
| KR (1) | KR20010082234A (https=) |
| CN (1) | CN1329509A (https=) |
| AU (1) | AU757688B2 (https=) |
| BR (1) | BR9914643A (https=) |
| CA (1) | CA2348068A1 (https=) |
| IL (1) | IL142721A0 (https=) |
| MX (1) | MXPA01004067A (https=) |
| WO (1) | WO2000023116A1 (https=) |
Families Citing this family (89)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6436392B1 (en) * | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| US6797505B2 (en) * | 1998-05-27 | 2004-09-28 | Cell Genesys, Inc. | Recombinant AAV vectors for gene therapy of hemophilia A |
| EP1082413B1 (en) * | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| US6200560B1 (en) * | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US7122335B1 (en) | 1999-06-08 | 2006-10-17 | University Of Iowa Research Foundation | Compounds and methods to enhance rAAV transduction |
| 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 |
| US7241447B1 (en) * | 1999-10-07 | 2007-07-10 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| AU7879000A (en) * | 1999-10-12 | 2001-04-23 | University Of North Carolina At Chapel Hill, The | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| AU1775901A (en) * | 1999-11-17 | 2001-05-30 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| CA2400634A1 (en) * | 2000-02-17 | 2001-08-23 | Chester Li | Genetic modification of the lung as a portal for gene delivery |
| US6855314B1 (en) * | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| US6593123B1 (en) * | 2000-08-07 | 2003-07-15 | Avigen, Inc. | Large-scale recombinant adeno-associated virus (rAAV) production and purification |
| WO2002055110A2 (en) * | 2000-10-25 | 2002-07-18 | Genzyme Corp | Methods for treating blood coagulation disorders |
| US20020159978A1 (en) * | 2001-02-06 | 2002-10-31 | James Allen | Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions |
| WO2002071843A1 (en) * | 2001-03-14 | 2002-09-19 | Genteric, Inc. | Recombinant adeno-assocaited virus-mediated gene transfer via retroductal infusion of virions |
| CN1520259A (zh) * | 2001-04-25 | 2004-08-11 | ���ݴ�ѧУ��ίԱ�� | 利用球状载体将第八因子/第九因子进行肝脏基因转移治疗血友病的方法 |
| ATE437221T1 (de) * | 2001-05-14 | 2009-08-15 | Gbp Ip Llc | Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie |
| US8241622B2 (en) * | 2001-07-13 | 2012-08-14 | University Of Iowa Research Foundation | Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences |
| EP1418810A4 (en) * | 2001-08-03 | 2006-08-02 | Us Gov Health & Human Serv | TREATMENT OF HEMOPHILIA ORAL |
| PT1567641E (pt) | 2001-08-24 | 2012-08-21 | Uvic Industry Partnerships Inc | Proaerolisina contendo sequência de activação de proteases e métodos de uso para o tratamento do cancro da próstata |
| US20030235555A1 (en) * | 2002-04-05 | 2003-12-25 | David Shealey | Asthma-related anti-IL-13 immunoglobulin derived proteins, compositions, methods and uses |
| US7419817B2 (en) * | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
| KR100535325B1 (ko) * | 2002-09-24 | 2005-12-09 | 한국생명공학연구원 | Aav 유전자 전달체 제조용 헬퍼 플라스미드 |
| US20040092008A1 (en) * | 2002-11-12 | 2004-05-13 | Snyder Richard O. | Recombinant AAV vectors for gene therapy of hemophilia A |
| JP2007524379A (ja) * | 2003-03-31 | 2007-08-30 | 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 | 上皮ナトリウムチャンネル関連障害のPharmico−遺伝子治療 |
| US7963956B2 (en) * | 2003-04-22 | 2011-06-21 | Antisense Pharma Gmbh | Portable equipment for administration of fluids into tissues and tumors by convection enhanced delivery technique |
| WO2005017101A2 (en) | 2003-05-19 | 2005-02-24 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH | Avian adenoassociated virus (aaav) and uses thereof |
