CA3124963A1 - Dux4 rna silencing using rna targeting crispr-cas13b - Google Patents

Dux4 rna silencing using rna targeting crispr-cas13b Download PDF

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Publication number
CA3124963A1
CA3124963A1 CA3124963A CA3124963A CA3124963A1 CA 3124963 A1 CA3124963 A1 CA 3124963A1 CA 3124963 A CA3124963 A CA 3124963A CA 3124963 A CA3124963 A CA 3124963A CA 3124963 A1 CA3124963 A1 CA 3124963A1
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Canada
Prior art keywords
dux4
nucleic acid
seq
sequence set
nucleotide sequence
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Pending
Application number
CA3124963A
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English (en)
French (fr)
Inventor
Scott Quenton HARPER
Afrooz RASHNONEJAD
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Nationwide Childrens Hospital Inc
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Nationwide Childrens Hospital Inc
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Publication of CA3124963A1 publication Critical patent/CA3124963A1/en
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    • 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
    • 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
    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
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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
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    • 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
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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
    • C12N15/87—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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    • C12N9/00—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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    • 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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Neurology (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (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)
CA3124963A 2018-12-31 2019-12-31 Dux4 rna silencing using rna targeting crispr-cas13b Pending CA3124963A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862786670P 2018-12-31 2018-12-31
US62/786,670 2018-12-31
PCT/US2019/069048 WO2020142479A1 (en) 2018-12-31 2019-12-31 Dux4 rna silencing using rna targeting crispr-cas13b

Publications (1)

Publication Number Publication Date
CA3124963A1 true CA3124963A1 (en) 2020-07-09

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ID=69376003

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CA3124963A Pending CA3124963A1 (en) 2018-12-31 2019-12-31 Dux4 rna silencing using rna targeting crispr-cas13b

Country Status (8)

Country Link
US (1) US12545909B2 (https=)
EP (1) EP3906308A1 (https=)
JP (2) JP2022516515A (https=)
KR (1) KR20210110345A (https=)
AU (1) AU2019419494B2 (https=)
CA (1) CA3124963A1 (https=)
IL (1) IL284447B2 (https=)
WO (1) WO2020142479A1 (https=)

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JP2022516515A (ja) 2022-02-28
IL284447A (en) 2021-08-31
US12545909B2 (en) 2026-02-10
EP3906308A1 (en) 2021-11-10
US20220106592A1 (en) 2022-04-07
AU2019419494B2 (en) 2026-03-26
KR20210110345A (ko) 2021-09-07
IL284447B2 (en) 2026-04-01
IL284447B1 (en) 2025-12-01
JP2025032342A (ja) 2025-03-11
AU2019419494A1 (en) 2021-07-15
WO2020142479A1 (en) 2020-07-09

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