DE60039766D1 - Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen - Google Patents

Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen

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Publication number
DE60039766D1
DE60039766D1 DE60039766T DE60039766T DE60039766D1 DE 60039766 D1 DE60039766 D1 DE 60039766D1 DE 60039766 T DE60039766 T DE 60039766T DE 60039766 T DE60039766 T DE 60039766T DE 60039766 D1 DE60039766 D1 DE 60039766D1
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Prior art keywords
sequence
raav vector
raav
heterologous sequence
heterologous
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Expired - Lifetime
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DE60039766T
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English (en)
Inventor
Barrie Carter
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Ampliphi Biosciences Corp
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Targeted Genetics Corp
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/191Tumor necrosis factors [TNF], e.g. lymphotoxin [LT], i.e. TNF-beta
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70578NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00Preparation of peptides or proteins
    • C12P21/06Preparation of peptides or proteins produced by the hydrolysis of a peptide bond, e.g. hydrolysate products
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
DE60039766T 1999-08-09 2000-08-08 Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen Expired - Lifetime DE60039766D1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US16008099P 1999-08-09 1999-08-09
PCT/US2000/021693 WO2001011034A2 (en) 1999-08-09 2000-08-08 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

Publications (1)

Publication Number Publication Date
DE60039766D1 true DE60039766D1 (de) 2008-09-18

Family

ID=22575432

Family Applications (1)

Application Number Title Priority Date Filing Date
DE60039766T Expired - Lifetime DE60039766D1 (de) 1999-08-09 2000-08-08 Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen

Country Status (12)

Country Link
US (8) US6596535B1 (de)
EP (2) EP1204739B1 (de)
JP (2) JP4827353B2 (de)
AT (1) ATE403715T1 (de)
AU (1) AU781958C (de)
CA (1) CA2379166C (de)
CY (1) CY1107432T1 (de)
DE (1) DE60039766D1 (de)
DK (2) DK1204739T3 (de)
ES (2) ES2308989T3 (de)
PT (2) PT1204739E (de)
WO (1) WO2001011034A2 (de)

Families Citing this family (238)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6924128B2 (en) * 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6989264B2 (en) * 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6893865B1 (en) * 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
US6596535B1 (en) * 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
WO2001092551A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20040023390A1 (en) * 2002-08-05 2004-02-05 Davidson Beverly L. SiRNA-mediated gene silencing with viral vectors
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US8529885B2 (en) 2003-09-01 2013-09-10 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
PT1664315E (pt) * 2003-09-01 2012-07-31 Inst Nat Sante Rech Med Vectores aav para tratamento genético in vivo de artrite reumatóide
FR2874384B1 (fr) * 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
KR20070100241A (ko) * 2004-10-28 2007-10-10 유니버시티 오브 피츠버그 오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 척수 외상 통증을 위한 말초로 전달된 글루탐산데카르복실라제 유전자 치료법
WO2007149115A1 (en) * 2006-06-15 2007-12-27 Targeted Genetics Corporation Methods for treating target joints in inflammatory arthritis using aav vectors encoding a tnf antagonist
US8192441B2 (en) * 2008-10-03 2012-06-05 Howmedica Osteonics Corp. High tibial osteotomy instrumentation
HRP20212024T1 (hr) 2009-05-02 2022-04-01 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
MX342858B (es) 2010-03-29 2016-10-13 The Trustees Of The Univ Of Pennsylvania * Sistema de ablacion transgenica inducida farmacologicamente.
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
WO2012061744A2 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Stabilized step function opsin proteins and methods of using the same
JP6355335B2 (ja) 2010-11-05 2018-07-11 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 報酬関連行動の光遺伝学的制御
JP6328424B6 (ja) 2010-11-05 2018-07-11 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 記憶機能の制御および特性化
CN106947741A (zh) 2010-11-05 2017-07-14 斯坦福大学托管董事会 光活化嵌合视蛋白及其使用方法
MX2013009472A (es) 2011-02-15 2013-10-28 Immune Design Corp Metodos para mejorar respuestas inmunitarias especificas de inmunogenos por vacunas vectoriales.
