[go: up one dir, main page]

PE20241233A1 - Tratamiento aav de la enfermedad de huntington - Google Patents

Tratamiento aav de la enfermedad de huntington

Info

Publication number
PE20241233A1
PE20241233A1 PE2024001008A PE2024001008A PE20241233A1 PE 20241233 A1 PE20241233 A1 PE 20241233A1 PE 2024001008 A PE2024001008 A PE 2024001008A PE 2024001008 A PE2024001008 A PE 2024001008A PE 20241233 A1 PE20241233 A1 PE 20241233A1
Authority
PE
Peru
Prior art keywords
disease
mirna
huntington
htt
nucleic acid
Prior art date
Application number
PE2024001008A
Other languages
English (en)
Inventor
Christian Mueller
Neil Aronin
Edith L Pfister
Original Assignee
Univ Massachusetts
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Massachusetts filed Critical Univ Massachusetts
Publication of PE20241233A1 publication Critical patent/PE20241233A1/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • C12N15/113—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
    • 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]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

Landscapes

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

Abstract

Se refieren a composiciones y metodos utiles para tratar la enfermedad de Huntington. En algunas modalidades, se proporciona acidos nucleicos de interferencia (por ejemplo, miARNs artificiales) dirigidos al gen de la huntingtina (HTT) y metodos para tratar la enfermedad de Huntington utilizando el mismo. En particular, se refiere a un acido nucleico aislado que comprende un transgen que codifica un miARN monocatenario maduro, en el que la secuencia de acido nucleico del transgen que codifica el miARN monocatenario maduro comprende la secuencia expuesta en SEQ ID NO: 8, y esta flanqueada por una secuencia de esqueleto de miARN heterologa, en el que el miARN se hibrida especificamente e inhibe la expresion de la huntingtina humana (HTT).
PE2024001008A 2016-09-22 2017-09-22 Tratamiento aav de la enfermedad de huntington PE20241233A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662398487P 2016-09-22 2016-09-22
PCT/US2017/052902 WO2018057855A1 (en) 2016-09-22 2017-09-22 Aav treatment of huntington's disease

Publications (1)

Publication Number Publication Date
PE20241233A1 true PE20241233A1 (es) 2024-06-19

Family

ID=61691085

Family Applications (2)

Application Number Title Priority Date Filing Date
PE2019000712A PE20191351A1 (es) 2016-09-22 2017-09-22 Tratamiento aav de la enfermedad de huntington
PE2024001008A PE20241233A1 (es) 2016-09-22 2017-09-22 Tratamiento aav de la enfermedad de huntington

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PE2019000712A PE20191351A1 (es) 2016-09-22 2017-09-22 Tratamiento aav de la enfermedad de huntington

Country Status (19)