| WO2005017149A1 (en) * | 2003-06-03 | 2005-02-24 | Cell Genesys, Inc. | Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site |
| US7485291B2 (en) * | 2003-06-03 | 2009-02-03 | Cell Genesys, Inc. | Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof |
| WO2004111246A1 (en) * | 2003-06-19 | 2004-12-23 | Peter Bromley | Protein production system |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2005056807A2 (en) | 2003-12-04 | 2005-06-23 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health | Bovine adeno-associated viral (baav) vector and uses thereof |
| WO2006029196A1 (en) * | 2004-09-08 | 2006-03-16 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of H Ealth | Transcytosis of adeno-associated viruses |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| CA2604299A1 (en) * | 2005-04-14 | 2006-10-19 | Csl Behring Gmbh | Modified coagulation factor viii with enhanced stability and its derivates |
| US8283151B2 (en) * | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| EP4154903A1 (en) * | 2008-04-22 | 2023-03-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| CN103215308B (zh) * | 2013-02-01 | 2015-12-23 | 中国科学院苏州生物医学工程技术研究所 | 表达重组人fviii的整合质粒、细胞株及其构建方法和应用 |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| RS61039B1 (sr) | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| CN104087613B (zh) * | 2014-06-30 | 2017-08-29 | 中国科学院苏州生物医学工程技术研究所 | 基于aav‑itr的基因表达微载体及其构建方法和应用 |
| DK3193911T5 (da) * | 2014-07-25 | 2024-10-14 | Csl Behring Gmbh | Forbedrede faktor VII-præparater til brug ved behandling af hæmofili A |
| CA3237630A1 (en) * | 2014-08-13 | 2016-02-18 | The Children's Hospital Of Philadelphia | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders |
| SG11201703148TA (en) * | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| CN115074366A (zh) * | 2015-04-16 | 2022-09-20 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| EP3332012A4 (en) * | 2015-08-06 | 2019-01-16 | The Trustees of The University of Pennsylvania | GLP-1 AND USE THEREOF IN COMPOSITIONS FOR THE TREATMENT OF METABOLISM DISEASES |
| RU2742352C2 (ru) | 2015-09-24 | 2021-02-05 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| CA3002524A1 (en) | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| EA202092049A1 (ru) * | 2015-11-13 | 2020-11-10 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| EP3374387A1 (en) * | 2015-11-13 | 2018-09-19 | Baxalta Incorporated | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| US10385320B2 (en) | 2015-12-02 | 2019-08-20 | The Board Of Trustees Of The Leland Stanford Junior University | Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| WO2017139381A1 (en) | 2016-02-08 | 2017-08-17 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US10179176B2 (en) | 2016-02-16 | 2019-01-15 | The Board Of Trustees Of The Leland Stanford Junior University | Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies |
| EP3210632B1 (en) * | 2016-02-23 | 2022-09-28 | EyeServ GmbH | Gene therapy for the treatment of a retinal degeneration disease |
| CA3198936A1 (en) | 2016-03-07 | 2017-09-14 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| KR102450833B1 (ko) | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
| 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 |
| US11142775B2 (en) | 2017-01-13 | 2021-10-12 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | 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 | 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途 |
| JOP20190269A1 (ar) * | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| 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 |
