WO1997026336B1 - Compositions antianemiques - Google Patents

Compositions antianemiques

Info

Publication number
WO1997026336B1
WO1997026336B1 PCT/US1997/000980 US9700980W WO9726336B1 WO 1997026336 B1 WO1997026336 B1 WO 1997026336B1 US 9700980 W US9700980 W US 9700980W WO 9726336 B1 WO9726336 B1 WO 9726336B1
Authority
WO
WIPO (PCT)
Prior art keywords
muscle cell
nucleic acid
acid molecule
control elements
muscle
Prior art date
Application number
PCT/US1997/000980
Other languages
English (en)
Other versions
WO1997026336A1 (fr
WO1997026336A9 (fr
Filing date
Publication date
Priority claimed from US08/588,355 external-priority patent/US5858351A/en
Priority claimed from US08/785,750 external-priority patent/US5846528A/en
Application filed filed Critical
Priority to EP97902065A priority Critical patent/EP0934411A1/fr
Priority to CA2243470A priority patent/CA2243470C/fr
Priority to JP52628897A priority patent/JP2002504078A/ja
Publication of WO1997026336A1 publication Critical patent/WO1997026336A1/fr
Publication of WO1997026336B1 publication Critical patent/WO1997026336B1/fr
Publication of WO1997026336A9 publication Critical patent/WO1997026336A9/fr

Links

Abstract

L'invention porte sur l'emploi de virions de virus adéno-associés (AAV) de recombinaison pour fournir des molécules d'ADN aux cellules et aux tissus musculaires en vue du traitement de l'anémie. Elle recourt à une injection directe in vivo de virions d'AAV de recombinaison (par exemple par injection intramusculaire) dans les tissus musculaires et à la transduction in vitro de cellules de muscles qui sont ensuite administrées au patient pour le traiter. Elle porte par ailleurs sur l'expression continue à haut niveau d'une séquence nucléotidique administrée codant pour l'érythropoïétine et pour sa sécrétion in vivo à partir de cellules transduites de muscles, conduisant à une administration par voie générale.
PCT/US1997/000980 1996-01-18 1997-01-17 Compositions antianemiques WO1997026336A1 (fr)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP97902065A EP0934411A1 (fr) 1996-01-18 1997-01-17 Compositions antianemiques
CA2243470A CA2243470C (fr) 1996-01-18 1997-01-17 Virion de vaa recombine pour l'expression de l'epo dans un mammifere
JP52628897A JP2002504078A (ja) 1996-01-18 1997-01-17 貧血を処置するための組成物

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US08/588,355 US5858351A (en) 1996-01-18 1996-01-18 Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US08/588,355 1996-01-18
US08/785,750 1997-01-16
US08/785,750 US5846528A (en) 1996-01-18 1997-01-16 Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence

Publications (3)

Publication Number Publication Date
WO1997026336A1 WO1997026336A1 (fr) 1997-07-24
WO1997026336B1 true WO1997026336B1 (fr) 1997-10-09
WO1997026336A9 WO1997026336A9 (fr) 1997-11-13

Family

ID=27080263

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1997/000980 WO1997026336A1 (fr) 1996-01-18 1997-01-17 Compositions antianemiques

Country Status (5)

Country Link
US (6) US5846528A (fr)
EP (1) EP0934411A1 (fr)
JP (1) JP2002504078A (fr)
CA (2) CA2844461A1 (fr)
WO (1) WO1997026336A1 (fr)

