EA201591178A1 - Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии - Google Patents

Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии

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Publication number
EA201591178A1
EA201591178A1 EA201591178A EA201591178A EA201591178A1 EA 201591178 A1 EA201591178 A1 EA 201591178A1 EA 201591178 A EA201591178 A EA 201591178A EA 201591178 A EA201591178 A EA 201591178A EA 201591178 A1 EA201591178 A1 EA 201591178A1
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EA
Eurasian Patent Office
Prior art keywords
distrophia
improved compositions
treating muscle
extract passing
extract
Prior art date
Application number
EA201591178A
Other languages
English (en)
Inventor
Ричард К. Бестуик
Дайан Элизабет Франк
Original Assignee
Сарепта Терапьютикс, Инк.
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Publication date
Application filed by Сарепта Терапьютикс, Инк. filed Critical Сарепта Терапьютикс, Инк.
Publication of EA201591178A1 publication Critical patent/EA201591178A1/ru

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    • 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
    • C12N15/113—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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Abstract

Описаны антисмысловые молекулы, способные к связыванию с выбранным сайтом-мишенью в гене дистрофина человека для индуцирования пропускания экзона 53.
EA201591178A 2012-12-20 2013-12-20 Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии EA201591178A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261739968P 2012-12-20 2012-12-20
PCT/US2013/077216 WO2014100714A1 (en) 2012-12-20 2013-12-20 Improved exon skipping compositions for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
EA201591178A1 true EA201591178A1 (ru) 2015-11-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
EA201591178A EA201591178A1 (ru) 2012-12-20 2013-12-20 Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии

Country Status (13)

Country Link
US (5) US20150361428A1 (ru)
EP (2) EP2935584A1 (ru)
JP (3) JP2016502858A (ru)
KR (2) KR20200143739A (ru)
CN (2) CN106414739A (ru)
AU (3) AU2013364158A1 (ru)
BR (1) BR112015014987A2 (ru)
CA (1) CA2894899A1 (ru)
EA (1) EA201591178A1 (ru)
HK (1) HK1216902A1 (ru)
IL (1) IL239491A0 (ru)
MX (2) MX2015008035A (ru)
WO (1) WO2014100714A1 (ru)

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CN111440796A (zh) 2020-07-24
BR112015014987A2 (pt) 2017-08-15
JP2016502858A (ja) 2016-02-01
US20230104708A1 (en) 2023-04-06
AU2013364158A1 (en) 2015-07-09
US20220259592A1 (en) 2022-08-18
HK1216902A1 (zh) 2016-12-09
AU2019253778A1 (en) 2019-11-07
JP2018110601A (ja) 2018-07-19
WO2014100714A1 (en) 2014-06-26
IL239491A0 (en) 2015-08-31
US20170369875A1 (en) 2017-12-28
CA2894899A1 (en) 2014-06-26
AU2022201628A1 (en) 2022-03-31
EP3885439A1 (en) 2021-09-29
MX2015008035A (es) 2016-03-17
US20200339985A1 (en) 2020-10-29
KR20150099804A (ko) 2015-09-01
JP2019050834A (ja) 2019-04-04
KR20200143739A (ko) 2020-12-24
US20150361428A1 (en) 2015-12-17
EP2935584A1 (en) 2015-10-28
CN106414739A (zh) 2017-02-15
MX2019008164A (es) 2019-09-13

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