US9044420B2 (en) 2011-04-08 2015-06-02 Immune Design Corp. Immunogenic compositions and methods of using the compositions for inducing humoral and cellular immune responses
WO2012142526A1 (en) 2011-04-14 2012-10-18 Modiano Jaime Use of tumor fas expression to determine response to anti-cancer therapy
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
WO2013090356A2 (en) 2011-12-16 2013-06-20 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
ES2728077T3 (es) 2012-02-21 2019-10-22 Univ Leland Stanford Junior Composiciones para el tratamiento de trastornos neurogénicos del suelo pélvico
CN107540730B (zh) 2012-05-16 2021-07-16 免疫设计公司 用于hsv-2的疫苗
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
US20150284472A1 (en) 2012-11-05 2015-10-08 Genzyme Corporation Compositions and methods for treating proteinopathies
EP2922476B1 (de) 2012-11-21 2019-10-09 Circuit Therapeutics, Inc. System für optogenetische therapie
BR112015022208A8 (pt) 2013-03-13 2018-01-23 Genzyme Corp proteínas de fusão que compreendem pdgf e partes de ligação de vegf, e métodos de utilização do mesmo
SI2984166T1 (sl) 2013-03-15 2020-09-30 The Trustees Of The University Of Pennsylvania Sestavki za zdravljenje MPSI
WO2014144409A1 (en) 2013-03-15 2014-09-18 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic control of behavioral state
WO2014172560A1 (en) 2013-04-17 2014-10-23 Genzyme Corporation Compositions and methods for treating and preventing macular degeneration
US10220092B2 (en) 2013-04-29 2019-03-05 The Board Of Trustees Of The Leland Stanford Junior University Devices, systems and methods for optogenetic modulation of action potentials in target cells
US10821154B2 (en) 2013-05-01 2020-11-03 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
US10307609B2 (en) 2013-08-14 2019-06-04 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for controlling pain
SG10201804030WA (en) 2013-10-25 2018-07-30 Psioxus Therapeutics Ltd Oncolytic adenoviruses armed with heterologous genes
US20170007719A1 (en) 2014-02-06 2017-01-12 Genzyme Corporation Compositions and methods for treating and preventing macular degeneration
WO2015123496A1 (en) 2014-02-14 2015-08-20 Immune Design Corp. Immunotherapy of cancer through combination of local and systemic immune stimulation
BR122023023004A2 (pt) 2014-03-09 2023-12-26 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adeno-associado recombinante, composição farmacêutica, bem como uso dos mesmos
KR102130877B1 (ko) * 2014-03-20 2020-07-08 얀마 파워 테크놀로지 가부시키가이샤 엔진 장치 및 이것을 탑재한 정치형 작업기
EP3628334B1 (de) 2014-03-21 2023-06-28 Genzyme Corporation Gentherapie für retinitis pigmentosa
EP3581580A1 (de) 2014-03-28 2019-12-18 The Board of Trustees of the Leland Stanford Junior University Manipulierte lichtaktivierte anionenkanalproteine und verfahren zur verwendung davon
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
ES2876409T3 (es) 2014-04-25 2021-11-12 Univ Pennsylvania Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
MX2016014220A (es) 2014-05-02 2017-02-06 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc.
PT3142750T (pt) 2014-05-13 2020-09-22 Univ Pennsylvania Composições que compreendem aav expressando construções de anticorpos duplos e suas utilizações
MX2017000630A (es) 2014-07-15 2017-04-27 Immune Design Corp Regimenes de sensibilización-refuerzo con un adyuvante de agonista tlr4 y un vector lentiviral.
CA2960912A1 (en) 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP4012035A1 (de) 2014-09-16 2022-06-15 Genzyme Corporation Adeno-assoziierte virale vektoren zur behandlung von myocilin (myoc)-glaukom
WO2016044478A1 (en) 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
EP3978614A3 (de) 2015-01-07 2022-07-27 Universitat Autònoma de Barcelona Einzelvektor-genkonstrukt mit insulin und glucokinasegenen
EP3247793A1 (de) 2015-01-20 2017-11-29 Genzyme Corporation Analytische ultrazentrifugation zur charakterisierung von rekombinanten viruspartikeln
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
MX2017010370A (es) 2015-02-10 2017-12-14 Genzyme Corp Suministro mejorado de particulas virales al cuerpo estriado y al cortex.