Families Citing this family (91)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995011699A1 (fr) * 1993-10-29 1995-05-04 The Trustees Of Boston University Compositions physiologiquement stables d'acide butyrique, de sels et de derives d'acide butyrique, utilisees comme agents antineoplasiques
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
ES2337853T3 (es) * 1995-06-07 2010-04-29 University Of North Carolina At Chapel Hill Transduccion de mioblastos por vaa.
US6506379B1 (en) * 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US20030206891A1 (en) * 1995-06-07 2003-11-06 Ariad Gene Therapeutics, Inc. Rapamycin-based biological regulation
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5962313A (en) * 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
ES2247621T3 (es) 1996-02-15 2006-03-01 Biosense Webster, Inc. Sonda de excavacion.
US6443974B1 (en) * 1996-07-28 2002-09-03 Biosense, Inc. Electromagnetic cardiac biostimulation
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
CN1233291A (zh) * 1996-09-06 1999-10-27 宾西法尼亚大学托管会 重组腺伴随病毒定向基因治疗的方法
EP1005376B1 (fr) * 1997-03-14 2010-11-03 The Children's Hospital of Philadelphia Compositions utilisees en therapie genique dans le traitement de l'hemophilie
US20030113303A1 (en) * 1998-02-05 2003-06-19 Yitzhack Schwartz Homing of embryonic stem cells to a target zone in tissue using active therapeutics or substances
DE69838526T2 (de) 1998-02-05 2008-07-03 Biosense Webster, Inc., Diamond Bar Gerät zum Freisetzen eines Medikaments im Herzen
US20030129750A1 (en) * 1998-02-05 2003-07-10 Yitzhack Schwartz Homing of donor cells to a target zone in tissue using active therapeutics or substances
AU774861B2 (en) * 1998-02-11 2004-07-08 Douglas V Faller Compositions and methods for the treatment of cystic fibrosis
CA2326327C (fr) * 1998-04-24 2009-06-16 University Of Florida Vecteur recombinant de virus associe aux adenovirus encodant la proteine serique alpha-1-antitrypsine
US6291214B1 (en) * 1998-05-11 2001-09-18 Glaxo Wellcome Inc. System for generating recombinant viruses
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
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
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
CA2288341C (fr) * 1998-12-17 2005-01-04 Pfizer Products Inc. Erythropoietine dans le traitement de l'anemie chez les animaux domestiques et le betail
EP1520589A1 (fr) * 1998-12-17 2005-04-06 Pfizer Products Inc. Utilisation de l'érythropoiétine pour le traitement de l'anémie chez les animaux domestiques et le bétail
US7078387B1 (en) * 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
CA2359747A1 (fr) * 1999-02-09 2000-08-17 Lexicon Genetics Incorporated Proteines humaines bruleuses de graisses excedentaires et polynucleotides les codant
DE19905501B4 (de) * 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6893865B1 (en) * 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
EP1939300A1 (fr) 1999-05-28 2008-07-02 Targeted Genetics Corporation Procédés et compositions pour diminuer le niveau de facteur de nécrose de tumeur (TNF) pour des troubles associés au TNF
JP2003501043A (ja) * 1999-05-28 2003-01-14 ターゲティッド ジェネティクス コーポレイション 腫瘍壊死因子(tnf)関連障害において、tnfのレベルを低下させるための方法および組成物
AU782326B2 (en) 1999-10-12 2005-07-21 Lexicon Pharmaceuticals, Inc. Human LDL receptor family proteins and polynucleotides encoding the same
AU3642601A (en) * 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US6582692B1 (en) 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
FR2801319A1 (fr) * 1999-11-18 2001-05-25 Inst Nat Sante Rech Med Construction d'acide nucleique porteuse d'un systeme regulant l'expression d'un gene
AU1954001A (en) * 1999-12-10 2001-06-18 Ariad Gene Therapeutics, Inc. Methods for high level expression of genes in primates
US6790667B1 (en) * 2000-05-30 2004-09-14 Lexicon Genetics Incorporated Human mitochondrial proteins and polynucleotides encoding the same
AU2001296354A1 (en) 2000-09-25 2002-04-02 Baylor College Of Medicine Improved system for regulation of transgene expression
US7285414B2 (en) * 2000-09-26 2007-10-23 Emory University Viruses targeted to hypoxic cells and tissues
US20040101825A1 (en) * 2001-09-26 2004-05-27 Van Meir Erwin G. Viruses targeted to hypoxic cells and tissues
EP2017338A1 (fr) * 2001-05-24 2009-01-21 Genzyme Corporation Vecteurs d'expression spécifiques au muscle
CN102552261A (zh) * 2001-12-06 2012-07-11 法布罗根股份有限公司 提高内源性红细胞生成素(epo)的方法
AU2002359786A1 (en) * 2001-12-19 2003-07-09 Hiroaki Mizukami Adeno-associated virus-mediated delivery of gdnf to skeletal muscles
AU2003223497A1 (en) * 2002-04-05 2003-10-27 Centocor, Inc. Asthma-related anti-il-13 immunoglobulin derived proteins, compositions, methods and uses
TW200424214A (en) * 2003-04-21 2004-11-16 Advisys Inc Plasmid mediated GHRH supplementation for renal failures
ES2385837T3 (es) 2004-12-15 2012-08-01 The University Of North Carolina At Chapel Hill Vectores quiméricos
US7488848B2 (en) 2005-03-21 2009-02-10 Virobay, Inc. Alpha ketoamide compounds as cysteine protease inhibitors
CN101062407A (zh) 2006-04-29 2007-10-31 中国科学院上海生命科学研究院 促红细胞生成素在预防或治疗视网膜损伤中的用途
CA2652858A1 (fr) * 2006-06-15 2007-12-27 Targeted Genetics Corporation Procedes de traitement d'articulations cibles dans l'arthrite inflammatoire utilisant des vecteurs aav vectors codant pour un antagoniste du tnf
