WO2003092594A3 - Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use - Google Patents
Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use Download PDFInfo
- Publication number
- WO2003092594A3 WO2003092594A3 PCT/US2003/013323 US0313323W WO03092594A3 WO 2003092594 A3 WO2003092594 A3 WO 2003092594A3 US 0313323 W US0313323 W US 0313323W WO 03092594 A3 WO03092594 A3 WO 03092594A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- aav
- region
- adenovirus
- vector genome
- functional
- Prior art date
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- A61K48/0083—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 administration regime
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
- C12N9/2411—Amylases
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/525—Virus
- A61K2039/5256—Virus expressing foreign proteins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10344—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
Abstract
Priority Applications (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
AU2003223775A AU2003223775A1 (en) | 2002-04-30 | 2003-04-30 | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
US10/511,980 US7858367B2 (en) | 2002-04-30 | 2003-04-30 | Viral vectors and methods for producing and using the same |
US12/976,334 US20110294193A1 (en) | 2002-04-30 | 2010-12-22 | Viral vectors and methods for producing and using the same |
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US37639702P | 2002-04-30 | 2002-04-30 | |
US60/376,397 | 2002-04-30 |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US12/976,334 Division US20110294193A1 (en) | 2002-04-30 | 2010-12-22 | Viral vectors and methods for producing and using the same |
Publications (3)
Publication Number | Publication Date |
---|---|
WO2003092594A2 WO2003092594A2 (en) | 2003-11-13 |
WO2003092594A3 true WO2003092594A3 (en) | 2004-09-10 |
WO2003092594A8 WO2003092594A8 (en) | 2004-11-25 |
Family
ID=29401341
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2003/013323 WO2003092594A2 (en) | 2002-04-30 | 2003-04-30 | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
Country Status (3)
Country | Link |
---|---|
US (2) | US7858367B2 (en) |
AU (1) | AU2003223775A1 (en) |
WO (1) | WO2003092594A2 (en) |
Families Citing this family (43)
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ATE521701T1 (en) | 2003-01-22 | 2011-09-15 | Univ Duke | IMPROVED CONSTRUCTS FOR EXPRESSING LYSOSOMAL POLYPEPTIDES |
US9441244B2 (en) * | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
WO2005016250A2 (en) * | 2003-08-08 | 2005-02-24 | Genzyme Corporation | Cell therapy for neurometabolic disorders |
CA2836987C (en) | 2004-05-26 | 2016-07-05 | Psioxus Therapeutics Limited | Chimeric adenoviruses for use in cancer treatment |
CA2680063A1 (en) * | 2007-02-23 | 2008-08-28 | University Of Florida Research Foundation, Inc. | Compositions and methods for treating glycogen storage diseases |
US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
CA2833870C (en) | 2011-04-22 | 2020-03-10 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
WO2013096955A1 (en) * | 2011-12-23 | 2013-06-27 | Case Western Reserve University | Targeted gene modification using hybrid recombinant adeno-associated virus |
TWI702955B (en) | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | Treatment of amd using aav sflt-1 |
JP2015523998A (en) | 2012-06-19 | 2015-08-20 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | Compositions and methods for treating diseases |
CA2907799A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
CA2927968C (en) | 2013-10-25 | 2023-02-21 | Psioxus Therapeutics Limited | Oncolytic adenoviruses armed with heterologous genes |
US10000741B2 (en) | 2014-03-17 | 2018-06-19 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
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MX2017012097A (en) | 2015-03-24 | 2018-06-15 | Univ California | Adeno-associated virus variants and methods of use thereof. |
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CA3019315A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
PT3288573T (en) | 2015-04-30 | 2020-03-25 | Psioxus Therapeutics Ltd | Oncolytic adenovirus encoding a b7 protein |
GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
CA2995110A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
AU2016372576A1 (en) | 2015-12-17 | 2018-06-21 | Psioxus Therapeutics Limited | Virus encoding an anti-TCR-complex antibody or fragment |
GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