SG10201907396XA (en) 2015-02-10 2019-09-27 Genzyme Corp VARIANT RNAi
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
WO2016176191A1 (en) 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
MA48852A (fr) 2015-05-13 2020-04-01 Univ Pennsylvania Expression médiée par aav d'anticorps anti-grippaux et leurs procédés d'utilisation
RU2021132397A (ru) 2015-05-16 2022-02-24 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
EP4345455A2 (de) 2015-08-25 2024-04-03 Duke University Zusammensetzungen und verfahren zur verbesserung der spezifität in der genomischen manipulation unter verwendung von rna-geführten endonukleasen
US11117942B2 (en) 2015-08-31 2021-09-14 The Trustees Of The University Of Pennsylvania AAV-EPO for treating companion animals
JP7261583B2 (ja) 2015-09-24 2023-04-20 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 補体媒介性疾患を処置するための組成物及び方法
IL292830B2 (en) 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors
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
US11905526B2 (en) 2015-10-13 2024-02-20 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
CA3003435A1 (en) 2015-10-28 2017-05-04 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
MA43570A (fr) 2015-12-11 2018-11-14 Univ Pennsylvania Thérapie génique pour traiter l'hypercholestérolémie familiale
EP3389723A1 (de) 2015-12-14 2018-10-24 The Trustees Of The University Of Pennsylvania Zusammensetzung zur behandlung von crigler-najjar-syndrom
ES2826384T3 (es) 2015-12-14 2021-05-18 Univ Pennsylvania Terapia génica para trastornos oculares
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
JP2019504888A (ja) 2016-02-05 2019-02-21 エモリー ユニバーシティ 一本鎖アデノ随伴ウイルス9または自己相補型アデノ随伴ウイルス9の脳脊髄液への注射
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
MA43735A (fr) 2016-03-07 2018-11-28 Univ Iowa Res Found Expression médiée par aav utilisant un promoteur et un activateur synthétiques
CA3019426A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
MA43551A (fr) 2016-04-15 2018-11-07 Alpine Immune Sciences Inc Protéines immunomodulatrices à variants du ligand icos et leurs utilisations
EP3442597A1 (de) 2016-04-15 2019-02-20 The Trustees Of The University Of Pennsylvania Gentherapie zur behandlung von hämophilie b
KR20230125339A (ko) 2016-04-15 2023-08-29 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
IL262366B1 (en) 2016-04-15 2024-03-01 Alpine Immune Sciences Inc Immunomodulatory proteins and CD80 variants and their uses
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
IL264070B2 (en) 2016-07-08 2023-03-01 Univ Pennsylvania Methods and preparations for the treatment of disorders and diseases involving rdh12
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
EP3491013A1 (de) 2016-07-28 2019-06-05 Alpine Immune Sciences, Inc. Cd155-variante immunmodulatorische proteine und verwendungen davon
WO2018022945A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd112 variant immunomodulatory proteins and uses thereof
CN110168080A (zh) 2016-08-15 2019-08-23 建新公司 检测aav的方法
GB201713765D0 (en) 2017-08-28 2017-10-11 Psioxus Therapeutics Ltd Modified adenovirus
AU2018220212A1 (en) 2017-02-20 2019-08-08 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
KR20190132639A (ko) 2017-02-28 2019-11-28 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-연관 바이러스(aav) 클레이드 f 벡터 및 이의 용도
UY37620A (es) 2017-02-28 2018-08-31 Univ Pennsylvania Vacunas contra la gripe mediadas por aav novedosas
JP7211960B2 (ja) 2017-03-01 2023-01-24 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 眼疾患の遺伝子治療
CA3053812A1 (en) 2017-03-16 2018-09-20 Alpine Immune Sciences, Inc. Pd-l2 variant immunomodulatory proteins and uses thereof
KR20190140924A (ko) 2017-03-16 2019-12-20 알파인 이뮨 사이언시즈, 인코포레이티드 Cd80 변이체 면역조절 단백질 및 그의 용도
EP4306537A3 (de) 2017-03-16 2024-03-06 Alpine Immune Sciences, Inc. Pd-l1-variante immunmodulatorische proteine und verwendungen davon
SG11201908280SA (en) 2017-03-30 2019-10-30 Univ Queensland "chimeric molecules and uses thereof"
SG11201909361SA (en) 2017-04-14 2019-11-28 Regenxbio Inc Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells
EP3634986A4 (de) 2017-04-24 2021-09-08 The Trustees of The University of Pennsylvania Gentherapie für augenerkrankungen
SG11201910015SA (en) 2017-05-11 2019-11-28 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
WO2018220207A1 (en) * 2017-06-01 2018-12-06 Psioxus Therapeutics Limited Oncolytic virus and method
WO2018232149A1 (en) 2017-06-14 2018-12-20 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
KR20200053468A (ko) 2017-07-06 2020-05-18 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 I형 점액다당류증을 치료하기 위한 유전자 요법
MX2020003095A (es) 2017-09-22 2020-10-15 Genzyme Corp Arni variante.
