AU2014223243B2 - Talen-based gene correction - Google Patents

Talen-based gene correction Download PDF

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Publication number
AU2014223243B2
AU2014223243B2 AU2014223243A AU2014223243A AU2014223243B2 AU 2014223243 B2 AU2014223243 B2 AU 2014223243B2 AU 2014223243 A AU2014223243 A AU 2014223243A AU 2014223243 A AU2014223243 A AU 2014223243A AU 2014223243 B2 AU2014223243 B2 AU 2014223243B2
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Australia
Prior art keywords
talen
nucleic acid
cell
sequence
gene
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Ceased
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AU2014223243A
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AU2014223243A1 (en
Inventor
Bruce Blazar
Mark J. Osborn
Jakub Tolar
Daniel F. Voytas
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University of Minnesota System
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University of Minnesota Twin Cities
University of Minnesota System
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Publication of AU2014223243A1 publication Critical patent/AU2014223243A1/en
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Publication of AU2014223243B2 publication Critical patent/AU2014223243B2/en
Priority to AU2020200307A priority Critical patent/AU2020200307A1/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Rheumatology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
AU2014223243A 2013-03-01 2014-02-28 Talen-based gene correction Ceased AU2014223243B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2020200307A AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361771735P 2013-03-01 2013-03-01
US61/771,735 2013-03-01
PCT/US2014/019322 WO2014134412A1 (en) 2013-03-01 2014-02-28 Talen-based gene correction

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2020200307A Division AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Publications (2)

Publication Number Publication Date
AU2014223243A1 AU2014223243A1 (en) 2015-09-10
AU2014223243B2 true AU2014223243B2 (en) 2019-10-17

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
AU2014223243A Ceased AU2014223243B2 (en) 2013-03-01 2014-02-28 Talen-based gene correction
AU2020200307A Abandoned AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2020200307A Abandoned AU2020200307A1 (en) 2013-03-01 2020-01-16 Talen-based gene correction

Country Status (7)

Country Link
US (4) US9393257B2 (enExample)
EP (2) EP2961262B1 (enExample)
JP (3) JP6480874B2 (enExample)
AU (2) AU2014223243B2 (enExample)
CA (1) CA2902954A1 (enExample)
ES (1) ES2750550T3 (enExample)
WO (1) WO2014134412A1 (enExample)

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JP2019089821A (ja) 2019-06-13
US10172880B2 (en) 2019-01-08
US9393257B2 (en) 2016-07-19
EP2961262A4 (en) 2016-10-12
ES2750550T3 (es) 2020-03-26
AU2014223243A1 (en) 2015-09-10
US20160367588A1 (en) 2016-12-22
HK1219021A1 (en) 2017-03-24
CA2902954A1 (en) 2014-09-04
NZ711254A (en) 2021-02-26
US20190054111A1 (en) 2019-02-21
JP6893945B2 (ja) 2021-06-23
AU2020200307A1 (en) 2020-02-06
JP2021088565A (ja) 2021-06-10
JP6480874B2 (ja) 2019-03-13
JP2016512960A (ja) 2016-05-12
US20140256798A1 (en) 2014-09-11
EP2961262A1 (en) 2016-01-06
EP3567104A1 (en) 2019-11-13
EP2961262B1 (en) 2019-07-24
US20210187006A1 (en) 2021-06-24
US10973844B2 (en) 2021-04-13
WO2014134412A1 (en) 2014-09-04

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