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
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

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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

Abstract

An adeno-associated virus filler component comprising a nucleic acid of between 3300 and 4200 nucleotides in length is disclosed.
IN74KON2015 2012-07-06 2013-03-14 IN2015KN00074A (en)

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 (en) 2015-07-31

Family

ID=49882407

Family Applications (1)

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

Country Status (12)

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

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EP2870237B1 (en) * 2012-07-06 2018-09-26 University of Iowa Research Foundation Modified adeno-associated virus vector compositions
ES2759317T3 (en) * 2014-05-20 2020-05-08 Univ Iowa Res Found Therapeutic compounds for Huntington's disease
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11697825B2 (en) 2014-12-12 2023-07-11 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
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
KR102427379B1 (en) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. Compositions and methods for treating Huntington's disease
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP2020518258A (en) 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． Amyotrophic lateral sclerosis (ALS) treatment composition and method
CN111108198A (en) 2017-05-05 2020-05-05 沃雅戈治疗公司 Compositions and methods for treating huntington's disease
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3697905A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. 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
JP7244547B2 (en) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Adeno-associated virus compositions and methods of use thereof for restoring F8 gene function
CN112567035A (en) * 2018-07-02 2021-03-26 沃雅戈治疗公司 Treatment of amyotrophic lateral sclerosis and spinal cord related disorders
EP3969597A4 (en) * 2019-05-14 2023-01-25 Duke University Compositions and methods for the treatment of atpase-mediated diseases
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
EP4150102A2 (en) * 2020-05-11 2023-03-22 Gordian Biotechnology, Inc. Viral delivery vehicle selection
AU2022218196A1 (en) * 2021-02-05 2023-08-10 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
GB2623164A (en) * 2022-08-09 2024-04-10 Univ Sheffield Viral vector

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JPH1033175A (en) * 1996-07-24 1998-02-10 Hisamitsu Pharmaceut Co Inc Production of recombinant adeno-accompanying virus vector
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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
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US7387896B2 (en) * 2003-03-26 2008-06-17 The Regents Of The University Of Michigan MicroRNA vectors
NZ547185A (en) * 2003-10-20 2009-03-31 Nsgene As In vivo gene therapy of parkinson's disease
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
ES2739804T3 (en) 2011-02-12 2020-02-04 Univ Iowa Res Found Therapeutic compounds
EP2870237B1 (en) * 2012-07-06 2018-09-26 University of Iowa Research Foundation Modified adeno-associated virus vector compositions

Also Published As

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

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