AU2018335752A1 (en) 2017-09-22 2020-03-12 Christian HINDERER Gene therapy for treating Mucopolysaccharidosis type ii
EP4219540A3 (de) 2017-10-10 2023-12-06 Alpine Immune Sciences, Inc. Ctla-4-variante immunmodulatorische proteine und verwendungen davon
CN111712515A (zh) 2017-10-18 2020-09-25 高山免疫科学股份有限公司 变体icos配体免疫调节蛋白及相关组合物和方法
WO2019079494A1 (en) 2017-10-18 2019-04-25 Regenxbio, Inc. TREATMENT OF OCULAR DISEASES AND METASTATIC COLON CANCER WITH A VEGF TRAP WITH HUMAN POST-TRANSLATIONAL MODIFICATION
AU2018350992A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
KR20200104307A (ko) 2017-11-30 2020-09-03 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 뮤코다당류증 iiia형에 대한 유전자 요법
CA3083761A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iiib
US20230101432A1 (en) 2018-01-03 2023-03-30 Alpine Immune Sciences, Inc. Multi-domain immunomodulatory proteins and methods of use thereof
EP3788165A1 (de) 2018-04-29 2021-03-10 REGENXBIO Inc. Systeme und verfahren zur spektralfotometrie zur bestimmung des genomgehalts, des kapsidgehalts und der voll-/leer-verhältnisse von adeno-assoziierten viruspartikeln
EP3787771A1 (de) 2018-04-29 2021-03-10 REGENXBIO Inc. Skalierbares klärungsverfahren für die rekombinante aav-produktion
AU2019285186A1 (en) 2018-06-14 2021-01-07 Regenxbio Inc. Anion exchange chromatography for recombinant AAV production
US20210363219A1 (en) 2018-06-15 2021-11-25 Alpine Immune Sciences, Inc. Pd-1 variant immunomodulatory proteins and uses thereof
SG11202101072UA (en) 2018-08-03 2021-03-30 Genzyme Corp Variant rnai against alpha-synuclein
EP3833745A1 (de) 2018-08-10 2021-06-16 REGENXBIO Inc. Skalierbares verfahren zur herstellung von rekombinantem aav
CN110857440B (zh) 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
CA3112578A1 (en) 2018-09-19 2020-03-26 Alpine Immune Sciences, Inc. Methods and uses of variant cd80 fusion proteins and related constructs
WO2020077250A1 (en) 2018-10-12 2020-04-16 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
JP2022514112A (ja) 2018-10-15 2022-02-09 リジェネクスバイオ インコーポレイテッド 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
CN111118016B (zh) 2018-10-30 2022-04-15 上海朗昇生物科技有限公司 治疗视网膜色素变性疾病的基因治疗载体
JP2022510276A (ja) 2018-11-30 2022-01-26 アルパイン イミューン サイエンシズ インコーポレイテッド Cd86バリアント免疫調節タンパク質およびその使用
WO2020142714A1 (en) 2019-01-04 2020-07-09 Exonics Therapeutics, Inc. Aav expression cassette and aav vectors comprising the same
PE20212072A1 (es) 2019-02-26 2021-10-26 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe
