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MX2016011538A - Acidos nucleicos antisentido. - Google Patents

Acidos nucleicos antisentido.

Info

Publication number
MX2016011538A
MX2016011538A MX2016011538A MX2016011538A MX2016011538A MX 2016011538 A MX2016011538 A MX 2016011538A MX 2016011538 A MX2016011538 A MX 2016011538A MX 2016011538 A MX2016011538 A MX 2016011538A MX 2016011538 A MX2016011538 A MX 2016011538A
Authority
MX
Mexico
Prior art keywords
nucleic acid
antisense nucleic
exon
omission
dystrophin gene
Prior art date
Application number
MX2016011538A
Other languages
English (en)
Inventor
Wakayama Tatsushi
Seo Haruna
Satou Youhei
Takeda Shin´Ichi
Nagata Tetsuya
Original Assignee
Nippon Shinyaku Co Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Nippon Shinyaku Co Ltd filed Critical Nippon Shinyaku Co Ltd
Publication of MX2016011538A publication Critical patent/MX2016011538A/es

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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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
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  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se proporciona un fármaco que permite la omisión altamente eficiente del exón 51 en el gen de distrofina humana. La presente invención proporciona un oligómero antisentido que permite la omisión del exón 51 en el gen de distrofina humana.
MX2016011538A 2014-03-12 2015-03-11 Acidos nucleicos antisentido. MX2016011538A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2014048897 2014-03-12
PCT/JP2015/057180 WO2015137409A1 (ja) 2014-03-12 2015-03-11 アンチセンス核酸

Publications (1)

Publication Number Publication Date
MX2016011538A true MX2016011538A (es) 2017-04-13

Family

ID=54071850

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016011538A MX2016011538A (es) 2014-03-12 2015-03-11 Acidos nucleicos antisentido.

Country Status (31)

Country Link
US (5) US9988629B2 (es)
EP (2) EP3118311B1 (es)
JP (1) JP6606488B2 (es)
KR (1) KR102335801B1 (es)
CN (2) CN110951732A (es)
AU (2) AU2015227733B2 (es)
BR (1) BR112016020618B1 (es)
CA (1) CA2939948A1 (es)
CY (1) CY1121322T1 (es)
DK (1) DK3118311T3 (es)
ES (1) ES2710802T3 (es)
HR (1) HRP20190235T1 (es)
HU (1) HUE042283T2 (es)
IL (1) IL247663B (es)
LT (1) LT3118311T (es)
MX (1) MX2016011538A (es)
MY (1) MY193708A (es)
NZ (1) NZ724836A (es)
PH (1) PH12016501761A1 (es)
PL (1) PL3118311T3 (es)
PT (1) PT3118311T (es)
RS (1) RS58573B1 (es)
RU (2) RU2702424C2 (es)
SG (1) SG11201607095RA (es)
SI (1) SI3118311T1 (es)
SM (1) SMT201900169T1 (es)
TR (1) TR201901939T4 (es)
TW (2) TWI672314B (es)
UA (1) UA120849C2 (es)
WO (1) WO2015137409A1 (es)
ZA (1) ZA201605864B (es)

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