JP7066635B2 - 調節性ポリヌクレオチド - Google Patents

調節性ポリヌクレオチド Download PDF

Info

Publication number
JP7066635B2
JP7066635B2 JP2018560625A JP2018560625A JP7066635B2 JP 7066635 B2 JP7066635 B2 JP 7066635B2 JP 2018560625 A JP2018560625 A JP 2018560625A JP 2018560625 A JP2018560625 A JP 2018560625A JP 7066635 B2 JP7066635 B2 JP 7066635B2
Authority
JP
Japan
Prior art keywords
nucleic acid
acid sequence
sequence encoding
flanking region
region
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2018560625A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019519218A (ja
JP2019519218A5 (enExample
Inventor
ウェン－イー サー、ディナー
ホウ、ジンジャオ
ノンネンマッチャー、マシュー
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Voyager Therapeutics Inc
Original Assignee
Voyager Therapeutics Inc
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 Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of JP2019519218A publication Critical patent/JP2019519218A/ja
Publication of JP2019519218A5 publication Critical patent/JP2019519218A5/ja
Priority to JP2022072798A priority Critical patent/JP7374254B2/ja
Application granted granted Critical
Publication of JP7066635B2 publication Critical patent/JP7066635B2/ja
Priority to JP2023182415A priority patent/JP2023182824A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
JP2018560625A 2016-05-18 2017-05-18 調節性ポリヌクレオチド Active JP7066635B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022072798A JP7374254B2 (ja) 2016-05-18 2022-04-27 調節性ポリヌクレオチド
JP2023182415A JP2023182824A (ja) 2016-05-18 2023-10-24 調節性ポリヌクレオチド

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662338137P 2016-05-18 2016-05-18
US62/338,137 2016-05-18
US201762485050P 2017-04-13 2017-04-13
US62/485,050 2017-04-13
PCT/US2017/033268 WO2017201248A1 (en) 2016-05-18 2017-05-18 Modulatory polynucleotides

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022072798A Division JP7374254B2 (ja) 2016-05-18 2022-04-27 調節性ポリヌクレオチド

Publications (3)

Publication Number Publication Date
JP2019519218A JP2019519218A (ja) 2019-07-11
JP2019519218A5 JP2019519218A5 (enExample) 2020-06-25
JP7066635B2 true JP7066635B2 (ja) 2022-05-13

Family

ID=60325551

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018560625A Active JP7066635B2 (ja) 2016-05-18 2017-05-18 調節性ポリヌクレオチド
JP2022072798A Active JP7374254B2 (ja) 2016-05-18 2022-04-27 調節性ポリヌクレオチド
JP2023182415A Pending JP2023182824A (ja) 2016-05-18 2023-10-24 調節性ポリヌクレオチド

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022072798A Active JP7374254B2 (ja) 2016-05-18 2022-04-27 調節性ポリヌクレオチド
JP2023182415A Pending JP2023182824A (ja) 2016-05-18 2023-10-24 調節性ポリヌクレオチド

Country Status (13)

Country Link
US (4) US10584337B2 (enExample)
EP (1) EP3458588B1 (enExample)
JP (3) JP7066635B2 (enExample)
KR (3) KR102392236B1 (enExample)
CN (2) CN110214187B (enExample)
AU (2) AU2017267665C1 (enExample)
CA (2) CA3024448C (enExample)
IL (2) IL302748B2 (enExample)
MX (2) MX2018014154A (enExample)
RU (1) RU2758488C2 (enExample)
SG (1) SG11201809699XA (enExample)
WO (1) WO2017201248A1 (enExample)
ZA (1) ZA201807401B (enExample)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230145206A (ko) * 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
US10584337B2 (en) * 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
MX2020010465A (es) * 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
CN119390787A (zh) 2019-01-31 2025-02-07 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
WO2020223232A1 (en) * 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
AU2020265110A1 (en) * 2019-05-02 2021-11-25 Boehringer Ingelheim International Gmbh Viral vectors and nucleic acids for use in the treatment of PF-ILD and IPF
CN114072500B (zh) * 2019-08-02 2024-05-10 香港科技大学 控制microRNA表达的方法
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
JP7853297B2 (ja) * 2020-11-04 2026-04-28 ベーリンガー インゲルハイム インターナショナル ゲゼルシャフト ミット ベシュレンクテル ハフツング Ｉｌｄ、ｐｆ－ｉｌｄおよびｉｐｆ治療用ウイルスベクターおよび核酸
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2023214512A1 (ja) * 2022-05-06 2023-11-09 学校法人常翔学園 人工ｒｎａ分子
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
CN120082552A (zh) * 2023-12-01 2025-06-03 艾码生物科技(南京)有限公司 初级-miRNA及其应用
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025228394A1 (zh) * 2024-04-30 2025-11-06 艾码生物科技(南京)有限公司 递送初级微小rna的方法及其应用
WO2026101977A1 (en) 2024-11-06 2026-05-15 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110020816A1 (en) 2008-06-20 2011-01-27 Chang-Zheng Chen Precursor miRNA loop-modulated target regulation
WO2013126605A1 (en) 2012-02-21 2013-08-29 The Johns Hopkins University EXPRESSION CONSTRUCT FOR A LIN28-RESISTANT Let-7 PRECURSOR MICRORNA
WO2014016817A2 (en) 2012-07-17 2014-01-30 Universite De Geneve Nucleic acids for down-regulation of gene expression

