IL128780A0 - An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase - Google Patents

An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase

Info

Publication number
IL128780A0
IL128780A0 IL12878097A IL12878097A IL128780A0 IL 128780 A0 IL128780 A0 IL 128780A0 IL 12878097 A IL12878097 A IL 12878097A IL 12878097 A IL12878097 A IL 12878097A IL 128780 A0 IL128780 A0 IL 128780A0
Authority
IL
Israel
Prior art keywords
polymerase
production
associated viruses
recombinant adeno
viruses utilizing
Prior art date
Application number
IL12878097A
Other languages
English (en)
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of IL128780A0 publication Critical patent/IL128780A0/xx

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Classifications

    • 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
    • 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
IL12878097A 1996-09-06 1997-09-04 An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase IL128780A0 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US2469996P 1996-09-06 1996-09-06
PCT/US1997/015716 WO1998010088A1 (fr) 1996-09-06 1997-09-04 Procede inductible de production de virus adeno-associes recombines au moyen de la polymerase t7

Publications (1)

Publication Number Publication Date
IL128780A0 true IL128780A0 (en) 2000-01-31

Family

ID=21821933

Family Applications (1)

Application Number Title Priority Date Filing Date
IL12878097A IL128780A0 (en) 1996-09-06 1997-09-04 An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase

Country Status (6)

Country Link
EP (1) EP0931158A1 (fr)
JP (1) JP2001500015A (fr)
AU (1) AU722624B2 (fr)
CA (1) CA2264482A1 (fr)
IL (1) IL128780A0 (fr)
WO (1) WO1998010088A1 (fr)

Families Citing this family (216)

