CA2951208A1 - Reducing intron retention - Google Patents

Reducing intron retention Download PDF

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Publication number
CA2951208A1
CA2951208A1 CA2951208A CA2951208A CA2951208A1 CA 2951208 A1 CA2951208 A1 CA 2951208A1 CA 2951208 A CA2951208 A CA 2951208A CA 2951208 A CA2951208 A CA 2951208A CA 2951208 A1 CA2951208 A1 CA 2951208A1
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Prior art keywords
seq
acid polymer
polynucleic acid
sequence
protein
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CA2951208A
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English (en)
French (fr)
Inventor
Igor Vorechovsky
Jana KRALOVICOVA
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University of Southampton
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University of Southampton
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
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    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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    • 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
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    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CA2951208A 2014-06-16 2015-06-16 Reducing intron retention Pending CA2951208A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1410693.4A GB201410693D0 (en) 2014-06-16 2014-06-16 Splicing modulation
GB1410693.4 2014-06-16
PCT/GB2015/051756 WO2015193651A1 (en) 2014-06-16 2015-06-16 Reducing intron retention

Publications (1)

Publication Number Publication Date
CA2951208A1 true CA2951208A1 (en) 2015-12-23

Family

ID=51266659

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2951208A Pending CA2951208A1 (en) 2014-06-16 2015-06-16 Reducing intron retention

Country Status (12)

Country Link
US (6) US9745577B2 (enExample)
EP (2) EP4015648B1 (enExample)
JP (3) JP7043169B2 (enExample)
KR (1) KR102638276B1 (enExample)
CN (1) CN107073137A (enExample)
AU (2) AU2015275870B2 (enExample)
CA (1) CA2951208A1 (enExample)
ES (1) ES2907033T3 (enExample)
GB (1) GB201410693D0 (enExample)
IL (2) IL249392B (enExample)
SG (2) SG11201610480XA (enExample)
WO (1) WO2015193651A1 (enExample)

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