Country Link
US (4) US10457940B2 (es)
EP (1) EP3515505A4 (es)
JP (4) JP6884430B2 (es)
KR (1) KR102271675B1 (es)
CN (2) CN110035776B (es)
AU (2) AU2017330411B2 (es)
BR (2) BR122020019033B1 (es)
CA (1) CA3037929A1 (es)
CL (1) CL2019000732A1 (es)
CO (1) CO2019003846A2 (es)
IL (3) IL265528B2 (es)
MX (2) MX2019003376A (es)
MY (1) MY195386A (es)
PE (2) PE20191351A1 (es)
PH (1) PH12019500626A1 (es)
RU (1) RU2749971C2 (es)
SA (1) SA519401379B1 (es)
WO (1) WO2018057855A1 (es)
ZA (1) ZA201902226B (es)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
JP2020518259A (ja) * 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
WO2019159884A1 (ja) * 2018-02-13 2019-08-22 東レ株式会社 認知症の検出のためのキット又はデバイス及び方法
EP3775214A2 (en) * 2018-03-30 2021-02-17 University of Geneva Micro rna expression constructs and uses thereof
EP3788141A4 (en) 2018-04-30 2023-03-08 The Brigham and Women's Hospital, Inc. COMPOSITIONS AND THERAPEUTIC METHODS OF MICRORNA GENE DELIVERY
EP3794125A4 (en) * 2018-05-15 2022-07-13 University of Massachusetts MODIFIED AAV CONSTRUCTS AND USES THEREOF
EP3887522A1 (en) * 2018-11-29 2021-10-06 University of Massachusetts Modulation of sptlc1 via recombinant adeno-associated vectors
MX2021008331A (es) * 2019-01-09 2021-08-05 Univ De Coimbra Arn bicatenario y usos del mismo.
CN114728018B (zh) * 2019-11-01 2024-07-19 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
CA3163034A1 (en) * 2019-12-20 2021-06-24 SiewHui LOW Methods for treating huntington's disease
WO2022061378A2 (en) * 2020-09-21 2022-03-24 Asklepios Biopharmaceutical, Inc. Methods for treating neurological disease
US20240084323A1 (en) * 2021-01-13 2024-03-14 Dignity Health Modulation of chitinase protein expression
CN115141848A (zh) * 2021-03-30 2022-10-04 南京大学 一种用于治疗亨廷顿病的rna递送系统
CN115487315B (zh) * 2022-04-20 2023-08-04 暨南大学 治疗亨廷顿病的药物
US20240360443A1 (en) 2023-04-09 2024-10-31 Dystrogen Gene Therapies Inc. Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
WO2025027116A1 (en) 2023-08-01 2025-02-06 Institut Curie Nanoparticles comprising nucleic acid sequences encoding cyclic gmp-amp synthase
WO2025078598A1 (en) * 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Novel nucleic acid regulation of huntingtin gene
WO2025160434A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease

