EA201892006A1 - Aav-опосредованная экспрессия с использованием синтетического промотора и энхансера - Google Patents

Aav-опосредованная экспрессия с использованием синтетического промотора и энхансера

Info

Publication number
EA201892006A1
EA201892006A1 EA201892006A EA201892006A EA201892006A1 EA 201892006 A1 EA201892006 A1 EA 201892006A1 EA 201892006 A EA201892006 A EA 201892006A EA 201892006 A EA201892006 A EA 201892006A EA 201892006 A1 EA201892006 A1 EA 201892006A1
Authority
EA
Eurasian Patent Office
Prior art keywords
enchanser
aav
synthetic promoter
mediated expression
promoter
Prior art date
Application number
EA201892006A
Other languages
English (en)
Inventor
Джон Ф. Энгельхард
Цзыин Янь
Original Assignee
Юниверсити Оф Айова Рисерч Фаундейшн
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 Юниверсити Оф Айова Рисерч Фаундейшн filed Critical Юниверсити Оф Айова Рисерч Фаундейшн
Publication of EA201892006A1 publication Critical patent/EA201892006A1/ru

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Confectionery (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)

Abstract

Изобретение относится к выделенному рекомбинантному парвовирусному вектору, который содержит синтетический энхансер, содержащий множество энхансерных последовательностей, функционально связанный с промотором, и к способам применения этого вектора.
EA201892006A 2016-03-07 2017-03-07 Aav-опосредованная экспрессия с использованием синтетического промотора и энхансера EA201892006A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662304656P 2016-03-07 2016-03-07
PCT/US2017/021124 WO2017155973A1 (en) 2016-03-07 2017-03-07 Aav-mediated expression using a synthetic promoter and enhancer

Publications (1)

Publication Number Publication Date
EA201892006A1 true EA201892006A1 (ru) 2019-04-30

Family

ID=58361143

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201892006A EA201892006A1 (ru) 2016-03-07 2017-03-07 Aav-опосредованная экспрессия с использованием синтетического промотора и энхансера

Country Status (9)

Country Link
US (2) US11684679B2 (ru)
EP (1) EP3426787A1 (ru)
AU (3) AU2017229347A1 (ru)
CA (2) CA3016985C (ru)
EA (1) EA201892006A1 (ru)
IL (1) IL261642A (ru)
MA (1) MA43735A (ru)
MX (1) MX2018010842A (ru)
WO (1) WO2017155973A1 (ru)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11999965B2 (en) 2017-01-13 2024-06-04 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
ES2878451T3 (es) 2014-11-14 2021-11-18 Voyager Therapeutics Inc Polinucleótidos moduladores
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2020214672A1 (en) * 2019-04-15 2020-10-22 University Of Iowa Research Foundation Methods and compositions for transgene expression
CA3137015A1 (en) * 2019-04-15 2020-10-22 University Of Iowa Research Foundation Compositions and methods for treatment of cystic fibrosis
WO2022006253A2 (en) * 2020-06-30 2022-01-06 University Of Iowa Research Foundation Methods and compositions for administering recombinant viral vectors
EP4036238A1 (en) 2021-01-27 2022-08-03 The University Court of the University of Edinburgh Functional nucleic acid molecule and method
GB202211123D0 (en) 2022-07-29 2022-09-14 Univ Edinburgh Cancer treatment