Family Cites Families (513)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
AU7906691A (en) 1990-05-23 1991-12-10 United States of America, as represented by the Secretary, U.S. Department of Commerce, The Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
JPH10501686A (ja) 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのｄｎａのａａｖ仲介送達
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5625048A (en) 1994-11-10 1997-04-29 The Regents Of The University Of California Modified green fluorescent proteins
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
WO1998010086A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6083716A (en) 1996-09-06 2000-07-04 The Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
EP0968284B1 (en) 1996-11-20 2006-12-13 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral vectors
CA2270285A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by 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
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU9774998A (en) 1997-09-19 1999-04-12 Trustees Of The University Of Pennsylvania, The Method for gene transfer using bcl2 and compositions useful therein
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
JP2001517454A (ja) 1997-09-19 2001-10-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株
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
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
WO1999032647A1 (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
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
WO1999043360A1 (en) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6521426B1 (en) 1998-04-08 2003-02-18 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
EP1078096A1 (en) 1998-05-11 2001-02-28 Ariad Gene Therapeutics, Inc. Multiviral compositions and uses thereof
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1849872A1 (en) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
WO1999061595A2 (en) 1998-05-27 1999-12-02 Cell Genesys, Inc. Adeno-associated viral vector-mediated expression of factor viii activity
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
US6416992B1 (en) 1998-10-13 2002-07-09 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
WO2000024916A1 (en) 1998-10-27 2000-05-04 Crucell Holland B.V. Improved aav vector production
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
JP2002542805A (ja) 1999-04-30 2002-12-17 ユニバーシティ オブ フロリダ アデノ随伴ウイルス送達リボザイム組成物および使用方法
JP2003501067A (ja) 1999-06-02 2003-01-14 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法
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
AU781478B2 (en) 1999-08-20 2005-05-26 Johns Hopkins University School Of Medicine, The Methods and compositions for the construction and use of fusion libraries
WO2001023001A2 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Rapid peg-modification
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
EP1224313A1 (en) 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-associated viruses and uses thereof
WO2001032711A2 (en) 1999-10-21 2001-05-10 Board Of Trustees Of The University Of Arkansas Adeno-associated virus aav rep78 major regulatory protein, mutants thereof and uses thereof
AU3642601A (en) 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
WO2001036603A2 (en) 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
AU1954001A (en) 1999-12-10 2001-06-18 Ariad Gene Therapeutics, Inc. Methods for high level expression of genes in primates
EP1228234A2 (en) 2000-03-14 2002-08-07 Neurologix, Inc. Production of chimeric capsid vectors
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
US20020106381A1 (en) 2000-06-13 2002-08-08 High Katherine A. Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
EP1302542B1 (en) 2000-07-18 2007-06-13 Takeda Pharmaceutical Company Limited Novel physiologically active peptide and use thereof
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
ES2288993T3 (es) 2000-08-17 2008-02-01 Keiya Ozawa Distribucion de factores angiogenicos mediada por virus adenoasociados.
DE10066104A1 (de) 2000-09-08 2003-01-09 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2002153278A (ja) 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
WO2002070719A2 (en) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Regulatable gene expression system
FR2821624B1 (fr) 2001-03-01 2004-01-02 Sod Conseils Rech Applic Nouveau polynucleotide utilisable pour moduler la proliferation des cellules cancereuses
CA2441454A1 (en) 2001-03-14 2002-09-19 Avigen, Inc. Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions
AU2002252705A1 (en) * 2001-04-19 2002-11-05 Bristol-Myers Squibb Company Polynucleotides and polypeptides associated with the nf-kb pathway
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
US7344872B2 (en) 2001-06-22 2008-03-18 The Trustees Of The University Of Pennsylvania Method for rapid screening of bacterial transformants and novel simian adenovirus proteins
BR0211111A (pt) 2001-07-12 2004-06-22 Univ Massachusetts Molécula de ácido nucleico isolada, vetor, célula hospedeira, transgene, precursor de rna engenheirado, animal transgênico não humano, e, método de induzir a interferência de ácido ribonucleico de um gene alvo em uma célula
EP1900815B1 (en) 2001-07-12 2016-09-07 University of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1279740A1 (en) 2001-07-26 2003-01-29 Vrije Universiteit Brussel Recombinant vector derived from adeno-associated virus for gene therapy
EP1427835B1 (en) 2001-08-08 2016-01-20 The Trustees of the University of Pennsylvania Method for purification of viral vectors having proteins which bind sialic acid
US20030092161A1 (en) 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
EP2428570B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
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