| TW202545984A (zh) * | 2017-08-09 | 2025-12-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| MX2020008932A (es) | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| US11608510B2 (en) | 2018-03-30 | 2023-03-21 | The Board Of Trustees Of The Leland Stanford Junior University | Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| SG11202011296VA (en) | 2018-05-15 | 2020-12-30 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease |
| CN118638832A (zh) * | 2018-05-15 | 2024-09-13 | 杜克大学 | 用于对阿克曼菌进行基因操纵的系统和方法 |
| WO2020033863A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| JP7603013B2 (ja) * | 2019-02-20 | 2024-12-19 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | がんを標的とする、ウイルスによってコードされる、調節可能なt(catvert)またはnk細胞(catvern)リンカー |
| PE20220014A1 (es) | 2019-04-29 | 2022-01-11 | Univ Pennsylvania | Nuevas capsides de aav y composiciones que las contienen |
| AR118928A1 (es) * | 2019-05-14 | 2021-11-10 | Biomarin Pharm Inc | Métodos de redosificación de vectores de terapia génica |
| KR20220031000A (ko) * | 2019-07-03 | 2022-03-11 | 각꼬호우징 닛뽄 이까다이가꾸 | 핵산 봉입aav 중공입자의 제조방법 |
| CN111876400B (zh) * | 2020-08-06 | 2022-05-24 | 昆明理工大学 | 一种常温裂解酶Sly和编码此酶的多核苷酸 |
| JP2023537070A (ja) | 2020-08-07 | 2023-08-30 | アミカス セラピューティックス インコーポレイテッド | 小胞を標的とするタンパク質及びその使用 |
| IL300410A (en) | 2020-08-14 | 2023-04-01 | Univ Pennsylvania | Novel AAV capsids and compositions containing them |
| IL302127A (en) | 2020-10-18 | 2023-06-01 | Univ Pennsylvania | An improved adeno-associated virus (AAV) vector and its uses |
| AU2021369793A1 (en) | 2020-10-29 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Aav capsids and compositions containing same |
| TW202309066A (zh) | 2021-04-27 | 2023-03-01 | 賓州大學委員會 | 衍生自豬的腺相關病毒衣殼及其用途 |
| JP2024524270A (ja) | 2021-06-23 | 2024-07-05 | アスクバイオ・ユーケー・リミテッド | 調節性核酸配列 |
| WO2024015966A2 (en) | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| JP2025542309A (ja) | 2022-12-20 | 2025-12-25 | ナノセル セラピューティクス ホールディングス ビー.ブイ. | 組み込み型ウイルスベクターおよび自己不活性化ウイルスベクターならびにその構築物および使用 |
| EP4389899A1 (en) | 2022-12-22 | 2024-06-26 | Synpromics Ltd. | Regulatory nucleic acid sequences |
| WO2025239324A1 (ja) * | 2024-05-13 | 2025-11-20 | 中外製薬株式会社 | 遺伝子改変非ヒト動物 |
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1999
- 1999-07-30 US US09/364,862 patent/US6221349B1/en not_active Expired - Lifetime
- 1999-10-19 EP EP99955065A patent/EP1124583A4/en not_active Withdrawn
- 1999-10-19 AU AU11256/00A patent/AU757688B2/en not_active Expired
- 1999-10-19 KR KR1020017004962A patent/KR20010082234A/ko not_active Ceased
- 1999-10-19 IL IL14272199A patent/IL142721A0/xx unknown
- 1999-10-19 CN CN99814128A patent/CN1329509A/zh active Pending
- 1999-10-19 CA CA002348068A patent/CA2348068A1/en not_active Abandoned
- 1999-10-19 MX MXPA01004067A patent/MXPA01004067A/es not_active Application Discontinuation
- 1999-10-19 WO PCT/US1999/024495 patent/WO2000023116A1/en not_active Ceased
- 1999-10-19 JP JP2000576889A patent/JP2002527493A/ja active Pending
- 1999-10-19 BR BR9914643-6A patent/BR9914643A/pt not_active Application Discontinuation
Also Published As
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| AU1125600A (en) | 2000-05-08 |
| MXPA01004067A (es) | 2003-03-10 |
| EP1124583A1 (en) | 2001-08-22 |
| IL142721A0 (en) | 2002-03-10 |
| EP1124583A4 (en) | 2002-04-17 |
| JP2002527493A (ja) | 2002-08-27 |
| CA2348068A1 (en) | 2000-04-27 |
| AU757688B2 (en) | 2003-03-06 |
| KR20010082234A (ko) | 2001-08-29 |
| CN1329509A (zh) | 2002-01-02 |
| US6221349B1 (en) | 2001-04-24 |
| WO2000023116A1 (en) | 2000-04-27 |
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