Family Cites Families (143)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4487934A (en) 1980-06-06 1984-12-11 Hoechst-Roussel Pharmaceuticals Inc. Method of preparing poly-substituted acylbenzenes
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
DE3326472A1 (de) 1983-07-22 1985-02-14 Hoechst Ag, 6230 Frankfurt Neue insulin-derivate, verfahren zu deren herstellung und deren verwendung sowie pharmazeutische mittel zur behandlung des diabetes mellitus
EP0158255B1 (en) 1984-04-06 1989-01-04 ZAMBON S.p.A. Optically active ketals, processes for their preparation and their use in the synthesis of apha-arylakanoic acids
US5658772A (en) 1989-12-22 1997-08-19 E. I. Du Pont De Nemours And Company Site-specific recombination of DNA in plant cells
KR930701992A (ko) 1990-10-05 1993-09-08 웨인 알.조운즈 환자의 폐에 잔존하는 점액 분비물의 제거 방법
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5292498A (en) 1991-06-19 1994-03-08 The University Of North Carolina At Chapel Hill Method of treating lung disease with uridine triphosphates
US6083713A (en) 1992-08-31 2000-07-04 Bristol-Myers Squibb Company Cloning and expression of βAPP-C100 receptor (C100-R)
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
US5686436A (en) 1993-05-13 1997-11-11 Hiv Diagnostics, Inc. Multi-faceted method to repress reproduction of latent viruses in humans and animals
US5512269A (en) 1993-06-09 1996-04-30 Burroughs Wellcome, Co. Method of treating retained pulmonary secretions
US5498531A (en) 1993-09-10 1996-03-12 President And Fellows Of Harvard College Intron-mediated recombinant techniques and reagents
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5750398A (en) 1993-11-30 1998-05-12 David C. Johnson Vector, element and method for inhibiting immune recognition
WO1995022323A1 (en) 1994-02-16 1995-08-24 Idun Pharmaceuticals Methods for controlling free radical generation by inflammatory cells
US5604090A (en) 1994-06-06 1997-02-18 Fred Hutchinson Cancer Research Center Method for increasing transduction of cells by adeno-associated virus vectors
US5656256A (en) 1994-12-14 1997-08-12 The University Of North Carolina At Chapel Hill Methods of treating lung disease by an aerosol containing benzamil or phenamil
US5843742A (en) 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5635160A (en) 1995-06-07 1997-06-03 The University Of North Carolina At Chapel Hill Dinucleotides useful for the treatment of cystic fibrosis and for hydrating mucus secretions
US5853716A (en) 1995-07-28 1998-12-29 Yale University Genetically engineered chimeric viruses for the treatment of diseases associated with viral transactivators
US5628984A (en) 1995-07-31 1997-05-13 University Of North Carolina At Chapel Hill Method of detecting lung disease
AU7008896A (en) 1995-08-21 1997-03-12 Duke University A method to increase the density of antigen on antigen presenting cells
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US5855918A (en) 1995-09-12 1999-01-05 Genentech, Inc. Cystic fibrosis therapy
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
EP0883344B2 (en) 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing
DE19605279A1 (de) 1996-02-13 1997-08-14 Hoechst Ag Zielzellspezifische Vektoren für die Einschleusung von Genen in Zellen, Arzneimittel enthaltend derartige Vektoren und deren Verwendung
US6420347B1 (en) 1997-03-27 2002-07-16 Inspire Pharmaceuticals, Inc. Method of treating ciliary dyskinesia with uridine triphosphates and related compounds
US5939460A (en) 1996-07-08 1999-08-17 Idun Pharmaceuticals, Inc. Method of inhibiting NADPH oxidase
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
IL128779A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US6110744A (en) 1996-11-13 2000-08-29 Board Of Regents, The University Of Texas System Diminishing viral gene expression by promoter replacement
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
US6287569B1 (en) 1997-04-10 2001-09-11 The Regents Of The University Of California Vaccines with enhanced intracellular processing
FR2763845B1 (fr) 1997-05-30 2003-04-18 Centre Nat Rech Scient Produits anti-cancereux pour le traitement de la mucoviscidose
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6037177A (en) 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production
ID29198A (id) 1997-08-29 2001-08-09 Univ North Carolina Pemakaian uridin 5'-difosfat dan persamaannya untuk mengobati penyakit paru-paru
WO1999018227A1 (en) 1997-10-08 1999-04-15 Advanced Research And Technology Institute Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
EP1025243B1 (en) 1997-10-21 2009-07-01 Targeted Genetics Corporation TRANSCRIPTIONALLY-ACTIVATED AAV INVERTED TERMINAL REPEATS (ITRs) FOR USE WITH RECOMBINANT AAV VECTORS
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
CA2316414A1 (en) 1997-12-23 1999-07-01 Introgene B.V. Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
DE69905464T2 (de) 1998-05-22 2004-01-15 Inspire Pharmaceuticals Inc Therapeutische dinucleotide und deren derivate
ATE402254T1 (de) 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
IL128017A0 (en) 1998-07-22 1999-11-30 Technion Res & Dev Foundation Method for detecting protein-protein interactions and a kit therefor