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
EP1944043A1 (en) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
MXPA04004876A (es) 2001-11-21 2004-07-30 Univ Pennsylvania Secuencias de acido nucleico y de aminoacido de adenovirus de simio, vectores que contienen los mismos y metodos de uso.
CA2469491A1 (en) 2001-12-12 2003-06-19 Kerrie Setiawan Composition for viral preservation
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
CA2469658A1 (en) 2001-12-19 2003-07-03 Lijun Wang Adeno-associated virus-mediated delivery of gdnf to skeletal muscles
CN1617938A (zh) 2002-01-16 2005-05-18 戴诺生物技术有限公司 从单个样品中分离核酸和蛋白质的方法
DK2348119T3 (en) 2002-02-01 2017-08-21 Oxford Biomedica (Uk) Ltd Polycistronic lentiviral vector
US20030180756A1 (en) 2002-03-21 2003-09-25 Yang Shi Compositions and methods for suppressing eukaryotic gene expression
GB0208390D0 (en) 2002-04-11 2002-05-22 Univ London Adeno-associated virus producer system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US7235393B2 (en) 2002-04-29 2007-06-26 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues
DE60327069D1 (de) 2002-04-30 2009-05-20 Oncolytics Biotech Inc Verbesserte reinigungsmethode für viren
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
CA2524569C (en) 2002-05-03 2013-10-22 Duke University A method of regulating gene expression
EP1506287B1 (en) 2002-05-14 2007-04-25 Merck & Co., Inc. Methods of adenovirus purification
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
EP2801614B1 (en) 2002-08-29 2019-05-15 The Board of Trustees of the Leland Stanford Junior University Circular nucleic acid vectors and methods for making and using the same
EP1546344A4 (en) 2002-09-18 2007-10-03 Isis Pharmaceuticals Inc EFFICIENT PRODUCTION OF TARGET RNAS USING SINGLE AND DOUBLE-STRONG OLIGOMER COMPOUNDS
EP1418185A1 (en) 2002-11-11 2004-05-12 Aventis Pharma Deutschland GmbH Use of EDG2 receptor in an animal model of heart failure
US7169612B2 (en) 2002-11-11 2007-01-30 Sanofi-Aventis Deutschland Gmbh Use of EDG2 receptor in an animal model of heart failure
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US20080318210A1 (en) 2003-08-27 2008-12-25 Rosetta Genomics Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof
JP2006523103A (ja) 2003-02-21 2006-10-12 ザ ペン ステイト リサーチ ファウンデーション Ｒｎａ干渉組成物および方法
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
WO2005001103A2 (en) 2003-06-20 2005-01-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
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
ES2391975T3 (es) 2003-07-25 2012-12-03 Genvec, Inc. Vacunas a base de vector adenovírico
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
WO2005027980A1 (en) 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
US20050064489A1 (en) 2003-09-24 2005-03-24 Zhang Fang Liang Engineered U6 and H1 promoters
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
ES2687645T3 (es) 2003-10-27 2018-10-26 Merck Sharp & Dohme Corp. Método de diseño de ARNip para el silenciamiento de genes
ATE508190T1 (de) 2004-03-05 2011-05-15 Benitec Inc Mehrfachpromotor-expressionskassetten zurgleichzeitigen zuführung von rnai-agentien
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
ES2932278T3 (es) 2004-06-01 2023-01-17 Genzyme Corp Composiciones y métodos para prevenir la agregación del vector AAV
WO2006042090A1 (en) 2004-10-05 2006-04-20 Avigen, Inc. Stepped cannula
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
CA2587854C (en) 2004-11-18 2014-01-21 The Board Of Trustees Of The University Of Illinois Multicistronic sirna constructs to inhibit tumors
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
WO2006066203A2 (en) 2004-12-16 2006-06-22 Alsgen, Llc Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod)
US7803611B2 (en) 2005-02-03 2010-09-28 Benitec, Inc. RNAi expression constructs
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
US8999943B2 (en) 2005-03-14 2015-04-07 Board Of Regents, The University Of Texas System Antigene oligomers inhibit transcription
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
AU2006243776A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
EP2239328A3 (en) 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
JP5238503B2 (ja) 2005-08-23 2013-07-17 ザ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・カリフォルニア 対流増加送達を用いた治療薬の慢性的な送達のための逆流防止カニューレ及びシステム
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
RU2448974C2 (ru) * 2005-11-01 2012-04-27 Элнилэм Фармасьютикалз, Инк. РНКи-ИНГИБИРОВАНИЕ РЕПЛИКАЦИИ ВИРУСА ГРИППА
CA2629664A1 (en) 2005-11-17 2007-08-02 Board Of Regents, The University Of Texas System Modulation of gene expression by oligomers targeted to chromosomal dna
US20070259827A1 (en) * 2006-01-25 2007-11-08 University Of Massachusetts Compositions and methods for enhancing discriminatory RNA interference
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
CA3042781C (en) 2006-04-03 2021-10-19 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
JP2009535339A (ja) 2006-04-28 2009-10-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
CN101437538A (zh) 2006-04-28 2009-05-20 宾夕法尼亚州立大学托管会 修饰的腺病毒六邻体蛋白和其应用
US20080003565A1 (en) 2006-05-02 2008-01-03 Government Of The Us, As Represented By The Secretary, Department Of Health And Human Services Viral nucleic acid microarray and method of use
US7951925B2 (en) 2006-05-25 2011-05-31 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
WO2008011636A2 (en) 2006-07-21 2008-01-24 California Institute Of Technology Targeted gene delivery for dendritic cell vaccination
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
EP3146982B1 (en) 2006-10-03 2019-08-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
WO2008067480A2 (en) 2006-11-29 2008-06-05 Nationwide Children's Hospital Myostatin inhibition for enhancing muscle and/or improving muscle function