CN111718418B (zh) 2019-03-19 2021-08-27 华东师范大学 一种增强基因编辑的融合蛋白及其应用
WO2020206098A1 (en) 2019-04-03 2020-10-08 Regenxbio Inc. Gene therapy for eye pathologies
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
SI3953483T1 (sl) 2019-04-11 2024-02-29 Regenxbio Inc. Postopki gelske izključitvene kromatografije za karakterizacijo spojin rekombinantnih adeno-povezanih virusov
JP2022529059A (ja) 2019-04-17 2022-06-16 アルパイン イミューン サイエンシズ インコーポレイテッド バリアントicosリガンド(icosl)融合タンパク質の方法および使用
US20220143115A1 (en) 2019-04-19 2022-05-12 Regenxbio Inc. Adeno-Associated Virus Vector Formulations and Methods
CN114144197A (zh) 2019-04-24 2022-03-04 再生生物股份有限公司 完全人类翻译后修饰的抗体治疗剂
CN112180094A (zh) 2019-07-04 2021-01-05 上海东慈生物科技有限公司 Dkk1抑制剂在预防和/或治疗肿瘤恶病质与糖尿病伴随疾病中的应用
EP3997225A1 (de) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren zur behandlung von epilepsie
CA3145112A1 (en) 2019-07-26 2021-02-04 Chunping Qiao Engineered nucleic acid regulatory element and methods of uses thereof
EP4039801A4 (de) 2019-08-16 2023-10-18 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences Verwendung von ptbp1-inhibitoren zur vorbeugung und/oderbehandlung von erkrankungen des nervensystems in zusammenhang mit funktionellem neuronalem tod
KR20220051246A (ko) 2019-08-26 2022-04-26 리젠엑스바이오 인크. 완전 인간 번역 후 변형된 항-VEGF Fab를 사용한 당뇨병성 망막병증의 치료
AU2020362119A1 (en) 2019-10-07 2022-05-26 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
EP4061943A1 (de) 2019-11-19 2022-09-28 Institut National de la Santé et de la Recherche Médicale (INSERM) Antisense-oligonukleotide und deren verwendung zur behandlung von krebs
CA3159516A1 (en) 2019-11-28 2021-06-03 Chunping Qiao Microdystrophin gene therapy constructs and uses thereof
JP2023511382A (ja) 2020-01-22 2023-03-17 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα-L-イズロニダーゼ(IDUA)を用いたムコ多糖症I型の治療
EP4096631A1 (de) 2020-01-29 2022-12-07 RegenxBio Inc. Behandlung von mukopolysaccharidose ii mit rekombinanter humaner iduronat-2-sulfatase (ids), produziert von humanen nerven- oder gliazellen
AR121201A1 (es) 2020-01-29 2022-04-27 Regenxbio Inc Tratamiento de mucopolisacaridosis iva
GB202004498D0 (en) 2020-03-27 2020-05-13 Ucl Business Ltd Activity-dependent gene therapy for neurological disorders
MX2022012209A (es) 2020-03-30 2022-10-27 Wuhan Neurophth Biotechnology Ltd Company Vector de expresion del factor 2 relacionado con el factor nuclear humano e2 y aplicacion del vector de expresion.