* 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
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
AU5819599A (en) * 1998-09-11 2000-04-03 Regents Of The University Of California, The Recombinant adenovirus for tissue specific expression in heart
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
US6793926B1 (en) 1999-05-27 2004-09-21 Genovo, Inc. Methods for production of a recombinant adeno-associated virus
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
SG2013034475A (en) 2001-11-21 2016-10-28 Univ Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
EP1944043A1 (fr) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Séquences d'acides aminés et d'acides nucléiques d'adénovirus simien, vecteurs les contenant, et procédés d'utilisation.
JP4769417B2 (ja) 2001-12-17 2011-09-07 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 アデノ随伴ウイルス(aav)血清型9の配列、それを含むベクターおよびその使用
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
JP2005532789A (ja) 2002-03-15 2005-11-04 ワイス・ホールデイングス・コーポレーシヨン 減少した酵素活性を有する型別不能なインフルエンザ菌(Haemophilusinfluenza)のP4タンパク質の変異体
ES2359473T3 (es) 2003-07-21 2011-05-23 Transgene S.A. Citoquinas multifuncionales.
WO2005030292A2 (fr) 2003-09-26 2005-04-07 The Trustees Of The University Of Pennsylvania Methodes, compositions et dispositifs permettant d'administrer des molecules heterologues aux cellules
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
TW200613554A (en) 2004-06-17 2006-05-01 Wyeth Corp Plasmid having three complete transcriptional units and immunogenic compositions for inducing an immune response to HIV
US20090054823A1 (en) 2004-09-30 2009-02-26 The Trustees Of The University Of Pennsylvania Perfusion circuit and use therein in targeted delivery of macromolecules
US7517870B2 (en) 2004-12-03 2009-04-14 Fondazione Telethon Use of compounds that interfere with the hedgehog signaling pathway for the manufacture of a medicament for preventing, inhibiting, and/or reversing ocular diseases related with ocular neovascularization
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
MX2009000813A (es) 2006-07-27 2009-02-03 Wyeth Corp Proceso de fermentacion alimentada de alta densidad celular para producir proteinas recombinantes.
MX347246B (es) 2007-11-28 2017-04-19 Univ Pennsylvania Adenovirus e simianos sadv-39, sadv-25.2, sadv-26, sadv-30, sadv-37 y sadv-38.
WO2009105084A2 (fr) 2007-11-28 2009-08-27 The Trustees Of The University Of Pennsylvania Adénovirus c de la sous-famille simiesque sadv-40, -31, et -34 et leurs utilisations
US8470310B2 (en) 2008-03-04 2013-06-25 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-36, -42.1, -42.2, and -44 and uses thereof
US8940290B2 (en) 2008-10-31 2015-01-27 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-43, -45, -46, -47, -48, -49, and -50 and uses thereof
PL2425000T3 (pl) 2009-04-30 2019-08-30 The Trustees Of The University Of Pennsylvania Kompozycje do kierowania komórek przewodzących do dróg oddechowych zawierające konstrukty wirusów związanych z adenowirusami
CN102575232B (zh) 2009-05-29 2015-07-22 宾夕法尼亚大学托管会 猿腺病毒41及其应用
US20120309011A1 (en) * 2009-11-02 2012-12-06 Salk Institute For Biological Studies Targeting of modifying enzymes for protein evolution
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
AU2011305249B2 (en) 2010-09-26 2015-07-23 Da Yu Enterprises, L.L.C. Method of recombinant macromolecular production
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
EP3202897B1 (fr) 2010-12-09 2020-04-15 Institut Pasteur Utilisation diagnostique d'un polypeptide de fusion comprenant une proteine virale et une enzyme mgmt
BR112013020734A2 (pt) 2011-02-17 2017-06-13 Univ Pennsylvania composições e métodos para alterar a especificidade do tecido e aprimorar a transferência gênica mediada por aav9
EP2710130B1 (fr) 2011-05-19 2022-06-29 Fundación Pública Andaluza Progreso Y Salud Système de vecteur lentiviral de type "tet-on" à deux promoteurs hautement inductible
ES2716723T3 (es) 2011-09-20 2019-06-14 Univ North Carolina Chapel Hill Regulación de los canales de sodio por las proteínas PLUNC
US10640785B2 (en) 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US9638692B2 (en) 2011-12-09 2017-05-02 Institut Pasteur Multiplex immuno screening assay
PT2839014T (pt) 2012-04-18 2021-03-19 Childrens Hospital Philadelphia ¿composição e métodos para transferência de genes altamente eficiente com a utilização de variantes de capsídeo de aav
KR20150014505A (ko) 2012-05-18 2015-02-06 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아과 e 원숭이 아데노바이러스 a1302, a1320, a1331 및 a1337 및 이것들의 사용
EP2912069B1 (fr) 2012-10-23 2019-07-31 Emory University Conjugués de gm-csf et d'il-4, compositions et procédés associés
WO2014068072A1 (fr) 2012-10-31 2014-05-08 Institut Gustave-Roussy Identification, évaluation et traitement de la thrombocytémie essentielle associée à une résistance aux inhibiteurs de jak2
WO2014071182A1 (fr) 2012-11-01 2014-05-08 Massachusetts Institute Of Technology Évolution dirigée d'une batterie de gènes synthétiques
EP2917341B1 (fr) 2012-11-08 2017-09-27 Centre National de la Recherche Scientifique (CNRS) Nouvelles enzymes mutantes de phosphodeoxyribosyltransferase et leurs utilisations
JP6591956B2 (ja) 2013-03-15 2019-10-16 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 Mps1を治療するための組成物および方法
EP3019620A4 (fr) 2013-07-12 2017-01-18 The Children's Hospital of Philadelphia Vecteur aav et analyse pour anticorps de neutralisation anti-aav (virus adéno-associé)
WO2015013313A2 (fr) 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Compositions et variants de virus adéno-associés, et méthodes et utilisations pour un transfert de gènes dans des cellules, des organes et des tissus
