IN2015KN00074A - - Google Patents

Info

Publication number
IN2015KN00074A
IN2015KN00074A IN74KON2015A IN2015KN00074A IN 2015KN00074 A IN2015KN00074 A IN 2015KN00074A IN 74KON2015 A IN74KON2015 A IN 74KON2015A IN 2015KN00074 A IN2015KN00074 A IN 2015KN00074A
Authority
IN
India
Prior art keywords
adeno
nucleotides
disclosed
length
nucleic acid
Prior art date
Application number
Other languages
English (en)
Inventor
Beverly L Davidson
Maria Scheel
Ryan Boudreau
Original Assignee
Univ Iowa Res Found
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 Iowa Res Found filed Critical Univ Iowa Res Found
Publication of IN2015KN00074A publication Critical patent/IN2015KN00074A/en

Links

Classifications

    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
IN74KON2015 2012-07-06 2013-03-14 IN2015KN00074A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261668839P 2012-07-06 2012-07-06
PCT/US2013/031644 WO2014007858A1 (en) 2012-07-06 2013-03-14 Modified adeno-associated virus vector compositions

Publications (1)

Publication Number Publication Date
IN2015KN00074A true IN2015KN00074A (zh) 2015-07-31

Family

ID=49882407

Family Applications (1)

Application Number Title Priority Date Filing Date
IN74KON2015 IN2015KN00074A (zh) 2012-07-06 2013-03-14

Country Status (12)

Country Link
US (2) US9540659B2 (zh)
EP (1) EP2870237B1 (zh)
JP (1) JP6366581B2 (zh)
CN (1) CN104619832B (zh)
AU (1) AU2013287261B2 (zh)
BR (1) BR112015000161A2 (zh)
CA (1) CA2878401C (zh)
ES (1) ES2703177T3 (zh)
HK (1) HK1210214A1 (zh)
IN (1) IN2015KN00074A (zh)
RU (1) RU2679843C2 (zh)
WO (1) WO2014007858A1 (zh)

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ES2703177T3 (es) * 2012-07-06 2019-03-07 Univ Iowa Res Found Composiciones de vector viral adenoasociado modificado
CA2949437C (en) * 2014-05-20 2023-08-15 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
JP6401871B2 (ja) 2014-11-05 2018-10-10 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． パーキンソン病の治療のためのａａｄｃポリヌクレオチド
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US20190000940A1 (en) * 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
AU2017234929A1 (en) 2016-03-18 2018-10-04 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017201258A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
EP3458588A4 (en) 2016-05-18 2020-01-15 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
EP3831281A1 (en) 2016-08-30 2021-06-09 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
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
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
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
JP2021529513A (ja) * 2018-07-02 2021-11-04 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および脊髄に関連する障害の治療
JP2022531809A (ja) * 2019-05-14 2022-07-11 デューク ユニバーシティ ＡＴＰａｓｅ媒介性疾患の処置のための組成物および方法
US11905523B2 (en) 2019-10-17 2024-02-20 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
EP4150102A2 (en) * 2020-05-11 2023-03-22 Gordian Biotechnology, Inc. Viral delivery vehicle selection
US20240093189A1 (en) * 2021-02-05 2024-03-21 Maze Therapeutics, Inc. Vectors comprising stuffer polynucleotide sequences
WO2022235614A2 (en) * 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
WO2024033329A2 (en) * 2022-08-09 2024-02-15 University Of Sheffield Viral vector

Family Cites Families (12)

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Publication number Priority date Publication date Assignee Title
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
JPH1033175A (ja) * 1996-07-24 1998-02-10 Hisamitsu Pharmaceut Co Inc 組換えアデノ随伴ウイルスベクターの製造方法
US6171855B1 (en) * 1998-05-28 2001-01-09 The Regents Of The University Of Michigan Vectors
US6943153B1 (en) 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US6995010B1 (en) * 1999-10-29 2006-02-07 Takara Bio Inc. Gene transfer method
US7132277B1 (en) * 2000-01-31 2006-11-07 Merck & Co., Inc. Helper dependent vector system for gene therapy
US7387896B2 (en) 2003-03-26 2008-06-17 The Regents Of The University Of Michigan MicroRNA vectors
DE602004025959D1 (de) * 2003-10-20 2010-04-22 Nsgene As Viraler Vektor zur Verwendung in der in-vivo-Gentherapie von Morbus Parkinson
WO2009134681A2 (en) * 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
CA2833905C (en) * 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
US9181544B2 (en) 2011-02-12 2015-11-10 University Of Iowa Research Foundation Therapeutic compounds
ES2703177T3 (es) * 2012-07-06 2019-03-07 Univ Iowa Res Found Composiciones de vector viral adenoasociado modificado

Also Published As

Publication number Publication date
ES2703177T3 (es) 2019-03-07
AU2013287261A1 (en) 2015-01-22
CA2878401A1 (en) 2014-01-09
WO2014007858A1 (en) 2014-01-09
JP2015522280A (ja) 2015-08-06
US9540659B2 (en) 2017-01-10
RU2015103901A (ru) 2016-08-27
EP2870237A1 (en) 2015-05-13
JP6366581B2 (ja) 2018-08-08
BR112015000161A2 (pt) 2017-06-27
CN104619832B (zh) 2021-03-16
CA2878401C (en) 2022-07-19
US20150184197A1 (en) 2015-07-02
EP2870237A4 (en) 2015-12-23
RU2679843C2 (ru) 2019-02-13
US20170306355A1 (en) 2017-10-26
AU2013287261B2 (en) 2019-03-07
CN104619832A (zh) 2015-05-13
EP2870237B1 (en) 2018-09-26
HK1210214A1 (zh) 2016-04-15

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