AU2017227776C1 - AAV vectors for treatment of dominant retinitis pigmentosa - Google Patents

AAV vectors for treatment of dominant retinitis pigmentosa Download PDF

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Publication number
AU2017227776C1
AU2017227776C1 AU2017227776A AU2017227776A AU2017227776C1 AU 2017227776 C1 AU2017227776 C1 AU 2017227776C1 AU 2017227776 A AU2017227776 A AU 2017227776A AU 2017227776 A AU2017227776 A AU 2017227776A AU 2017227776 C1 AU2017227776 C1 AU 2017227776C1
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rna
rho
rna molecule
sequence
vector
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AU2017227776B2 (en
AU2017227776A1 (en
Inventor
Gustavo D. Aguirre
William BELTRAN
Artur CIDECIYAN
William W. Hauswirth
Samuel Jacobson
Alfred S. Lewin
Michael T. Massengill
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University of Pennsylvania Penn
University of Florida Research Foundation Inc
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University of Florida
University of Pennsylvania Penn
University of Florida Research Foundation Inc
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122Hairpin
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
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    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pigments, Carbon Blacks, Or Wood Stains (AREA)
  • Inorganic Compounds Of Heavy Metals (AREA)
  • Paper (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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AU2017227776A 2016-03-01 2017-03-01 AAV vectors for treatment of dominant retinitis pigmentosa Active AU2017227776C1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023237106A AU2023237106B2 (en) 2016-03-01 2023-09-27 Aav vectors for treatment of dominant retinitis pigmentosa

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662302122P 2016-03-01 2016-03-01
US62/302,122 2016-03-01
US201662398451P 2016-09-22 2016-09-22
US62/398,451 2016-09-22
PCT/US2017/020289 WO2017151823A1 (en) 2016-03-01 2017-03-01 Aav vectors for treatment of dominant retinitis pigmentosa

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2023237106A Division AU2023237106B2 (en) 2016-03-01 2023-09-27 Aav vectors for treatment of dominant retinitis pigmentosa

Publications (3)

Publication Number Publication Date
AU2017227776A1 AU2017227776A1 (en) 2018-08-23
AU2017227776B2 AU2017227776B2 (en) 2023-07-06
AU2017227776C1 true AU2017227776C1 (en) 2024-01-25

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AU2017227776A Active AU2017227776C1 (en) 2016-03-01 2017-03-01 AAV vectors for treatment of dominant retinitis pigmentosa
AU2023237106A Active AU2023237106B2 (en) 2016-03-01 2023-09-27 Aav vectors for treatment of dominant retinitis pigmentosa

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US (2) US11118185B2 (enExample)
EP (2) EP3423582B1 (enExample)
JP (3) JP7272795B2 (enExample)
CN (2) CN109154002B (enExample)
AU (2) AU2017227776C1 (enExample)
CA (1) CA3014671A1 (enExample)
ES (1) ES2923877T3 (enExample)
IL (2) IL261014B2 (enExample)
WO (1) WO2017151823A1 (enExample)

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IL261014B2 (en) 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa
EP3707257A4 (en) * 2017-11-07 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR CIRCULAR RNA MOLECULES
EP3793566A4 (en) * 2018-05-15 2022-03-16 University of Washington Compositions and methods for reducing spliceopathy and treating rna dominance disorders
JP7563694B2 (ja) * 2018-06-01 2024-10-08 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 優性網膜色素変性症の処置のための組成物および方法
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
CN113038971B (zh) * 2020-04-21 2022-06-24 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
CN111926044B (zh) * 2020-10-12 2021-01-22 北京大学第三医院(北京大学第三临床医学院) 结合突变rho基因的核酸分子和试剂盒
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2025231487A1 (en) * 2024-05-03 2025-11-06 The Trustees Of The University Of Pennsylvania Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration

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Publication number Publication date
JP2019511216A (ja) 2019-04-25
EP3423582A4 (en) 2019-11-13
EP3423582B1 (en) 2022-05-04
JP2025016451A (ja) 2025-02-04
IL261014B2 (en) 2023-10-01
CN109154002A (zh) 2019-01-04
CA3014671A1 (en) 2017-09-08
CN109154002B (zh) 2022-08-30
EP4119668A1 (en) 2023-01-18
JP7642211B2 (ja) 2025-03-10
JP2022185052A (ja) 2022-12-13
US11118185B2 (en) 2021-09-14
AU2017227776B2 (en) 2023-07-06
US20210324387A1 (en) 2021-10-21
AU2017227776A1 (en) 2018-08-23
JP7272795B2 (ja) 2023-05-12
AU2023237106B2 (en) 2025-08-28
AU2023237106A1 (en) 2023-10-19
IL302455A (en) 2023-06-01
ES2923877T3 (es) 2022-10-03
EP3423582A1 (en) 2019-01-09
WO2017151823A1 (en) 2017-09-08
IL261014B1 (en) 2023-06-01
IL261014A (en) 2018-10-31
US20190093111A1 (en) 2019-03-28
CN115896106A (zh) 2023-04-04

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