EP2647711B1 (en) 2006-11-29 2018-01-10 University of Iowa Research Foundation Alternative export pathways for vector expressed RNA interference
US8071752B2 (en) 2007-01-29 2011-12-06 City Of Hope Multi-targeting short interfering RNAs
EA017403B1 (ru) 2007-02-02 2012-12-28 Байоджен Айдек Эмэй Инк. Использование семафорина 6а в качестве промотора миелинизации и дифференцировки олигодендроцитов
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
WO2008128251A1 (en) 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Humanized viral vectors and methods of use thereof
WO2008134646A2 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2158211B1 (en) 2007-05-31 2016-08-10 Medigene AG Mutated structural protein of a parvovirus
DK2164967T3 (en) 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
US8053186B2 (en) 2007-06-15 2011-11-08 The Ohio State University Research Foundation Oncogenic ALL-1 fusion proteins for targeting Drosha-mediated microRNA processing
AU2008271658B8 (en) 2007-06-29 2013-07-04 F. Hoffmann-La Roche Ag Promoter
CA2728571A1 (en) 2007-06-29 2009-01-08 Boston Biomedical, Inc. Bacteria-mediated gene modulation via microrna machinery
CA2693712C (en) 2007-07-14 2018-05-29 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
HUE029744T2 (en) 2007-07-26 2017-04-28 Uniqure Ip Bv Baculoviral vectors comprising repeated coding sequences with differential codon biases
EP3121281B1 (en) 2007-08-27 2021-03-31 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
US8420372B2 (en) 2007-09-04 2013-04-16 Alexander Bello Porcine adeno-associated viruses
US9115373B2 (en) 2007-09-19 2015-08-25 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
AU2008308499A1 (en) 2007-10-04 2009-04-09 Board Of Regents, The University Of Texas System Modulating gene expression with agRNA and gapmers targeting antisense transcripts
WO2009051421A2 (en) 2007-10-18 2009-04-23 Lg Electronics Inc. Method and system for transmitting and receiving signals
WO2009073103A2 (en) 2007-11-28 2009-06-11 The Trustees Of The University Of Pennsylvania Simian subfamily b adenoviruses sadv-28,27,-29,-32,-33, and -35 and uses thereof
CN102131920B (zh) 2007-11-28 2013-11-06 宾夕法尼亚大学托管会 猿猴亚家族C腺病毒SAdV-40、-31和-34及其应用
KR101662574B1 (ko) 2007-11-28 2016-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 유인원 ｅ 아데노바이러스 ｓａｄｖ-39, -25.2, -26, -30, -37, 및 -38
EP2235033A4 (en) * 2007-12-28 2011-11-02 Univ California METHOD AND COMPOSITIONS FOR INCREASING GENE EXPRESSION
CA2710398A1 (en) 2008-01-18 2009-10-15 Genentech, Inc. Methods and compositions for targeting polyubiquitin
EP2242840B1 (en) 2008-01-29 2019-07-24 Applied Genetic Technologies Corporation Recombinant adeno-associated virus production using bhk cells in suspension
EP3643782A1 (en) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
AU2009215987B2 (en) 2008-02-19 2015-01-22 Uniqure Ip B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
EP2325298B1 (en) 2008-03-04 2016-10-05 The Trustees Of The University Of Pennsylvania SIMIAN ADENOVIRUSES SAdV-36, -42.1, -42.2, AND -44 AND USES THEREOF
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
WO2009146301A1 (en) 2008-05-27 2009-12-03 Yale University TARGETING TGF-β AS A THERAPY FOR ALZHEIMER'S DISEASE
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
CA2726052A1 (en) 2008-06-04 2009-12-10 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
US8951979B2 (en) 2008-06-13 2015-02-10 Cornell University Pain treatment using ERK2 inhibitors
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
US8945885B2 (en) 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
WO2010014857A2 (en) 2008-07-30 2010-02-04 University Of Massachusetts Chromosome therapy
KR20110086553A (ko) 2008-09-29 2011-07-28 암스테르담 몰레큘러 테라퓨틱스 비. 브이. 포르포빌리노겐 디아미나아제 유전자 치료
CA2741089A1 (en) 2008-10-22 2010-04-29 Genentech, Inc. Modulation of axon degeneration
EP2774985B1 (en) 2008-10-31 2016-12-14 The Trustees Of The University Of Pennsylvania Simian adenovirus SAdV-43 and uses thereof
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CA2754335C (en) 2009-03-04 2019-12-24 Medigene Ag Assembly activating protein (aap) and its use for the manufacture of parvovirus particles essentially consisting of vp3
AU2010227419A1 (en) 2009-03-27 2011-10-20 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
DK2425000T3 (da) 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2010136549A2 (en) 2009-05-28 2010-12-02 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
US8846031B2 (en) 2009-05-29 2014-09-30 The Trustees Of The University Of Pennsylvania Simian adenovirus 41 and uses thereof
CN102575248A (zh) 2009-06-05 2012-07-11 薛大寓 用于抑制单或多靶基因的多顺反子shRNA表达盒
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えａａｖベクターの改良された精製方法
JP2014501489A (ja) 2009-07-06 2014-01-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド 細胞ベースのバイオプロセシング
JP2014501097A (ja) 2009-07-06 2014-01-20 アルナイラム ファーマシューティカルズ， インコーポレイテッド 生物由来物質の産生を高めるための組成物及び方法
US20120201794A1 (en) 2009-07-15 2012-08-09 Calimmune Inc. Dual vector for inhibition of human immunodeficiency virus
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
AU2010316982B2 (en) 2009-11-09 2013-09-12 Genepod Therapeutics Ab Novel viral vector construct for neuron specific continuous DOPA synthesis in vivo
WO2011062298A1 (ja) 2009-11-19 2011-05-26 国立大学法人岡山大学 遺伝子発現を上昇させるシステム及び該システムを保持したベクター
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2011097456A2 (en) 2010-02-05 2011-08-11 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
WO2011101869A1 (en) 2010-02-22 2011-08-25 Transgene Biotek Ltd. Adeno-associated virus 2/8 - micro rna-101 therapy for liver cancer
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
CN103068980B (zh) 2010-08-02 2017-04-05 瑟纳治疗公司 使用短干扰核酸（siNA）的RNA干扰介导的联蛋白（钙粘蛋白关联蛋白质），β1（CTNNB1）基因表达的抑制