SI3116900T1 (sl) 2014-03-09 2021-02-26 The Trustees Of The University Of Pennsylvania Sestavki uporabni pri zdravljenju pomanjkanja ornitin transkarbamilaze(OTC)
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
HUE054768T2 (hu) 2014-05-02 2021-09-28 Genzyme Corp AAV vektorok retina és CNS génterápiára
WO2015191508A1 (fr) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
PL3169341T3 (pl) 2014-07-16 2019-12-31 Transgene Sa Wirus onkolityczny do ekspresji modulatorów punktu kontroli immunologicznej
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるaavベクター
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
WO2016094783A1 (fr) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions et méthodes pour la production de scaav
CN107427666B (zh) 2015-01-30 2022-11-04 加利福尼亚大学校董 脊髓软膜下基因递送系统
AU2016219396B2 (en) 2015-02-10 2022-03-17 Genzyme Corporation Variant RNAi
SG10201907399RA (en) 2015-02-10 2019-09-27 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
WO2016131945A1 (fr) 2015-02-20 2016-08-25 Transgene Sa Produit de combinaison modulateur de l'autophagie
CA2978917A1 (fr) 2015-03-10 2016-09-15 The Trustees Of Columbia University In The City Of New York Constructions de vecteur viral adeno-associe glut1 de recombinaison et procedes associes permettant de restaurer l'expression de glut1
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CA3019315A1 (fr) 2015-04-23 2016-10-27 University Of Massachusetts Modulation de l'expression d'un transgene du vecteur aav
US11896651B2 (en) 2015-05-16 2024-02-13 Genzyme Corporation Gene editing of deep intronic mutations
KR102415896B1 (ko) 2015-06-23 2022-06-30 더 칠드런스 호스피탈 오브 필라델피아 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도
CN115418357A (zh) 2015-07-13 2022-12-02 皮沃特生物公司 改良植物性状的方法及组合物
AU2016336328A1 (en) 2015-10-05 2018-04-19 Massachusetts Institute Of Technology Nitrogen fixation using refactored nif clusters
DK3364997T3 (da) 2015-10-22 2024-04-22 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
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
CA3012195A1 (fr) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Therapie genique pour traiter la mucopolysaccharidose de type i
CA3012344A1 (fr) 2016-02-12 2017-08-17 University Of Massachusetts Agents therapeutiques a base de micro-arn anti-angiogeniques pour l'inhibition de la neovascularisation corneenne
EP3417055B1 (fr) 2016-02-16 2021-10-13 The Board of Trustees of the Leland Stanford Junior University Nouveaux capsides de virus adéno-associés de recombinaison résistants à des anticorps neutralisants humains pré-existants
AU2017222450A1 (en) 2016-02-22 2018-09-27 The University Of North Carolina At Chapel Hill Peptide inhibitors of calcium channels
RU2752882C2 (ru) 2016-03-03 2021-08-11 Юниверсити Оф Массачусетс Линейная дуплексная днк с закрытым концом для невирусного переноса генов
EP3448987A4 (fr) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
WO2017189959A1 (fr) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
CA3023022A1 (fr) 2016-05-04 2017-11-09 Transgene Sa Polytherapie avec un ligand de tlr9 cpg
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
CA3027784A1 (fr) 2016-06-16 2017-12-21 Charite - Universitatsmedizin Berlin Vecteurs d'expression de neuropeptides et methodes de traitement de l'epilepsie
MA45493A (fr) 2016-06-27 2019-05-01 Aicuris Anti Infective Cures Gmbh Inhibiteurs d'entrée de hcmv.
KR20190096329A (ko) 2016-07-05 2019-08-19 유니버시티 오브 매사추세츠 녹내장에서 신경보호 요법으로서 sfasl의 aav2-매개된 유전자 전달
CA3030156A1 (fr) 2016-07-07 2018-01-11 Iovance Biotherapeutics, Inc. Proteines de liaison au ligand (1) de mort programmee (1) (pd-l1) et leurs procedes d'utilisation
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
TN2019000047A1 (en) 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
JP2019530737A (ja) 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
WO2018049261A1 (fr) 2016-09-09 2018-03-15 Icellhealth Consulting Llc Virus oncolytique exprimant des modulateurs du point de contrôle immunitaire
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
EP3518966A1 (fr) 2016-09-29 2019-08-07 GlaxoSmithKline Biologicals S.A. Compositions et méthodes de traitement d'une infection par hpv persistante
US20190328869A1 (en) 2016-10-10 2019-10-31 Transgene Sa Immunotherapeutic product and mdsc modulator combination therapy
TWI788307B (zh) 2016-10-31 2023-01-01 美商艾歐凡斯生物治療公司 用於擴增腫瘤浸潤性淋巴細胞之工程化人造抗原呈現細胞
WO2018091680A1 (fr) 2016-11-18 2018-05-24 Transgene Sa Vecteurs oncolytiques à base de la variole bovine
GB201620968D0 (en) 2016-12-09 2017-01-25 Glaxosmithkline Biologicals Sa Adenovirus polynucleotides and polypeptides
KR20190097240A (ko) 2016-12-28 2019-08-20 트랜스진 에스.에이. 종양용해성 바이러스 및 치료 분자
WO2018132774A1 (fr) 2017-01-12 2018-07-19 Pivot Bio, Inc. Procédés et compositions destinés à l'amélioration des caractéristiques d'une plante
JP7307480B2 (ja) 2017-04-05 2023-07-12 ユニバーシティ オブ マサチューセッツ ミニ遺伝子療法
EP3619299A1 (fr) 2017-05-03 2020-03-11 BioMarin Pharmaceutical Inc. Lentivirus améliorés pour la transduction de cellules souches hématopoïétiques
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
RU2020100074A (ru) 2017-06-13 2021-08-03 Флэгшип Пайониринг Инновейшнз V, Инк. Композиции, содержащие куроны, и пути их применения
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11773407B2 (en) 2017-06-26 2023-10-03 Arizona Board Of Regents On Behalf Of Arizona State University CRISPR logic circuits for safer and controllable gene therapies
AU2018298133A1 (en) 2017-07-06 2020-01-23 The Trustees Of The University Of Pennsylvania AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