CA3011894A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
CA3021057A1 (en) | 2016-04-15 | 2017-10-19 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy |
ES2908467T3 (en) | 2016-04-29 | 2022-04-29 | Adverum Biotechnologies Inc | Evasion of neutralizing antibodies by a recombinant adeno-associated virus |
WO2018011572A1 (en) * | 2016-07-12 | 2018-01-18 | The University Of Manchester | Gene therapy |
EP3827812A1 (en) | 2016-07-29 | 2021-06-02 | The Regents of the University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
GB201713765D0 (en) | 2017-08-28 | 2017-10-11 | Psioxus Therapeutics Ltd | Modified adenovirus |
AU2017345470B2 (en) | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
US10443055B2 (en) | 2016-12-22 | 2019-10-15 | Beth Israel Deaconess Medical Center | Compounds that target MYC microRNA responsive elements for the treatment of MYC-associated cancer |
KR102616820B1 (en) | 2017-03-17 | 2023-12-21 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | Compositions and methods for enhanced gene expression |
NZ760232A (en) | 2017-06-07 | 2023-05-26 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
AU2018324477A1 (en) | 2017-08-28 | 2019-10-17 | The Regents Of The University Of California | Adeno-associated virus capsid variants and methods of use thereof |
JP2021533762A (en) | 2018-08-10 | 2021-12-09 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | Modified oligonucleotides that target SNPs |
CN114072501A (en) | 2019-05-06 | 2022-02-18 | 马萨诸塞大学 | anti-C9 ORF72 oligonucleotides and related methods |
CN112301058B (en) * | 2019-07-30 | 2023-02-21 | 上海市第一人民医院 | Recombinant adeno-associated virus vector and preparation method and application thereof |
AU2021411580A1 (en) * | 2020-12-30 | 2023-07-13 | Duke University | Compositions for and methods of treating and/or preventing glycogen storage disease type vi and type ix |
EP4359539A1 (en) | 2021-06-23 | 2024-05-01 | University Of Massachusetts | Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
US20230346977A1 (en) | 2022-04-13 | 2023-11-02 | Universitat Autònoma De Barcelona | Treatment of neuromuscular diseases via gene therapy that expresses klotho protein |
Citations (3)
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US5962313A (en) * | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
WO2000011149A1 (en) * | 1998-08-24 | 2000-03-02 | Uab Research Foundation | Methods of producing high titer recombinant adeno-associated virus |
US6251677B1 (en) * | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
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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 |
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-
2003
- 2003-04-30 AU AU2003223775A patent/AU2003223775A1/en not_active Abandoned
- 2003-04-30 WO PCT/US2003/013323 patent/WO2003092594A2/en not_active Application Discontinuation
- 2003-04-30 US US10/511,980 patent/US7858367B2/en not_active Expired - Fee Related
-
2010
- 2010-12-22 US US12/976,334 patent/US20110294193A1/en not_active Abandoned
Patent Citations (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5962313A (en) * | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
US6251677B1 (en) * | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
WO2000011149A1 (en) * | 1998-08-24 | 2000-03-02 | Uab Research Foundation | Methods of producing high titer recombinant adeno-associated virus |
Non-Patent Citations (4)
Title |
---|
DING ET AL: "Long-term efficacy after (E1-, polymerase-) adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice", HUMAN GENE THERAPY, vol. 12, 20 May 2001 (2001-05-20), pages 955 - 965, XP002978924 * |
LIEBER ET AL: "Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes", JOURNAL OF VIROLOGY, vol. 73, no. 11, November 1999 (1999-11-01), pages 9314 - 9324, XP002152920 * |
PAULY ET AL: "Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy pompe disease", HUMAN GENE THERAPY, vol. 12, 20 March 2001 (2001-03-20), pages 527 - 538, XP002978923 * |
SUN ET AL: "Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector", MOLECULAR THERAPY, vol. 7, no. 2, February 2003 (2003-02-01), pages 193 - 201, XP002978925 * |
Also Published As
Publication number | Publication date |
---|---|
US20110294193A1 (en) | 2011-12-01 |
US7858367B2 (en) | 2010-12-28 |
WO2003092594A8 (en) | 2004-11-25 |
AU2003223775A1 (en) | 2003-11-17 |
US20050220766A1 (en) | 2005-10-06 |
WO2003092594A2 (en) | 2003-11-13 |
AU2003223775A8 (en) | 2003-11-17 |
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