CA2990699A1 - Modified crispr rna and modified single crispr rna and uses thereof - Google Patents

Modified crispr rna and modified single crispr rna and uses thereof Download PDF

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Publication number
CA2990699A1
CA2990699A1 CA2990699A CA2990699A CA2990699A1 CA 2990699 A1 CA2990699 A1 CA 2990699A1 CA 2990699 A CA2990699 A CA 2990699A CA 2990699 A CA2990699 A CA 2990699A CA 2990699 A1 CA2990699 A1 CA 2990699A1
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CA
Canada
Prior art keywords
compound
modified
crrna
scrrna
recognition portion
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Abandoned
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CA2990699A
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English (en)
French (fr)
Inventor
Meghdad RAHDAR
Thazha P. Prakash
Eric E. Swayze
Frank C. Bennett
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of CA2990699A1 publication Critical patent/CA2990699A1/en
Abandoned legal-status Critical Current

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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
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    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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CA2990699A 2015-06-29 2016-06-29 Modified crispr rna and modified single crispr rna and uses thereof Abandoned CA2990699A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562186363P 2015-06-29 2015-06-29
US62/186,363 2015-06-29
US201562233281P 2015-09-25 2015-09-25
US62/233,281 2015-09-25
US201562256649P 2015-11-17 2015-11-17
US62/256,649 2015-11-17
PCT/US2016/040191 WO2017004261A1 (en) 2015-06-29 2016-06-29 Modified crispr rna and modified single crispr rna and uses thereof

Publications (1)

Publication Number Publication Date
CA2990699A1 true CA2990699A1 (en) 2017-01-05

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CA2990699A Abandoned CA2990699A1 (en) 2015-06-29 2016-06-29 Modified crispr rna and modified single crispr rna and uses thereof

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Country Link
US (2) US11414657B2 (https=)
EP (2) EP4545544A3 (https=)
JP (1) JP2018519811A (https=)
AU (1) AU2016285724A1 (https=)
CA (1) CA2990699A1 (https=)
WO (1) WO2017004261A1 (https=)

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US11414657B2 (en) 2022-08-16
EP4545544A2 (en) 2025-04-30
EP3313989A4 (en) 2018-12-05
EP3313989B1 (en) 2024-12-25
EP4545544A3 (en) 2025-10-08
US20180179521A1 (en) 2018-06-28
JP2018519811A (ja) 2018-07-26
US20230159919A1 (en) 2023-05-25

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