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
CN101972476B (zh) 2010-09-14 2012-12-19 中国人民解放军第二军医大学 利用MicroRNA-155的核酸疫苗佐剂及其构建方法
CN103124787B (zh) 2010-10-05 2015-04-29 宝生物工程株式会社 制造病毒载体的方法
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
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
EP2637702A4 (en) 2010-11-11 2014-11-26 Univ Miami METHODS, COMPOSITIONS, CELLS AND KITS FOR TREATING ISCHEMIC INJURIES
EP2643465B1 (en) 2010-11-23 2016-05-11 The Trustees Of The University Of Pennsylvania Subfamily e simian adenovirus a1321 and uses thereof
ES2739804T3 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
CN103502458B (zh) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP3219313B1 (en) 2011-04-18 2020-10-28 National Center of Neurology and Psychiatry Drug delivery particle and method for producing the same
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
WO2012149646A1 (en) 2011-05-05 2012-11-08 Sunnybrook Research Institute Mirna inhibitors and their uses
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
US9598468B2 (en) 2011-05-18 2017-03-21 University Of Florida Research Foundation, Incorporated Polypeptides and vectors for targeting HER2/neu expressing cells and uses thereof
CA2831969A1 (en) 2011-06-06 2012-12-30 Biocartis S.A. Selective lysis of cells by ionic surfactants
CA2842798C (en) 2011-07-25 2021-02-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013014294A2 (en) 2011-07-27 2013-01-31 Genethon Improved baculovirus expression systems
US8852911B2 (en) 2011-08-04 2014-10-07 The Regents Of The University Of California Method of producing dicer
US20130129668A1 (en) * 2011-09-01 2013-05-23 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
JP6198278B2 (ja) 2011-09-08 2017-09-20 ユニキュアー アイピー ビー．ブイ． Ａａｖ調製物からの混入ウイルスの除去
WO2013036889A1 (en) 2011-09-09 2013-03-14 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
US9464322B2 (en) 2011-09-09 2016-10-11 University Of Kentucky Research Foundation Methods for diagnosing and treating alzheimer's disease (AD) using the molecules that stabilize intracellular calcium (Ca2+) release
EP3275461A1 (en) 2011-09-19 2018-01-31 Axon Neuroscience SE Protein-based therapy and diagnosis of tau-mediated pathology in alzheimer's disease field
EP2771455B1 (en) 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
EP2782596A4 (en) 2011-11-22 2015-07-29 Philadelphia Children Hospital VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENIC ADMINISTRATION
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
WO2013103896A1 (en) 2012-01-06 2013-07-11 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
US20150301068A1 (en) 2012-01-30 2015-10-22 Vib Vzw Means and method for diagnosis and treatment of alzheimer's disease
JP6562632B2 (ja) 2012-02-14 2019-08-21 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
WO2013122605A1 (en) 2012-02-17 2013-08-22 Evernote Corporation Site memory processing
CA2865011C (en) 2012-02-29 2021-06-15 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
JP6342886B2 (ja) 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
CN104395323B (zh) 2012-05-08 2017-08-01 默沙东公司 可透过性糖苷酶抑制剂及其用途
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
AU2013262626B2 (en) 2012-05-18 2018-11-29 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1302, A1320, A1331 and A1337 and uses thereof
US20150183850A1 (en) 2012-05-18 2015-07-02 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
EP2852388A4 (en) 2012-05-23 2016-01-13 Univ Johns Hopkins COMPOUNDS AND METHOD FOR USE THEREOF FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
HK1210214A1 (en) 2012-07-06 2016-04-15 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
KR101738438B1 (ko) 2012-07-06 2017-05-22 다카라 바이오 가부시키가이샤 아데노-관련 바이러스 벡터의 생산세포
CA2880653C (en) 2012-08-01 2022-05-17 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
CA2885216A1 (en) 2012-09-17 2014-03-20 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
AU2013204200B2 (en) 2012-10-11 2016-10-20 Brandeis University Treatment of amyotrophic lateral sclerosis
US9733237B2 (en) 2012-10-31 2017-08-15 The Trustees Of Columbia University In The City Of New York Methods for identifying candidates for the treatment of neurodegenerative diseases
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
EP2940131B1 (en) 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
DK2943567T3 (en) 2013-01-08 2018-01-02 Genzyme Corp USE OF INOS INHIBITORS TO INCREASE VIRAL YIELD IN CULTURE
WO2014107763A1 (en) 2013-01-08 2014-07-17 Benitec Biopharma Limited Age-related macular degeneration treatment
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
JP2016517278A (ja) 2013-03-15 2016-06-16 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ スタッファー／フィラーポリヌクレオチド配列を含むベクターおよびその使用方法
EP2970966A4 (en) 2013-03-15 2016-10-26 Univ North Carolina SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES
WO2014144844A1 (en) 2013-03-15 2014-09-18 The Board Of Trustees Of The Leland Stanford Junior University tRNA DERIVED SMALL RNAs (tsRNAs) INVOLVED IN CELL VIABILITY
AU2014251099B2 (en) 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
US9862945B2 (en) 2013-04-20 2018-01-09 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
JP2016523835A (ja) 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
SG11201509595PA (en) * 2013-05-28 2015-12-30 Dcb Usa Llc Antibody locker for the inactivation of protein drug
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2014201308A1 (en) 2013-06-12 2014-12-18 Washington University Endothelial-targeted adenoviral vectors, methods and uses therefor
WO2014201252A2 (en) 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
CA2916499A1 (en) * 2013-07-03 2015-01-08 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