Family Cites Families (142)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
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
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6544523B1 (en) 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
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
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
US20030110526A1 (en) 1998-08-25 2003-06-12 Robert H. Brown Dysferlin mutations
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
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US6544786B1 (en) 1999-10-15 2003-04-08 University Of Pittsburgh Of The Commonwealth Of Higher Education Method and vector for producing and transferring trans-spliced peptides
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US20050032219A1 (en) 2001-07-03 2005-02-10 Patrick Aubourg Methods of administering vectors to synaptically connected neurons
JP4210737B2 (ja) * 2001-07-12 2009-01-21 ユニバーシティー オブ マサチューセッツ 遺伝子サイレンシングを仲介する低分子干渉リボ核酸のインビボにおける製造方法
JP2004534543A (ja) 2001-07-13 2004-11-18 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 シュードタイプ化アデノ関連ウイルスおよびその使用
US20030109476A1 (en) * 2001-08-07 2003-06-12 Kmiec Eric B. Compositions and methods for the prevention and treatment of Huntington's disease
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
US20050137153A1 (en) 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
WO2003089011A1 (en) 2002-04-19 2003-10-30 University Of Florida rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
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
US20060093589A1 (en) 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
JP2006518372A (ja) 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用
US20040219528A1 (en) 2003-04-15 2004-11-04 Morris David W. Novel therapeutic targets in cancer
US7387896B2 (en) 2003-03-26 2008-06-17 The Regents Of The University Of Michigan MicroRNA vectors
US20060228800A1 (en) 2003-05-15 2006-10-12 Shi-Lung Lin Novel Transgenic Methods Using intronic RNA
EP3502252B1 (en) 2003-06-02 2023-04-05 University of Massachusetts Methods and compositions for controlling efficacy of rna silencing
WO2008005562A2 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
US8680063B2 (en) * 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
EP2292780B1 (en) 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
CA2558771C (en) 2004-03-05 2013-01-08 Benitec, Inc. Multiple promoter expression cassettes for simultaneous delivery of rnai agents
US7498316B2 (en) 2004-04-06 2009-03-03 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using RNA interference
WO2005116224A2 (en) 2004-05-18 2005-12-08 Children's Memorial Hospital Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
EP1877556B1 (en) 2005-03-25 2011-09-14 Medtronic, Inc. Use of anti-tnf or anti-il1 rnai to suppress pro- inflammatory cytokine actions locally to treat pain
EP2359865B1 (en) 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
CA2609142C (en) 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
ATE494372T1 (de) 2005-08-29 2011-01-15 Regulus Therapeutics Inc Verfahren für mir-122a-modulation
ES2508893T3 (es) 2006-01-05 2014-10-16 The Ohio State University Research Foundation Métodos basados en microARN para el diagnóstico de cánceres de estómago
EP1979485A2 (en) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
DK2495327T3 (da) 2006-03-03 2017-01-02 Promis Neurosciences Inc Fremgangsmåder og sammensætninger til at behandle og opdage sygdomme fremkaldt af fejlfoldet SOD1
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US20080199961A1 (en) * 2006-08-25 2008-08-21 Avi Biopharma, Inc. ANTISENSE COMPOSITION AND METHOD FOR INHIBITION OF miRNA BIOGENESIS
DK2548438T3 (en) 2006-11-08 2015-10-19 Veritas Bio LLC IN VIVO SUBMITTING DOUBLE-STRENGTH RNA TO A CELL
EP3492596A1 (en) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
WO2008125846A2 (en) 2007-04-12 2008-10-23 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
US8399248B2 (en) 2007-05-03 2013-03-19 Merck Sharp & Dohme Corp. Methods of using MIR34 as a biomarker for TP53 functional status
US8841267B2 (en) 2007-05-23 2014-09-23 Ge Healthcare Dharmacon, Inc. Micro-RNA scaffolds, non-naturally occurring micro-RNAs, and methods for optimizing non-naturally occurring micro-RNAs
CA2691617C (en) 2007-05-31 2020-07-21 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US9090894B2 (en) 2007-12-17 2015-07-28 California Institute Of Technology Modulating immune system development and function through microRNA MIR-146
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP2268811A1 (en) 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2009146178A1 (en) 2008-04-15 2009-12-03 President And Fellows Of Harvard College Angiogenin and amyotrophic lateral sclerosis
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2297322A1 (en) 2008-06-04 2011-03-23 The Board of Regents of The University of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
EP2310507A4 (en) 2008-07-08 2013-03-20 David Gladstone Inst METHOD AND COMPOSITIONS FOR ANGIOGENIC MODULATION
EP2329275A2 (en) 2008-09-04 2011-06-08 Galenea Corporation Synaptic vesicle cycling assays and systems
WO2010034314A1 (en) 2008-09-24 2010-04-01 Aarhus Universitet Retroviral delivery of synthectic gene cassettes
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
US20110305772A1 (en) 2009-02-26 2011-12-15 The Johns Hopkins University Compositions and methods for ex vivo hepatic nucleic acid delivery
LT2424571T (lt) * 2009-04-30 2020-08-10 Ospedale San Raffaele S.R.L. Genų vektorius
DK3421603T3 (da) 2009-05-02 2022-01-10 Genzyme Corp Genterapi for neurodegenerative forstyrrelser
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2261242A1 (en) 2009-06-10 2010-12-15 Universite Catholique De Louvain Aspartate-N-acetyltransferase enzyme, diagnostic method and therapeutic method
KR102279458B1 (ko) * 2009-09-11 2021-07-21 아이오니스 파마수티컬즈, 인코포레이티드 헌팅틴 발현의 조절
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
KR20120089743A (ko) 2009-11-09 2012-08-13 제네포드 테라퓨틱스 에이비 생체 내에서 최적화된 뉴런 특이적 연속 도파 합성을 위한 신규한 바이러스 벡터 구성물
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
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
MX340102B (es) 2010-01-28 2016-06-24 The Children's Hospital Of Philadelphia * Una plataforma de fabricacion ajustable en escala, para la purificacion de vector viral y vectores virales asi purificados para el uso en terapia de genes.
WO2011109380A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
US9102949B2 (en) 2010-04-23 2015-08-11 University Of Massachusetts CNS targeting AAV vectors and methods of use thereof
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
US8785413B2 (en) 2010-09-27 2014-07-22 Florida Atlantic University Research Corporation Materials and methods for the treatment of pathological neovascularization in the eye
EP2673286B1 (en) 2011-02-12 2019-07-03 University of Iowa Research Foundation Therapeutic compounds
AU2012219380B2 (en) 2011-02-17 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving AAV9-mediated gene transfer
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
US20140147418A1 (en) 2011-04-15 2014-05-29 The Us Of America, As Represented By The Secretary Department Of Health And Human Services Aav mediated ctla-4 gene transfer to treat sjogren's syndrome
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
US8609088B2 (en) 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
US10184151B2 (en) 2011-10-11 2019-01-22 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
US20130142861A1 (en) 2011-12-05 2013-06-06 National Yang Ming University Compositions And Method For Detecting And Treating Abnormal Liver Homeostasis And Hepatocarcinogenesis
CA2864879C (en) 2012-02-17 2021-07-20 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
EP3536795A3 (en) 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression
US20140066595A1 (en) * 2012-09-04 2014-03-06 Thermo Fisher Scientific Biosciences Inc. Modulators of Protein Production in a Human Cell Line and Cell-free Extracts Produced Therefrom
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
NZ629569A (en) 2013-01-14 2018-07-27 Recombinetics Inc Hornless livestock
AU2014244167A1 (en) 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
CN105163764B (zh) 2013-03-15 2019-11-12 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
US10006049B2 (en) 2013-05-16 2018-06-26 University Of Florida Research Foundation, Incorporated Hairpin mRNA elements and methods for the regulation of protein translation
US10704060B2 (en) 2013-06-05 2020-07-07 Duke University RNA-guided gene editing and gene regulation
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
US10155794B2 (en) 2013-07-16 2018-12-18 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to CEP290
CA2919995A1 (en) 2013-08-05 2015-02-12 Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp
PT3039146T (pt) 2013-08-27 2020-08-11 The Res Institute At Nationwide Childrens Hospital Produtos e métodos para tratamento de esclerose lateral amiotrófica
US10369201B2 (en) * 2013-11-11 2019-08-06 Sangamo Therapeutics, Inc. Methods and compositions for treating Huntington'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
PL3119892T3 (pl) 2014-03-17 2021-11-29 The Hospital For Sick Children Warianty białka beta-heksozaminidazy oraz związane z nimi sposoby do leczenia gangliozydoz gm2
AU2015231294B2 (en) 2014-03-18 2020-10-29 University Of Massachusetts rAAV-based compositions and methods for treating amyotrophic lateral sclerosis
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
PE20170260A1 (es) 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
CA2964272A1 (en) 2014-10-21 2016-04-28 Guangping Gao Recombinant aav variants and uses thereof
SMT201900347T1 (it) * 2014-12-24 2019-07-11 Uniqure Ip Bv Soppressione del gene huntingtina indotta da rnai
HRP20201200T1 (hr) * 2015-04-03 2020-11-13 University Of Massachusetts OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease

Also Published As

Publication number Publication date
AU2017330411B2 (en) 2024-10-10
RU2019111648A (ru) 2020-10-22
KR102271675B1 (ko) 2021-07-02
JP7205933B2 (ja) 2023-01-17
JP2021129567A (ja) 2021-09-09
JP2019533439A (ja) 2019-11-21
AU2017330411A1 (en) 2019-04-11
JP7613759B2 (ja) 2025-01-15
CN117025677A (zh) 2023-11-10
JP2025063053A (ja) 2025-04-15
CA3037929A1 (en) 2018-03-29
BR122020019033B1 (pt) 2022-06-21
KR20190053236A (ko) 2019-05-17
IL265528A (en) 2019-05-30
ZA201902226B (en) 2025-10-29
IL323940A (en) 2025-12-01
RU2019111648A3 (es) 2020-11-23
EP3515505A1 (en) 2019-07-31
US20240084303A1 (en) 2024-03-14
PH12019500626A1 (en) 2019-08-19
US11773392B2 (en) 2023-10-03
CN110035776B (zh) 2023-08-25
AU2025200036A1 (en) 2025-01-23
CL2019000732A1 (es) 2019-10-18
JP6884430B2 (ja) 2021-06-09
IL308514A (en) 2024-01-01
MX2019003376A (es) 2019-09-13
CO2019003846A2 (es) 2019-06-28
US20180094264A1 (en) 2018-04-05
CN110035776A (zh) 2019-07-19
US20220010312A1 (en) 2022-01-13
MX2022013356A (es) 2022-11-30
MY195386A (en) 2023-01-18
US20200087664A1 (en) 2020-03-19
US11046957B2 (en) 2021-06-29
SA519401379B1 (ar) 2022-08-07
JP2023052024A (ja) 2023-04-11
IL265528B1 (en) 2023-12-01
EP3515505A4 (en) 2020-04-22
WO2018057855A1 (en) 2018-03-29
IL265528B2 (en) 2024-04-01
BR112019005548B1 (pt) 2022-06-14
IL308514B1 (en) 2025-11-01
US10457940B2 (en) 2019-10-29
RU2749971C2 (ru) 2021-06-21
PE20191351A1 (es) 2019-10-01
BR112019005548A2 (pt) 2019-06-18