WO2019020543A1 (fr) 2017-07-28 2019-01-31 Transgene Sa Virus oncolytiques exprimant des agents ciblant des modulateurs immunitaires métaboliques
KR20200044793A (ko) 2017-08-03 2020-04-29 보이저 테라퓨틱스, 인크. Aav의 전달을 위한 조성물 및 방법
KR20200056428A (ko) 2017-09-22 2020-05-22 젠자임 코포레이션 변이체 RNAi
WO2019079242A1 (fr) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
JP2021502060A (ja) 2017-10-16 2021-01-28 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)の治療
BR112020012336A2 (pt) 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
EP3762500A1 (fr) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Génomes aav partiels auto-complémentaires fabriqués par des cellules d'insectes
JP2021519065A (ja) 2018-03-23 2021-08-10 ユニバーシティ オブ マサチューセッツ 骨障害を処置するための遺伝子治療法
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
EP3784290A4 (fr) 2018-04-27 2022-03-23 University of Massachusetts Capsides de vaa identifiés par la sélection d'une bibliothèque in vivo
AU2019257755A1 (en) 2018-04-27 2020-11-12 Voyager Therapeutics, Inc. Methods for measuring the potency of AADC viral vectors
EP3790394A4 (fr) 2018-05-08 2022-07-27 Rutgers, the State University of New Jersey Protéines de polymérisation de lieur-laminine compatibles avec aav
SG11202010830WA (en) 2018-05-09 2020-11-27 Biomarin Pharm Inc Methods of treating phenylketonuria
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
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
WO2019222441A1 (fr) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Sérotypes de vaa pour l'administration de charge utile spécifique au cerveau
BR112020024285A2 (pt) 2018-06-12 2021-03-02 Glaxosmithkline Biologicals S.A. polinucleotídeos e polipeptídeos de adenovírus
EP3807404A1 (fr) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Régions 5' non traduites (5'utr) modifiées pour la production d'aav
CN112567035A (zh) 2018-07-02 2021-03-26 沃雅戈治疗公司 肌萎缩侧索硬化症及脊髓相关病症的治疗
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
EP3830265A1 (fr) 2018-08-03 2021-06-09 Genzyme Corporation Variante d'arni contre l'alpha-synucléine
CN113166208A (zh) 2018-10-02 2021-07-23 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
TW202028458A (zh) 2018-10-05 2020-08-01 美商航海家醫療公司 編碼腺相關病毒(aav)生產蛋白之經基因工程化核酸構築體
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3867389A1 (fr) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Vecteurs d'expression pour la production à grande échelle de raav dans le système baculovirus/sf9
CN117969861A (zh) 2018-10-25 2024-05-03 瑞泽恩制药公司 用于分析病毒衣壳蛋白组成的方法
WO2020097344A1 (fr) 2018-11-08 2020-05-14 Arizona Board of Regents on Behalf Arizona State University Immunomodulation synthétique avec un super-répresseur de crispr in vivo
WO2020112967A1 (fr) 2018-11-29 2020-06-04 University Of Massachusetts Modulation de sptlc1 par l'intermédiaire de vecteurs adéno-associés recombinés
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
WO2020136232A1 (fr) 2018-12-28 2020-07-02 Transgene Sa Protéine m2 à propriété immunosuppressive
AU2020208467A1 (en) 2019-01-18 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing AAV particles
US20220054655A1 (en) 2019-02-22 2022-02-24 University Of Massachusetts Oxr1 gene therapy
US20200299637A1 (en) 2019-03-19 2020-09-24 Massachusetts Institute Of Technology Control of nitrogen fixation in rhizobia that associate with cereals
TW202106879A (zh) 2019-04-29 2021-02-16 美商航海家醫療公司 於生物反應器中生產經桿狀病毒感染之昆蟲細胞(biic)之系統及方法
WO2020227515A1 (fr) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions et méthodes d'augmentation vectorisée de la destruction, de l'expression et/ou de la régulation de protéines
US20200362368A1 (en) 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
EP4007814A1 (fr) 2019-07-26 2022-06-08 Akouos, Inc. Méthodes de traitement de la perte auditive à l'aide d'une protéine cible sécrétée
EP4010465A1 (fr) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs
US20220364114A1 (en) 2019-08-26 2022-11-17 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021046155A1 (fr) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Édition vectorisée d'acides nucléiques pour corriger des mutations manifestes
CN114555814A (zh) 2019-09-13 2022-05-27 罗特格斯新泽西州立大学 Aav相容的层粘连蛋白-连接子聚合蛋白
JP2022549679A (ja) 2019-09-27 2022-11-28 バイオマリン ファーマシューティカル インコーポレイテッド サイズ排除クロマトグラフィー及び多角度光散乱技術を使用した遺伝子療法ウイルス粒子のキャラクタリゼーション
KR20220133941A (ko) 2020-01-29 2022-10-05 젠자임 코포레이션 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법
AU2021225035A1 (en) 2020-02-21 2022-10-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
MX2022012279A (es) 2020-03-31 2023-02-23 Univ Massachusetts Variantes de capsides de aav y usos de los mismos.
US20230131352A1 (en) 2020-04-01 2023-04-27 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
TW202144579A (zh) 2020-04-09 2021-12-01 美商4Mvac有限責任公司 病毒載體在冠狀病毒疫苗生產的用途
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
EP4165194A2 (fr) 2020-05-13 2023-04-19 Akouos, Inc. Compositions et méthodes de traitement de perte auditive associée à kcnq4
CA3183171A1 (fr) 2020-05-13 2021-11-18 Akouos, Inc. Compositions et methodes pour traiter une perte auditive associee a slc26a4
WO2021230987A1 (fr) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection de tropisme de capsides de vaa
WO2021247995A2 (fr) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la douleur neuropathique
KR20230038496A (ko) 2020-07-13 2023-03-20 트랜스진 면역 억제의 치료
EP4192514A1 (fr) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs
US20240043494A1 (en) 2020-08-07 2024-02-08 Amicus Therapeutics, Inc. Vesicle Targeting Proteins And Uses Of Same
