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

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/38Vector systems having a special element relevant for transcription being a stuffer

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IN74KON2015 2012-07-06 2013-03-14 IN2015KN00074A (enExample)

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 (enExample) 2015-07-31

Family

ID=49882407

Family Applications (1)

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

Country Status (12)

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

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IN2015KN00074A (enExample) * 2012-07-06 2015-07-31 Univ Iowa Res Found
JP6663859B2 (ja) * 2014-05-20 2020-03-13 ユニバーシティー オブ アイオワ リサーチ ファウンデーション ハンチントン病の治療化合物
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
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
WO2017161273A1 (en) * 2016-03-18 2017-09-21 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
US11299751B2 (en) 2016-04-29 2022-04-12 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
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics, Inc. composições e métodos de tratamento da doença de huntington
WO2018044933A1 (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
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
CN111712569A (zh) * 2017-12-11 2020-09-25 爱迪塔斯医药公司 用于基因编辑的Cpf1-相关方法和组合物
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US20210254103A1 (en) * 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP7698583B2 (ja) 2019-03-21 2025-06-25 ギンコ バイオワークス インコーポレイテッド 組換えアデノ随伴ウイルスベクター
MX2021013913A (es) * 2019-05-14 2022-03-17 Univ Duke Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa.
AR122285A1 (es) * 2019-07-18 2022-08-31 Lysogene Composiciones y métodos para el tratamiento de la enfermedad de sanfilippo y otros trastornos
IL292297A (en) 2019-10-17 2022-06-01 Stridebio 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
CN115768900A (zh) * 2020-05-11 2023-03-07 戈迪安生物技术公司 病毒递送媒剂选择
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
KR20230142576A (ko) * 2021-02-05 2023-10-11 메이즈 테라퓨틱스, 인코퍼레이티드 스터퍼 폴리뉴클레오티드 서열을 포함하는 벡터
US20240218397A1 (en) * 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
CN119654414A (zh) * 2022-08-09 2025-03-18 谢菲尔德大学 病毒载体

Family Cites Families (12)

* Cited by examiner, † Cited by third party
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
WO2001032899A1 (en) * 1999-10-29 2001-05-10 Takara Shuzo Co., Ltd. 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
CA2543189A1 (en) * 2003-10-20 2005-05-06 Nsgene A/S 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 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
IN2015KN00074A (enExample) * 2012-07-06 2015-07-31 Univ Iowa Res Found

Also Published As

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

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