CN104245935B - 三环硫代磷酸酯dna - Google Patents

三环硫代磷酸酯dna Download PDF

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Publication number
CN104245935B
CN104245935B CN201280057688.6A CN201280057688A CN104245935B CN 104245935 B CN104245935 B CN 104245935B CN 201280057688 A CN201280057688 A CN 201280057688A CN 104245935 B CN104245935 B CN 104245935B
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mrna
dna
nucleic acid
acid molecules
tricyclic
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CN104245935A (zh
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克里斯蒂安·卢曼
路易斯·加西亚
托马斯·沃伊特
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France National Medical Association Of Health And Research
Universitaet Bern
Centre National de la Recherche Scientifique CNRS
Universite Pierre et Marie Curie
Association Institut de Myologie
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France National Medical Association Of Health And Research
Universitaet Bern
Centre National de la Recherche Scientifique CNRS
Universite Pierre et Marie Curie
Association Institut de Myologie
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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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
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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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2320/33Alteration of splicing

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CN201280057688.6A 2011-10-13 2012-10-12 三环硫代磷酸酯dna Active CN104245935B (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201161546942P 2011-10-13 2011-10-13
EP11185129.1 2011-10-13
US61/546,942 2011-10-13
EP11185129.1A EP2581448B1 (en) 2011-10-13 2011-10-13 Tricyclo-phosphorothioate DNA
CA2776651A CA2776651C (en) 2011-10-13 2012-04-27 Tricyclo-phosphorothioate dna
CA2,776,651 2012-04-27
PCT/EP2012/070349 WO2013053928A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna

Publications (2)

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CN104245935A CN104245935A (zh) 2014-12-24
CN104245935B true CN104245935B (zh) 2018-05-08

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US (1) US9738891B2 (https=)
EP (2) EP2581448B1 (https=)
JP (1) JP6181653B2 (https=)
CN (1) CN104245935B (https=)
AU (1) AU2012322903B2 (https=)
BR (1) BR112014009066B8 (https=)
CA (2) CA2776651C (https=)
DK (1) DK2581448T3 (https=)
ES (2) ES2535654T3 (https=)
IL (1) IL231983B (https=)
IN (1) IN2014DN03463A (https=)
PL (1) PL2581448T3 (https=)
PT (1) PT2581448E (https=)
WO (1) WO2013053928A1 (https=)

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JP2015501144A (ja) 2015-01-15
CA2776651A1 (en) 2013-04-13
BR112014009066B1 (pt) 2020-08-25
WO2013053928A1 (en) 2013-04-18
EP2581448A1 (en) 2013-04-17
CA2851970A1 (en) 2013-04-18
IL231983A0 (en) 2014-05-28
ES2535654T3 (es) 2015-05-13
CN104245935A (zh) 2014-12-24
ES2651216T3 (es) 2018-01-25
HK1186205A1 (en) 2014-03-07
BR112014009066A2 (pt) 2017-04-18
AU2012322903B2 (en) 2017-09-14
JP6181653B2 (ja) 2017-08-16
EP2766479B1 (en) 2017-09-13
IN2014DN03463A (https=) 2015-06-05
PL2581448T3 (pl) 2015-08-31
BR112014009066A8 (pt) 2018-01-09
EP2766479A1 (en) 2014-08-20
AU2012322903A1 (en) 2014-05-29
IL231983B (en) 2018-04-30
EP2581448B1 (en) 2015-01-28
BR112014009066B8 (pt) 2021-02-23
DK2581448T3 (en) 2015-04-27
US20140296323A1 (en) 2014-10-02
US9738891B2 (en) 2017-08-22
CA2776651C (en) 2021-06-08
PT2581448E (pt) 2015-05-21

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