BR112022022433A2 (pt) 2020-05-08 2022-12-13 Alpine Immune Sciences Inc Proteínas imunomoduladoras inibitórias april e baff e métodos de uso das mesmas
JP2023526310A (ja) 2020-05-12 2023-06-21 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 導入遺伝子発現のdrg特異的低減のための組成物
JP2023526311A (ja) 2020-05-12 2023-06-21 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 クラッベ病の治療に有用な組成物
CN115968302A (zh) 2020-06-17 2023-04-14 宾夕法尼亚州大学信托人 用于治疗基因疗法患者的组合物和方法
IL299771A (en) 2020-07-10 2023-03-01 Inst Nat Sante Rech Med Methods and compounds for the treatment of epilepsy
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
US20230265456A1 (en) 2020-08-10 2023-08-24 Fundacion Para La Investigacion Medica Aplicada Gene therapy vector expressing cyp27a1 for the treatment of cerebrotendinous xanthomatosis
US20230372539A1 (en) 2020-08-24 2023-11-23 The Trustees Of The University Of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
WO2022060915A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized lanadelumab and administration thereof
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
AU2021355481A1 (en) 2020-10-01 2023-06-08 Genzyme Corporation Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
KR20230106598A (ko) 2020-10-07 2023-07-13 리젠엑스바이오 인크. 겔 제형과 같은 맥락막위 투여용 제형
AU2021358964A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
WO2022076549A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
IL301647A (en) 2020-10-07 2023-05-01 Regenxbio Inc Adeno-associated viruses for ocular delivery of gene therapy
WO2022076582A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
WO2022076803A1 (en) 2020-10-09 2022-04-14 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
AR123838A1 (es) 2020-10-18 2023-01-18 Univ Pennsylvania Vector mejorado de virus adenoasociado (aav) y usos de este
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
TW202233841A (zh) 2020-10-28 2022-09-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗TNF-α抗體
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
CN116457373A (zh) 2020-10-29 2023-07-18 再生生物股份有限公司 用于眼部适应症的载体化TNF-α拮抗剂
WO2022094255A2 (en) 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
EP4236975A1 (de) 2020-10-29 2023-09-06 The Trustees of The University of Pennsylvania Aav-kapside und diese enthaltende zusammensetzungen
JP2024016295A (ja) * 2020-11-30 2024-02-07 タカラバイオ株式会社 二価の陽イオンを利用した非エンベロープウイルスの製造方法
IL303236A (en) 2020-12-01 2023-07-01 Univ Pennsylvania New compounds with specific tissue-targeting motifs and preparations containing them
CN116761892A (zh) 2020-12-16 2023-09-15 再生生物股份有限公司 生产重组腺相关病毒颗粒的方法
EP4267197A1 (de) 2020-12-23 2023-11-01 Vivet Therapeutics Durch minimale gallensäure induzierbare promotoren zur gentherapie
TW202241943A (zh) 2020-12-29 2022-11-01 美商銳進科斯生物股份有限公司 Tau特異性抗體基因療法組合物、方法及其用途
US20240084329A1 (en) 2021-01-21 2024-03-14 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AU2022221284A1 (en) 2021-02-10 2023-08-17 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
JP2024511409A (ja) 2021-03-22 2024-03-13 ジェンザイム・コーポレーション 空および完全aavキャプシドのサイズ排除クロマトグラフィー解析
TW202305124A (zh) 2021-04-23 2023-02-01 賓州大學委員會 具有腦特異性靶向模體的新穎構成物及含有其之組成物
CA3216744A1 (en) 2021-04-26 2022-11-03 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2022235614A2 (en) 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
IL308336A (en) 2021-05-07 2024-01-01 Alpine Immune Sciences Inc Methods of dosing and treatment with TACI-FC modulatory fusion protein
WO2022241030A1 (en) 2021-05-11 2022-11-17 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
CA3226402A1 (en) 2021-07-19 2023-01-26 Dounia ABBADI Adeno-associated viral vector compositions and methods of promoting muscle regeneration
CA3226452A1 (en) 2021-07-19 2023-01-26 New York University Auf1 combination therapies for treatment of muscle degenerative disease
WO2023010133A2 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023062652A1 (en) * 2021-10-13 2023-04-20 Haystackanalyt Ics Pvt. Ltd Systems and methods for secure genomic analysis using a specialized edge computing device
WO2023077092A1 (en) 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023131682A1 (en) 2022-01-06 2023-07-13 Ucl Business Ltd Endogenous gene regulation to treat neurological disorders and diseases
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
GB202201744D0 (en) 2022-02-10 2022-03-30 Ucl Business Ltd Treatment of acquired focal epilepsy
WO2023172883A1 (en) 2022-03-07 2023-09-14 Alpine Immune Sciences, Inc. Immunomodulatory proteins of variant cd80 polypeptides, cell therapies thereof and related methods and uses