RU2664471C2 (ru) 2013-07-26 2018-08-17 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3068889B1 (en) 2013-09-26 2019-04-17 Universitat Autònoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
HUE052676T2 (hu) 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
US9725719B2 (en) 2013-11-05 2017-08-08 The Research Institute At Nationwide Children's Hospital Compositions and methods for inhibiting NF-κB and SOD-1 to treat amyotrophic lateral sclerosis
EP3066195A4 (en) 2013-11-08 2017-06-28 The Board of Trustees of the University of Arkansas Adeno-associated virus "x" oncogene
MX391904B (es) 2013-11-11 2025-03-12 Sangamo Biosciences Inc Un represor genetico para usarse en el tratamiento de la enfermedad de huntington.
JP6649265B2 (ja) 2013-11-26 2020-02-19 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 糖原病の処置のためのアデノ随伴ウイルスベクター
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
SG11201604505QA (en) 2013-12-03 2016-07-28 Agency Science Tech & Res Tailed Mirtron Effectors For RNAi-Mediated Gene Silencing
EP3572513B1 (en) 2013-12-09 2025-09-24 Baylor College of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
KR20160097338A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
WO2015106273A2 (en) 2014-01-13 2015-07-16 Trustees Of Boston University Methods and assays relating to huntingtons disease and parkinson's disease
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
MX367600B (es) 2014-02-19 2019-08-28 Hoffmann La Roche Transportador de la barrera hematoencefálica.
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015124546A1 (en) 2014-02-19 2015-08-27 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic Aav vectors for the treatment of ischemic and non-ischemic heart disease
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
EP3450571B1 (en) 2014-02-24 2023-04-05 Celgene Corporation Methods of using an activator of cereblon for neural cell expansion and the treatment of central nervous system disorders
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
EP3119415A4 (en) 2014-03-07 2017-11-29 The Arizona Board of Regents on behalf of the University of Arizona Non-narcotic crmp2 peptides targeting sodium channels for chronic pain
WO2015137802A1 (en) 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
HUE050704T2 (hu) 2014-04-01 2020-12-28 Biogen Ma Inc Összetételek a SOD-1 expressziójának modulálására
EP3129485B2 (en) 2014-04-09 2022-12-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
US11555059B2 (en) 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
BR112016025849A2 (pt) 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
EP3142706A1 (en) 2014-05-16 2017-03-22 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
AU2015264263B2 (en) 2014-05-20 2021-08-05 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
JP7026440B2 (ja) 2014-05-28 2022-02-28 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ハイブリッドｔＲＮＡ／プレｍｉＲＮＡ分子および使用方法
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US20150376612A1 (en) * 2014-06-10 2015-12-31 The General Hospital Corporation CCCTC-Binding Factor (CTCF) RNA Interactome
US11207424B2 (en) 2014-06-13 2021-12-28 Mayo Foundation For Medical Education And Research Methods and materials for increasing viral vector infectivity
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
US10526583B2 (en) 2014-07-02 2020-01-07 University Of Florida Research Foundation, Incorporated Compositions and methods for purifying recombinant adeno-associated virus
US10023846B2 (en) 2014-07-10 2018-07-17 Takara Bio Inc. Production method for non-enveloped virus particles
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
EP3174981B1 (en) 2014-07-31 2020-03-25 Association Institut de Myologie Treatment of amyotrophic lateral sclerosis
WO2016019364A1 (en) 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
WO2016040347A2 (en) 2014-09-08 2016-03-17 University Of Iowa Research Foundation Microrna inhibitor system and methods of use thereof
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
US10842886B2 (en) 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
EP3690024A1 (en) 2014-11-05 2020-08-05 The Research Institute at Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
HUE046643T2 (hu) 2014-11-28 2021-11-29 Uniqure Ip Bv DNS szennyezõdések parvovirális virionokat tartalmazó készítményben
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
AU2015370903B2 (en) 2014-12-24 2021-06-17 Uniqure Ip B.V. RNAi induced huntingtin gene suppression
WO2016109649A1 (en) 2014-12-30 2016-07-07 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
AU2016206624B2 (en) 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3632923A1 (en) 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
CN107427666B (zh) 2015-01-30 2022-11-04 加利福尼亚大学校董 脊髓软膜下基因递送系统
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
CA3255657A1 (en) 2015-02-10 2025-12-01 Genzyme Corporation VARIANT RNAi
EA201791803A1 (ru) 2015-02-10 2017-11-30 Джензим Корпорейшн Улучшенная доставка вирусных частиц в полосатое тело и кору больших полушарий
US20180245073A1 (en) 2015-02-23 2018-08-30 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3270960A4 (en) 2015-03-20 2018-08-08 Bluebird Bio, Inc. Vector formulations
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
RS60762B1 (sr) 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CN115094062A (zh) 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US20160346359A1 (en) 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
HK1252512A1 (zh) 2015-05-07 2019-05-31 Massachusetts Eye & Ear Infirmary 向眼部遞送藥劑的方法
CA2985347A1 (en) 2015-05-11 2016-11-17 Alcyone Lifesciences, Inc. Drug delivery systems and methods
WO2016183297A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US20170067028A1 (en) 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