AU2021338361A1 (en) 2020-09-03 2023-04-06 Chen, Irvin S.Y Soluble alkaline phosphatase constructs and expression vectors including a polynucleotide encoding for soluble alkaline phosphatase constructs
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
AU2021372262A1 (en) 2020-11-02 2023-06-01 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
PE20240115A1 (es) 2020-12-01 2024-01-22 Akouos Inc Construcciones de anticuerpos anti-vegf y metodos relacionados para el tratamiento de los sintomas asociados al schwannoma vestibular
AU2021409952A1 (en) 2020-12-23 2023-07-06 Flagship Pioneering Innovations V, Inc. In vitro assembly of anellovirus capsids enclosing rna
EP4271419A1 (fr) 2020-12-29 2023-11-08 Akouos, Inc. Compositions et méthodes pour traiter une perte auditive et/ou une perte de la vision associées à clrn1
WO2022147481A1 (fr) 2020-12-30 2022-07-07 Ansun Biopharma Inc. Polythérapie d'un virus oncolytique délivrant un antigène étranger et cellule immunitaire modifiée exprimant un récepteur chimérique ciblant l'antigène étranger
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (fr) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Expression contrôlée de protéines virales
TW202309291A (zh) 2021-04-07 2023-03-01 法商新植物Sas公司 用於室內空氣修復之組合物及方法
CA3216719A1 (fr) 2021-05-03 2022-11-10 Astellas Institute For Regenerative Medicine Procedes de generation de cellules endotheliales corneennes matures
CA3227613A1 (fr) 2021-08-11 2023-02-16 William Dowdle Systemes inductibles pour modifier l'expression genique dans des cellules hypoimmunogenes
WO2023034994A1 (fr) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
WO2023034997A1 (fr) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
WO2023034989A1 (fr) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
WO2023034996A1 (fr) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
WO2023034980A1 (fr) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
WO2023034990A1 (fr) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
WO2023044483A2 (fr) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement du cancer positif her2
WO2023056329A1 (fr) 2021-09-30 2023-04-06 Akouos, Inc. Compositions et méthodes de traitement de perte auditive associée à kcnq4
TW202334436A (zh) 2021-11-02 2023-09-01 美商航海家醫療公司 Aav衣殼變異體及其用途
WO2023091948A1 (fr) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Variants de capsides d'aav et leurs utilisations
WO2023107188A1 (fr) 2021-12-10 2023-06-15 Massachusetts Institute Of Technology Prédiction, biosynthèse et intégration en tant que biodétecteurs de molécules portant des signes d'absorption de la lumière uniques, et de leur détection à distance sur le terrain au moyen de caméras multispectrales ou hyperspectrales
WO2023111580A1 (fr) 2021-12-16 2023-06-22 University Of Dundee Dégradation ciblée de l'alpha-synucléine
WO2023147374A2 (fr) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Système d'expression de baculovirus
WO2023150142A1 (fr) 2022-02-02 2023-08-10 Akouos, Inc. Constructions d'anticorps anti-vegf et méthodes associées pour le traitement de symptômes associés au schwannome vestibulaire
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Aav衣殼變異體及其用途
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
US20230365968A1 (en) 2022-04-06 2023-11-16 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2023200843A1 (fr) 2022-04-12 2023-10-19 The Broad Institute, Inc. Compositions et méthodes de criblage d'éléments régulateurs cis
US20230407255A1 (en) 2022-04-12 2023-12-21 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
WO2023201272A1 (fr) 2022-04-12 2023-10-19 Genzyme Corporation Utilisation de modulateurs irak4 pour la thérapie génique
WO2023213764A1 (fr) 2022-05-02 2023-11-09 Transgene Polypeptide de fusion comprenant un anti-pd-l1 sdab et un membre du tnfsf
WO2023213763A1 (fr) 2022-05-02 2023-11-09 Transgene Poxvirus codant pour un agent de liaison comprenant un sdab anti-pd-l1
WO2023220729A2 (fr) 2022-05-13 2023-11-16 Flagship Pioneering Innovations Vii, Llc Compositions d'adn à double brin et procédés associés
WO2023235791A1 (fr) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. Variants de capside de vaa et leurs utilisations
WO2024006741A1 (fr) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Variants de capside d'aav et leurs utilisations
WO2024003353A1 (fr) 2022-07-01 2024-01-04 Transgene Protéine de fusion comprenant une protéine d tensioactive et un élément de la tnfsf
WO2024011112A1 (fr) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Variants de capside d'aav et leurs utilisations
WO2024009280A1 (fr) 2022-07-08 2024-01-11 Baylor College Of Medicine Inhibiteurs de réponse au stress intégrés et leurs méthodes d'utilisation
WO2024054983A1 (fr) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Expression controlée de protéines virales
WO2024059739A1 (fr) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Composés de liaison à la protéine tau
WO2024064856A1 (fr) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Traitement de la cardiomyopathie au moyen de vecteurs de thérapie génique aav
WO2024064863A2 (fr) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Traitement de la cardiomyopathie arythmogène avec des vecteurs de thérapie génique aav
EP4345106A1 (fr) 2022-09-30 2024-04-03 Charité - Universitätsmedizin Berlin Vecteurs de thérapie génique pour l'expression de variants de préprodyrphine pour le traitement de l'épilepsie
WO2024069010A1 (fr) 2022-09-30 2024-04-04 Charité - Universitätsmedizin Berlin Vecteurs de thérapie génique pour l'expression de variants de préprodynorphine pour le traitement de l'épilepsie