TW202346590A (zh) 2022-03-13 2023-12-01 美商銳進科斯生物股份有限公司 經修飾之肌肉特異性啟動子
WO2023178220A1 (en) 2022-03-16 2023-09-21 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2023183623A1 (en) 2022-03-25 2023-09-28 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2023196842A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
WO2023196873A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene forsuprachoidal administration
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2023196862A1 (en) 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2023201207A1 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20230405151A1 (en) 2022-04-12 2023-12-21 Genzyme Corporation Use of irak4 modulators for gene therapy
WO2023201273A1 (en) 2022-04-12 2023-10-19 Genzyme Corporation Dendritic cell assay for innate immunogenicity to gene therapy agents
US20230405014A1 (en) 2022-04-12 2023-12-21 Genzyme Corporation Use of an irak4 modulator for gene therapy
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023201308A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023205610A2 (en) 2022-04-18 2023-10-26 Regenxbio Inc. Hybrid aav capsids
WO2023215806A2 (en) 2022-05-03 2023-11-09 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2023215807A1 (en) 2022-05-03 2023-11-09 Regenxbio Inc. VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
US20230398192A1 (en) 2022-05-16 2023-12-14 Genzyme Corporation Methods of treating metachromatic leukodystrophy
WO2023222829A1 (en) 2022-05-17 2023-11-23 Centre Hospitalier Universitaire Vaudois Designed biosensors for enhanced t cell therapy
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2024015881A2 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
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
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5605690A (en) 1989-09-05 1997-02-25 Immunex Corporation Methods of lowering active TNF-α levels in mammals using tumor necrosis factor receptor
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5869305A (en) * 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
DE69433592T2 (de) 1993-11-09 2005-02-10 Targeted Genetics Corp., Seattle Die erzielung hoher titer des rekombinanten aav-vektors
JP3952312B2 (ja) 1993-11-09 2007-08-01 メディカル カレッジ オブ オハイオ アデノ関連ウイルス複製遺伝子を発現可能な安定な細胞株
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6281010B1 (en) * 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5677158A (en) * 1995-06-07 1997-10-14 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
WO1997032990A1 (en) 1996-03-04 1997-09-12 Targeted Genetics Corporation Methods for transducing cells in blood vessels using recombinant aav vectors
AU741605B2 (en) 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
WO1998027207A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
US6605429B1 (en) 1997-01-23 2003-08-12 Immusol, Incorporated Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
JP2001514845A (ja) 1997-09-05 2001-09-18 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ベクターの高力価ヘルパーなし調製物を生成するための方法
DE69840948D1 (de) 1997-10-21 2009-08-13 Targeted Genetics Corp Invertierte terminale repeats von aav, die für die transkription aktiviert sind, zur verwendung in rekombinanten aav-vektoren
AU758541B2 (en) 1997-10-21 2003-03-27 Targeted Genetics Corporation Amplifiable adeno-associated virus (AAV) packaging cassettes for the production of recombinant AAV vectors
JP4472182B2 (ja) 1998-09-04 2010-06-02 ターゲティッド ジェネティクス コーポレイション 放出された組換えaavベクターの高力価のヘルパーを含まない調製物を生成するための方法
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
AU783037B2 (en) 1999-05-28 2005-09-15 Targeted Genetics Corporation Methods and compositions for lowering the level of tumor necrosis factor (TNF) in the TNF-associated disorders
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
WO2001092551A2 (en) * 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
JP6143466B2 (ja) 2013-01-10 2017-06-07 株式会社ジャパンディスプレイ タッチパネル内蔵型液晶表示装置

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ES2308989T3 (es) 2008-12-16
AU6626500A (en) 2001-03-05
DK1204739T3 (da) 2008-10-20
US20030219735A1 (en) 2003-11-27
JP2003506083A (ja) 2003-02-18
JP5277427B2 (ja) 2013-08-28
AU781958C (en) 2006-03-30
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US20120184602A1 (en) 2012-07-19
DK1916258T3 (da) 2014-07-28
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US7785888B2 (en) 2010-08-31
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