US10729790B2 (en) 2015-05-26 2020-08-04 Salk Institute For Biological Studies Motor neuron-specific expression vectors
RU2761564C9 (ru) 2015-05-29 2022-03-17 Дзе Трастиз Оф Дзе Юнивёрсити Оф Пенсильвания Композиции и способы деградации неправильно упакованных белков
WO2016196507A1 (en) 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
US11266748B2 (en) 2015-07-02 2022-03-08 University Of Florida Research Foundation, Incorporated Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
US20170007669A1 (en) 2015-07-07 2017-01-12 Mayo Foundation For Medical Education And Research Peptide-mediated delivery of active agents across the blood-brain barrier
US10801040B2 (en) 2015-07-07 2020-10-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
CA2988471A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
EP4134375A1 (en) 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US20180201937A1 (en) 2015-08-04 2018-07-19 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
US10047377B2 (en) 2015-09-22 2018-08-14 Loyola University Of Chicago Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor
DK3352776T3 (da) 2015-09-23 2025-06-23 Sangamo Therapeutics Inc Htt-repressorer og deres anvendelse
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
IL258538B2 (en) 2015-10-09 2023-04-01 Genzyme Corp Early isolation after the infection of cells (epic) for the production of biological material
EP3359662A4 (en) 2015-10-09 2019-06-19 The Children's Hospital of Philadelphia COMPOSITIONS AND METHODS OF TREATING HUNTINGTON DISEASE AND RELATED DISEASES
US10123969B2 (en) 2015-10-15 2018-11-13 Wisconsin Alumni Research Foundation Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
AU2016341428B2 (en) 2015-10-23 2021-12-02 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
US11135313B2 (en) 2015-10-28 2021-10-05 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
WO2017079768A1 (en) 2015-11-08 2017-05-11 Genentech, Inc. Methods of screening for multispecific antibodies
US10633662B2 (en) 2015-11-10 2020-04-28 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating AAV infection
EP3384015A4 (en) 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
WO2017095753A1 (en) 2015-12-01 2017-06-08 Invivo Therapeutics Corporation Compositions and methods for preparing an injectable medium for administration into the central nervous system
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
EP3383896A1 (en) 2015-12-03 2018-10-10 Genethon Compositions and methods for improving viral vector efficiency
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387138B2 (en) 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
US11124546B2 (en) 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
US11208630B2 (en) 2015-12-24 2021-12-28 University Of Florida Research Foundation, Incorporated AAV production using suspension adapted cells
ES2871527T3 (es) 2016-01-15 2021-10-29 Univ Jichi Medical Virión de virus adenoasociado para usar en el tratamiento de la epilepsia
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
HRP20231451T1 (hr) 2016-02-05 2024-03-01 Emory University Ubrizgavanje jednolančanog ili samo-komplementarnog adeno-povezanog virusa 9 u cerebrospinalnu tekućinu
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3014637A1 (en) 2016-02-16 2017-08-24 Mark A. Kay Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
US20190071681A1 (en) 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction
IL261524B2 (en) 2016-03-03 2023-10-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer
WO2017155973A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
AU2017234929B2 (en) 2016-03-18 2024-05-02 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
ES2938833T3 (es) 2016-03-28 2023-04-17 Ultragenyx Pharmaceutical Inc Métodos de inactivación térmica de adenovirus
CA3039910A1 (en) 2016-03-28 2017-10-05 The Regents Of The University Of California Anti-ryk antibodies and methods of using the same
WO2017173043A1 (en) 2016-03-30 2017-10-05 Spark Therapeutics, Inc. Cell line for recombinant protein and/or viral vector production
PH12018502104B1 (en) 2016-03-31 2024-03-27 Spark Therapeutics Inc Column-based fully scalable raav manufacturing process
CN109661470A (zh) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
RU2762948C2 (ru) 2016-04-16 2021-12-24 Юниверсити Оф Флорида Рисерч Фаундэйшн, Инкорпорейтед Способы усиления биологической активности рекомбинантного аденоассоциированного вуруса, продуцируемого бакуловирусной системой
CA3012699C (en) 2016-04-21 2023-11-21 Virovek, Inc Aav production in insect cells, methods and compositions therefor
EP3235516B1 (en) 2016-04-22 2019-06-26 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin Regulatable adeno-associated virus (aav) vector
EP3449250B1 (en) 2016-04-28 2020-11-04 Indiana University Research & Technology Corporation Methods and compositions for resolving components of a virus preparation
WO2017189963A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017261249B2 (en) 2016-05-03 2021-05-06 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
CA3059891A1 (en) 2017-04-14 2018-10-18 National Taiwan University Gene therapy for aadc deficiency
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US20200270635A1 (en) 2017-05-05 2020-08-27 Voyager Therapeutics, Inc. Modulatory polynucleotides
EP3635121A1 (en) 2017-06-02 2020-04-15 Institut National de la Sante et de la Recherche Medicale (INSERM) Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
GB201714027D0 (en) 2017-09-01 2017-10-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of huntington's disease
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
JP2022501016A (ja) * 2018-09-05 2022-01-06 アモネタ・ダイアグノスティクスAmoneta Diagnostics 脳障害、特に、認知障害の診断および治療のための長鎖ノンコーディングＲＮＡ（ｌｎｃＲＮＡ）