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5135855A (en) * 1986-09-03 1992-08-04 The United States Of America As Represented By The Department Of Health And Human Services Rapid, versatile and simple system for expressing genes in eukaryotic cells
US5478745A (en) * 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5591601A (en) * 1993-05-14 1997-01-07 Ohio University Edison Animal Biotechnology Institute DNA polymerase gene expression system utilizing an RNA polymerase co-delivered with the gene expression vector system
EP0728214B1 (fr) * 1993-11-09 2004-07-28 Medical College Of Ohio Lignees cellulaires stables aptes a exprimer le gene de replication du virus adeno-associe
DE69433592T2 (de) * 1993-11-09 2005-02-10 Targeted Genetics Corp., Seattle Die erzielung hoher titer des rekombinanten aav-vektors
US5693531A (en) * 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
JPH10507352A (ja) * 1994-10-13 1998-07-21 ドイチェス クレブスフォルシュンクスツェントルム スチフトゥング デス エッフェントリヒェン レヒツ rep陰性AAV変異体の調製およびそれに使用可能な細胞
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5872005A (en) * 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
WO1996017947A1 (fr) * 1994-12-06 1996-06-13 Targeted Genetics Corporation Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants
FR2735789B1 (fr) * 1995-06-23 1997-07-25 Centre Nat Rech Scient Adenovirus recombinants, leur utilisation pour preparer des aav, lignee cellulaire complementaire et compositions pharmaceutiques les contenant

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