Similar Documents

Publication Publication Date Title
PE20241233A1 (es) Tratamiento aav de la enfermedad de huntington
CO2017003434A2 (es) Moléculas de ácido nucleico de la subunidad alfa del coatómero copi que confiere resistencia contra plagas de coleópteros y hemípteros
HRP20190992T1 (hr) Suprimiranje gena za huntingtin inducirano s rnai
CO2017003428A2 (es) Moléculas de acido nucléico de sub-unidades de coatómero copi delta que confieren resistencia a plagas de coleópteros y hemípteros
CO2017003543A2 (es) Moleculas de acido nucleico de la subunidad beta coatomero copi que confieren resistencia a plagas de coleopteros y hemipteros
WO2013090457A3 (en) In vivo delivery of oligonucleotides
CO2017003427A2 (es) Moleculas de acido nucleico de la subunidad gamma coatomero copi que confieren resistencia a plagas de coleopteros y hemipteros.
MX387332B (es) Micro-acido ribonucleico (micro-arn) concordante artificial para controlar la expresion genetica y uso del mismo.
PE20190401A1 (es) Novedosas proteinas de la capside del virus adenoasociado
JP2012519484A5 (es)
AR084776A1 (es) Moleculas de acido nucleico que se dirigen a la subunidad h de la atpasa vacuolar y otorgan resistencia a las pestes de coleopteros
AR047348A1 (es) Promotor de metalotioneina de maiz 2 y metodos para usar el mismo
EP4257694A3 (en) Tissue-preferred promoters and methods of use
MX2011006577A (es) Metodos para modular el sexo de aviarios.
JP7189017B2 (ja) 細胞培養におけるウイルスの生産
AR088113A1 (es) ARN DE INTERFERENCIA (ARNi) PARA EL CONTROL DE HONGOS Y OOMICETOS POR LA INHIBICION DEL GEN DE SACAROPINA DESHIDROGENASA
EP4335502A3 (en) Modified u6 promoter system for tissue specific expression
MX381947B (es) Mirna natural para controlar la expresion de gen y uso del mismo.
MX2013011601A (es) Promotor especifico de semilla en alogodon.
MX2022001769A (es) Construcciones de vesícula extracelular-oligonucleótido antisentido (aso) que se dirigen a la proteína beta de unión al potenciador ccaat (cebp)/beta.
Abo-Al-Ela The emerging regulatory roles of noncoding RNAs in immune function of fish: MicroRNAs versus long noncoding RNAs
AR083445A1 (es) siARN CONTRA LA FIBROSIS
AR111946A1 (es) Moléculas de ácido nucleico de proteína de reconocimiento específico de la estructura 1 (ssrp1) para controlar plagas de insectos
NZ601951A (en) Nucleic acid construct comprising pyripyropene biosynthetic gene cluster and marker gene
AR094337A1 (es) Método y composiciones para el control genético de insectos plaga en plantas de algodón a través del silenciamiento de genes de quitina sintasas