US6290951B1 (en) 1998-08-01 2001-09-18 Alfacell Corporation Alteration of the cell cycle in vivo, and particularly for inducing apoptosis of tumor cells
ES2140354B1 (es) 1998-08-03 2000-11-01 S A L V A T Lab Sa Imidazo (1,2a) azinas sustituidas como inhibidores selectivos de la cox-2.
WO2000023023A1 (en) 1998-10-20 2000-04-27 The University Of North Carolina At Chapel Hill Methods of hydrating mucosal surfaces
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
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
CA2350415A1 (en) 1998-11-18 2000-05-25 Incyte Pharmaceuticals, Inc. Inflammation-associated genes
US6855549B1 (en) 1998-11-23 2005-02-15 The University Of Iowa Research Foundation Methods and compositions for increasing the infectivity of gene transfer vectors
US6926911B1 (en) 1998-12-22 2005-08-09 The University Of North Carolina At Chapel Hill Compounds and methods for the treatment of airway diseases and for the delivery of airway drugs
US6512102B1 (en) 1998-12-31 2003-01-28 Chiron Corporation Compositions and methods of diagnosis and treatment using casein kinase I
CA2362426A1 (en) 1999-02-10 2000-08-17 Takashi Tsuruo Anticancer drug enhancer
US6306663B1 (en) 1999-02-12 2001-10-23 Proteinex, Inc. Controlling protein levels in eucaryotic organisms
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
CA2270795A1 (en) 1999-05-05 2000-11-05 Gestilab Inc. Neuroprotective compositions and uses thereof
US7122335B1 (en) 1999-06-08 2006-10-17 University Of Iowa Research Foundation Compounds and methods to enhance rAAV transduction
JP4969002B2 (ja) 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
US6475509B1 (en) 1999-07-19 2002-11-05 The University Of North Carolina At Chapel Hill Covalent conjugates of sodium channel blockers and active compounds
US6475537B1 (en) 2000-07-27 2002-11-05 Rhodia Inc. Hops acid antibacterial compositions
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
US6416759B1 (en) 1999-09-30 2002-07-09 The Regents Of The University Of California Antiproliferative Sgk reagents and methods
WO2001025465A1 (en) 1999-10-07 2001-04-12 University Of Iowa Research Foundation Adeno-associated viruses and uses thereof
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
AU784420B2 (en) 1999-10-07 2006-03-30 University Of Iowa Research Foundation, The Adeno-associated viruses and uses thereof
WO2001027303A1 (en) 1999-10-12 2001-04-19 The University Of North Carolina At Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
WO2001029243A1 (en) 1999-10-15 2001-04-26 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
US20020132770A1 (en) 1999-10-27 2002-09-19 Caplan Michael T. Conductance of improperly folded proteins through the secretory pathway and related methods for treating disease
US7060497B2 (en) 2000-03-03 2006-06-13 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vector-based methods and compositions for introducing an expression cassette into a cell
CA2373110A1 (en) 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
CA2302627A1 (en) 2000-03-23 2001-09-23 Rashmi Kothary Constructs comprising the enac promoter sequence and a reporter gene for screening pharmaceuticals
US20020076754A1 (en) 2000-04-20 2002-06-20 Liangwu Sun Overcoming AAV vector size limitation through viral DNA hetero-dimerization
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
JP4860886B2 (ja) 2000-06-01 2012-01-25 ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二本鎖パルボウイルスベクター
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US6492429B1 (en) 2000-07-10 2002-12-10 N.V. Nutricia Composition for the treatment of osteoarthritis
US6485950B1 (en) 2000-07-14 2002-11-26 Council Of Scientific And Industrial Research Isozyme of autoclavable superoxide dismutase (SOD), a process for the identification and extraction of the SOD in cosmetic, food and pharmaceutical compositions
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
AU2001283278A1 (en) 2000-08-10 2002-02-25 Neurologix, Inc. Replication competent aav helper functions
US20020128203A1 (en) 2000-09-20 2002-09-12 Laurent Schild Methods of identifying inhibitory compounds and uses thereof
US7435427B2 (en) 2000-09-22 2008-10-14 Galephar M/F Pharmaceutical semi-solid composition of isotretinoin
AU2002211379A1 (en) 2000-10-04 2002-04-15 The Children's Hospital Of Philadelphia Compositions and methods for treatment of cystic fibrosis
US20030087818A1 (en) 2001-02-02 2003-05-08 Corixa Corporation Compositions and methods for the therapy and diagnosis of colon cancer
US20030013137A1 (en) 2001-03-13 2003-01-16 Barak Larry S. Automated methods of detecting receptor activity
WO2002085367A1 (en) 2001-04-19 2002-10-31 Uab Research Foundation Methods of treatment using an epithelial sodium channel blocker and an inhibitor of the mineralocorticoid receptor
US20040072254A1 (en) 2001-05-01 2004-04-15 Senomyx, Inc. High throughput cell-based assay for monitoring sodium channel activity and discovery of salty taste modulating compounds
US20030003583A1 (en) 2001-06-08 2003-01-02 Raphael Hirsch Regulation of transgene expression following AAV transduction
WO2003006614A2 (en) 2001-07-12 2003-01-23 Exelixis, Inc. UBE2s AS MODIFIERS OF THE p21 PATHWAY AND METHODS OF USE