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110020816A1 (en) 2008-06-20 2011-01-27 Chang-Zheng Chen Precursor miRNA loop-modulated target regulation
WO2013126605A1 (en) 2012-02-21 2013-08-29 The Johns Hopkins University EXPRESSION CONSTRUCT FOR A LIN28-RESISTANT Let-7 PRECURSOR MICRORNA
WO2014016817A2 (en) 2012-07-17 2014-01-30 Universite De Geneve Nucleic acids for down-regulation of gene expression

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Methods, Available online 2016.04.12, Vol.103, pp.157-166
PLOS ONE, 2014, Vol.9, Issue 3, e92188 (pp.1-13)

Also Published As

Publication number Publication date
EP3458588A1 (en) 2019-03-27
AU2023203585B2 (en) 2025-09-04
JP2019519218A (ja) 2019-07-11
US20190169616A1 (en) 2019-06-06
MX2018014154A (es) 2019-05-06
CN110214187B (zh) 2024-01-30
EP3458588A4 (en) 2020-01-15
CN110214187A (zh) 2019-09-06
CA3024448C (en) 2025-09-09
EP3458588B1 (en) 2025-10-29
WO2017201248A1 (en) 2017-11-23
JP2022106853A (ja) 2022-07-20
US10584337B2 (en) 2020-03-10
RU2018140495A (ru) 2020-06-18
MX2023010042A (es) 2023-09-05
JP2023182824A (ja) 2023-12-26
US11193129B2 (en) 2021-12-07
CA3024448A1 (en) 2017-11-23
US20200149045A1 (en) 2020-05-14
IL262784A (en) 2018-12-31
IL262784B2 (en) 2023-10-01
US20260117224A1 (en) 2026-04-30
KR20190005886A (ko) 2019-01-16
IL302748B2 (en) 2026-01-01
AU2017267665A1 (en) 2018-12-06
KR102652994B1 (ko) 2024-04-01
RU2758488C2 (ru) 2021-10-28
AU2017267665B2 (en) 2023-03-16
CN117904112A (zh) 2024-04-19
ZA201807401B (en) 2020-12-23
IL302748B1 (en) 2025-09-01
SG11201809699XA (en) 2018-12-28
JP7374254B2 (ja) 2023-11-06
AU2017267665C1 (en) 2023-10-05
US12084659B2 (en) 2024-09-10
AU2023203585A1 (en) 2023-09-07
RU2018140495A3 (enExample) 2020-10-14
KR20220054727A (ko) 2022-05-03
IL302748A (en) 2023-07-01
CA3252099A1 (en) 2025-06-05
KR20240056729A (ko) 2024-04-30
US20220162609A1 (en) 2022-05-26
BR112018073384A2 (pt) 2019-03-06
IL262784B1 (en) 2023-06-01
KR102392236B1 (ko) 2022-05-03

Similar Documents

Publication Publication Date Title
JP7374254B2 (ja) 調節性ポリヌクレオチド
JP7469538B2 (ja) 調節性ポリヌクレオチド
US20240131093A1 (en) Compositions and methods of treating huntington's disease
JP7220080B2 (ja) ハンチントン病治療組成物及び方法
JP7565218B2 (ja) 筋萎縮性側索硬化症および脊髄に関連する障害の治療
US20230399642A1 (en) Compositions and methods of treating huntington's disease
EP4454654A2 (en) Treatment of amyotrophic lateral sclerosis (als)
BR112018073384B1 (pt) Polinucleotídeo modulador, vetor, genoma viral, vírus aav recombinante, seu método de produção, seus usos, e composição farmacêutica
BR122024015196A2 (pt) Polinucleotídeo modulador, vetor, genoma viral, vírus aav recombinante, seu método de produção, seus usos, bem como composição farmacêutica compreendendo o mesmo

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190206

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200514

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200514

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210615

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210914

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211214

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220311

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220329

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220427

R150 Certificate of patent or registration of utility model

Ref document number: 7066635

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250