JP5623740B2 (ja) * 2006-07-25 2014-11-12 セラドン・コーポレーション 遺伝子治療用アデノ随伴ウイルスベクターの長期順行性の心外膜冠動脈注入
US8221738B2 (en) 2008-02-19 2012-07-17 Celladon Corporation Method for enhanced uptake of viral vectors in the myocardium
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010105112A1 (fr) * 2009-03-11 2010-09-16 Hemaquest Pharmaceuticals, Inc. Détection d'acides gras à chaîne courte dans des échantillons biologiques
US20110086869A1 (en) 2009-09-24 2011-04-14 The Trustees Of Boston University Methods for treating viral disorders
CN102802412A (zh) 2009-12-08 2012-11-28 海玛奎斯特医药公司 用于治疗红细胞病症的方法及低剂量方案
US20110245154A1 (en) 2010-03-11 2011-10-06 Hemaquest Pharmaceuticals, Inc. Methods and Compositions for Treating Viral or Virally-Induced Conditions
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
WO2012097256A1 (fr) * 2011-01-14 2012-07-19 University Of Tennessee Research Foundation Compositions et méthodes thérapeutiques utilisées pour le traitement de troubles associés à une dégénérescence neuronale
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
SG10202007103TA (en) 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CA2975583A1 (fr) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Polynucleotides modulateurs
WO2016094783A1 (fr) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions et méthodes pour la production de scaav
CA2974235A1 (fr) 2015-01-07 2016-07-14 Universitat Autonoma De Barcelona Construction genique a vecteur unique comprenant des genes d'insuline et de glucokinase
EP3344650A4 (fr) * 2015-08-31 2019-04-17 The Trustees Of The University Of Pennsylvania Aav-epo pour le traitement d'animaux de compagnie
WO2017189964A2 (fr) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CA3024448A1 (fr) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Polynucleotides modulateurs
EP3458589A4 (fr) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la maladie de huntington
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
CN110072883A (zh) 2016-09-28 2019-07-30 科巴公司 治疗性mots-c相关的肽
US11117930B2 (en) 2017-02-23 2021-09-14 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
CA3061368A1 (fr) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions et methodes de traitement de la maladie de huntington
AU2018261790A1 (en) 2017-05-05 2019-11-28 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
EP3634978A1 (fr) 2017-06-07 2020-04-15 Adrx, Inc. Inhibiteur d'agrégation de tau
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
CA3071978A1 (fr) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions et procedes permettant l'administration de virus adeno-associes
EP3697905A1 (fr) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
EP3697908A1 (fr) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
WO2019222328A1 (fr) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions et méthodes pour le traitement de la maladie de parkinson
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
JP2022518814A (ja) 2019-01-28 2022-03-16 コーバー、インコーポレイテッド 治療用ペプチド
KR20220034736A (ko) 2019-05-31 2022-03-18 비락타 서브시디어리 인크. 히스톤 데아세틸라제 억제제를 사용하여 바이러스 관련 암을 치료하는 방법
WO2021247995A2 (fr) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la douleur neuropathique
WO2022026409A1 (fr) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement des troubles neurologiques liés au déficit en glucosylcéramidase bêta
WO2022026410A2 (fr) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions et méthodes pour le traitement de la maladie de niemann-pick de type c1
AU2021350732A1 (en) * 2020-09-28 2023-05-11 Scout Bio, Inc. Composition and uses thereof
WO2022159414A1 (fr) 2021-01-22 2022-07-28 University Of Rochester Érythropoïétine pour un dysfonctionnement gastrointestinal
WO2023091949A2 (fr) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de troubles neurologiques liés à un déficit en bêta glucosylcéramidase
WO2023240236A1 (fr) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5858651A (en) * 1986-01-22 1999-01-12 Institut Pasteur Nucleotide sequences of human immunodeficiency virus type 2 (HIV-2), probes of HIV-2, and methods of using these probes
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5658656A (en) 1992-01-10 1997-08-19 Minnesota Mining And Manufacturing Company Use of materials comprising microbubbles as acoustical barriers
US5658565A (en) 1994-06-24 1997-08-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Inducible nitric oxide synthase gene for treatment of disease
US5478745A (en) * 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5589362A (en) * 1993-06-14 1996-12-31 Basf Aktiengesellschaft Tetracycline regulated transcriptional modulators with altered DNA binding specificities
AU1095595A (en) * 1993-11-10 1995-05-29 Amgen, Inc. Gene therapy vector for the treatment of low or defective red blood cell production
FR2716682B1 (fr) * 1994-01-28 1996-04-26 Centre Nat Rech Scient Procédé de préparation de virus adéno-associés (AAV) recombinants et utilisations.
JPH10501686A (ja) 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのdnaのaav仲介送達
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5872005A (en) 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
ES2337853T3 (es) * 1995-06-07 2010-04-29 University Of North Carolina At Chapel Hill Transduccion de mioblastos por vaa.
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) * 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme

Similar Documents

Publication Publication Date Title
WO1997026336B1 (fr) Compositions antianemiques
WO1997026337B1 (fr) Procedes de transmission d'adn a des cellules musculaires a l'aide de virions de virus adeno-associes de recombinaison
CA2243261A1 (fr) Procedes de transmission d'adn a des cellules musculaires a l'aide de virions de virus adeno-associes de recombinaison
CN109803977B (zh) 核酸产品及其施用方法
DE69534591T2 (de) Adenovirusfaser mit hinzugefügten liganden
Yoshida et al. Genomic sequence of hst, a transforming gene encoding a protein homologous to fibroblast growth factors and the int-2-encoded protein.
DE60034478T2 (de) Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leber
RU2346951C2 (ru) Выделенный полипептид, связывающий рецептор zalpha11-лиганда (варианты), кодирующий его полинуклеотид (варианты), вектор экспрессии (варианты) и клетка-хозяин (варианты)
US11707528B2 (en) Mannose-based mRNA targeted delivery system and use thereof
DE112005000737T5 (de) Mit Heparansulfat-Zuckerketten modifizierte Heparin-bindende Proteine, Verfahren zur Erzeugung derselben und Arzneimittel, welche dieselben enthalten
CA2102208A1 (fr) Domaines de liaison dans les proteines notch et delta
KR930703453A (ko) 유전자 치료법을 위한 안전한 벡터
CN101516388A (zh) 通过o-联糖基化序列的肽的糖基化
CA2223433A1 (fr) Compositions renfermant la proteine ob et procedes associes
ATE338821T1 (de) Strukturprotein von aav, seine herstellung und verwendung
WO2006033689A3 (fr) Diffusion de genes a destination de cellules cochleaires a mediation par aav
WO1998046781A3 (fr) Nouveau systeme d'expression de transgene pour persistance accrue
IL125081A0 (en) Ob protein receptor and related compositions and methods
WO1998022140A3 (fr) Compositions hybrides pour ciblage intracellulaire
WO1999001551A3 (fr) Nouvel inhibiteur de proliferation cellulaire
WO1997004104A3 (fr) Regions regulatrices de transcription d'hexokinase tumorale de type ii
RU2215787C2 (ru) АКТИН-РЕЗИСТЕНТНЫЙ ВАРИАНТ ДНКазы I (ВАРИАНТЫ), ВЫДЕЛЕННАЯ НУКЛЕИНОВАЯ КИСЛОТА, СПОСОБ СНИЖЕНИЯ ВЯЗКОУПРУГОСТИ ИЛИ ВЯЗКОЙ КОНСИСТЕНТНОСТИ МОКРОТЫ ПАЦИЕНТА
DE3638033A1 (de) Verfahren zum exprimieren von genen durch bacillus brevis
DE4233782A1 (de) Klonierung eines neuen Mitgliedes der Familie der Rezeptor-Protein-Tyrosin-Kinasen
DE69919515T2 (de) Biotinbindende rezeptormoleküle