AU2013262699A1 - Compositions and methods for modulating ATP2A2 expression - Google Patents

Compositions and methods for modulating ATP2A2 expression Download PDF

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Publication number
AU2013262699A1
AU2013262699A1 AU2013262699A AU2013262699A AU2013262699A1 AU 2013262699 A1 AU2013262699 A1 AU 2013262699A1 AU 2013262699 A AU2013262699 A AU 2013262699A AU 2013262699 A AU2013262699 A AU 2013262699A AU 2013262699 A1 AU2013262699 A1 AU 2013262699A1
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AU
Australia
Prior art keywords
oligonucleotide
single stranded
nucleotide
nucleotides
atp2a2
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Abandoned
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AU2013262699A
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English (en)
Inventor
Arthur M. Krieg
Jeannie T. Lee
James Mcswiggen
Romesh Subramanian
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General Hospital Corp
Translate Bio Inc
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General Hospital Corp
RaNA Therapeutics Inc
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Publication of AU2013262699A1 publication Critical patent/AU2013262699A1/en
Abandoned legal-status Critical Current

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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
    • C12N15/1137—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 against enzymes
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
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    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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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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    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03008—Ca2+-transporting ATPase (3.6.3.8)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • 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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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

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  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Veterinary Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2013262699A 2012-05-16 2013-05-16 Compositions and methods for modulating ATP2A2 expression Abandoned AU2013262699A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261647925P 2012-05-16 2012-05-16
US61/647,925 2012-05-16
US201361785832P 2013-03-14 2013-03-14
US61/785,832 2013-03-14
PCT/US2013/041381 WO2013173598A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating atp2a2 expression

Publications (1)

Publication Number Publication Date
AU2013262699A1 true AU2013262699A1 (en) 2015-01-22

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2013262699A Abandoned AU2013262699A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating ATP2A2 expression

Country Status (9)

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US (1) US10174323B2 (https=)
EP (1) EP2850182A4 (https=)
JP (1) JP2015523853A (https=)
CN (1) CN104583401A (https=)
AU (1) AU2013262699A1 (https=)
BR (1) BR112014028644A2 (https=)
CA (1) CA2873766A1 (https=)
EA (1) EA201492120A1 (https=)
WO (1) WO2013173598A1 (https=)

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US12576040B2 (en) 2020-10-14 2026-03-17 The Trustees Of The University Of Pennsylvania Ionizable lipids and methods of manufacture and use thereof

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EA201492120A1 (ru) 2015-10-30
WO2013173598A1 (en) 2013-11-21
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