CA2453405A1 (en) 2001-07-13 2003-01-23 University Of Iowa Research Foundation Pseudotyped adeno-associated viruses and uses thereof
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20030108920A1 (en) 2001-09-07 2003-06-12 Wei Zhang Tumor suppressor-like proteins that bind IGFBP2
JP2003201255A (ja) 2001-11-05 2003-07-18 Otsuka Pharmaceut Factory Inc アルツハイマー病予防および治療剤
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
AU2002360355B2 (en) 2001-11-09 2005-07-07 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003104392A2 (en) 2001-12-18 2003-12-18 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
AU2002364612A1 (en) 2001-12-31 2003-07-24 Algos Therapeutics, Inc. METHODS AND MATERIALS FOR MODULATING ENaC-BETA
WO2003087399A1 (en) 2002-04-17 2003-10-23 Glucox Ab Nad)p)h oxidase inhibitors for increased glucose uptake and treatment of type ii diabetes
ES2312780T3 (es) 2002-05-13 2009-03-01 Arexis Ab Afecciones autoinmunes y deficiencias de oxidasa nadph.
PT1534990E (pt) 2002-07-23 2007-02-28 Medi Physics Inc Sistemas automatizados de distribuição de gases hiperpolarizados, sensível a uma pressão dinâmica, métodos associados e produtos de programas de computadores
WO2004047772A2 (en) 2002-11-26 2004-06-10 Florida Atlantic University Catalytic antioxidants and methods of use
KR100522188B1 (ko) 2003-01-20 2005-10-18 주식회사 뉴로테크 뉴로트로핀에 의해 유도되는 세포괴사 억제 방법
EP1608763A2 (en) 2003-03-31 2005-12-28 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
PT2657247T (pt) 2003-06-19 2017-06-29 Genzyme Corp Viriões aav com imunorreatividade diminuída e seus usos
US7655467B2 (en) 2003-11-14 2010-02-02 The University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
US7407801B2 (en) 2003-12-05 2008-08-05 University Of Iowa Research Foundation Truncated CMV promoters and vectors containing same
US7067659B2 (en) 2004-04-23 2006-06-27 Duke University Reactive oxygen generating enzyme inhibitor with nitric oxide bioactivity and uses thereof
WO2005116224A2 (en) 2004-05-18 2005-12-08 Children's Memorial Hospital Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system
EP1602926A1 (en) 2004-06-04 2005-12-07 University of Geneva Novel means and methods for the treatment of hearing loss and phantom hearing
WO2006009975A2 (en) 2004-06-22 2006-01-26 Maxim Pharmaceuticals, Inc. Histamine to treat disorders affecting muscle function
WO2006116503A2 (en) 2005-04-26 2006-11-02 Irm Llc Methods and compositions for modulating wnt signaling pathway
US8110350B2 (en) 2005-11-17 2012-02-07 Karolinska Institutet Innovations Ab Human Bocavirus and methods of diagnosis and treatment
CA2634670A1 (en) 2005-12-30 2007-07-12 University Of Iowa Research Foundation Method of identifying compounds useful to treat neuronal degenerative diseases
WO2007127464A2 (en) 2006-04-28 2007-11-08 University Of Iowa Research Foundation Methods and compounds to modulate parvovirus transduction of mammalian cells or to alter virus infection, method to identify a viral receptor or co-receptor
EP2063904A1 (en) 2006-09-22 2009-06-03 Medizinische Hochschule Hannover Methods involving lef-1 regulation and use of lef-1 or compounds altering lef-1 signalling for treating or preventing diseases
JP5646990B2 (ja) 2007-04-23 2014-12-24 ストワーズ インスティテュート フォー メディカル リサーチ 幹細胞自己複製のための方法及び組成物
WO2009028387A1 (ja) 2007-08-24 2009-03-05 Kyowa Hakko Kirin Co., Ltd. プロテアーゼ阻害剤耐性を有する癌の治療薬
US20090241206A1 (en) 2008-03-04 2009-09-24 University Of Iowa Research Foundation Methods for cloning ferrets and transgenic ferret models for diseases
EP2268801A4 (en) 2008-03-14 2011-12-28 Us Gov Health & Human Serv COMPOSITIONS AND METHODS RELATED TO A HUMAN BOCAVIRUS
US8071751B2 (en) 2008-04-17 2011-12-06 Blood Systems, Inc. Human parvovirus: bocavirus
US20110054247A1 (en) 2009-03-04 2011-03-03 University Of Iowa Research Foundation Methods for cloning ferrets and transgenic ferret models for diseases
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
CN105431170B (zh) 2013-04-08 2019-10-29 爱荷华大学研究基金会 嵌合腺相关病毒/博卡病毒细小病毒载体
US20170049887A1 (en) 2014-04-25 2017-02-23 University Of Florida Research Foundation, Inc. Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus
AU2015342997B2 (en) 2014-11-05 2021-11-18 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
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
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
WO2019178267A2 (en) 2018-03-13 2019-09-19 University Of Iowa Research Foundation Inductive regeneration of the airway by transcriptional factor modulation of glandular myoepithelial stem cells
CA3137015A1 (en) 2019-04-15 2020-10-22 University Of Iowa Research Foundation Compositions and methods for treatment of cystic fibrosis
WO2020214672A1 (en) 2019-04-15 2020-10-22 University Of Iowa Research Foundation Methods and compositions for transgene expression
EA202192818A1 (ru) 2020-01-29 2022-03-03 Юниверсити Оф Айова Рисерч Фаундейшн Композиции и способы лечения муковисцидоза
EA202192819A1 (ru) 2020-01-29 2022-02-18 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для экспрессии трансгена
WO2022006253A2 (en) 2020-06-30 2022-01-06 University Of Iowa Research Foundation Methods and compositions for administering recombinant viral vectors

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
US11999965B2 (en) 2017-01-13 2024-06-04 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof

Also Published As

Publication number Publication date
EP3426787A1 (en) 2019-01-16
MX2018010842A (es) 2019-07-04
CA3016985C (en) 2023-07-04
US20240066147A1 (en) 2024-02-29
AU2020289851B2 (en) 2023-08-10
WO2017155973A1 (en) 2017-09-14
MA43735A (fr) 2018-11-28
US20190083657A1 (en) 2019-03-21
AU2023263461A1 (en) 2023-11-30
AU2017229347A1 (en) 2018-11-01
CA3016985A1 (en) 2017-09-14
IL261642A (en) 2018-10-31
CA3198936A1 (en) 2017-09-14
US11684679B2 (en) 2023-06-27
AU2020289851A1 (en) 2021-01-28

Similar Documents

Publication Publication Date Title
EA201892006A1 (ru) Aav-опосредованная экспрессия с использованием синтетического промотора и энхансера
EA201890640A1 (ru) Рекомбинантные векторы, содержащие пептид 2а
ZA202000842B (en) Improved dual specificity polypeptide molecule
EA201890093A1 (ru) Комбинированная терапия гемобластозов антителами к cd38 и ингибиторами сурвивина
EA201791117A1 (ru) Частичные агонисты инсулинового рецептора
EA201891338A1 (ru) Композиции и способы для иммуноонкологии
NZ731673A (en) Aav vectors targeted to the central nervous system
EA201791046A8 (ru) Макроциклические пептиды, используемые в качестве иммуномодуляторов
MX2017016811A (es) Metodos para mejorar el rendimiento del animal.
EA202092609A1 (ru) Антитела к cd38 для лечения острого миелолейкоза
TR201901582T4 (tr) Kapsid
CY1124761T1 (el) Ρυθμιστες της δραστικοτητας του συμπληρωματος
MX2022013454A (es) Moduladores de la actividad del complemento.
EA201890052A1 (ru) Регуляторы nrf2
EA201992232A1 (ru) Композиции и способы для иммуноонкологии
EA201792080A1 (ru) Гуманизированные анти-c1s антитела и способы их применения
TR201910057T4 (tr) Kauçuk bi̇leşi̇mleri̇
EA201890351A1 (ru) Рекомбинантный orf-вирусный вектор
EA201791249A1 (ru) Получаемые из cd44v6 циклические пептиды для лечения злокачественных опухолей и связанных с ангиогенезом заболеваний
EA201691632A1 (ru) Пиразины в качестве модуляторов gpr6
CL2019003167A1 (es) Proteínas de unión al antígeno anti-jagged1.
EA201692569A1 (ru) Полимиксины с низкой степенью замещения и композиции, их содержащие
NO341559B1 (en) Synthetic capsaicinoid derivatives and feed comprising such compounds as growth promotors
EA201790241A1 (ru) Эффективная селективность в отношении рекомбинантных белков
EA201790428A1 (ru) Синтез энт-прогестерона и его промежуточных соединений