KR20150133752A - 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 - Google Patents

근육 이영양증의 치료를 위한 엑손 스키핑 조성물 Download PDF

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KR20150133752A
KR20150133752A KR1020157028188A KR20157028188A KR20150133752A KR 20150133752 A KR20150133752 A KR 20150133752A KR 1020157028188 A KR1020157028188 A KR 1020157028188A KR 20157028188 A KR20157028188 A KR 20157028188A KR 20150133752 A KR20150133752 A KR 20150133752A
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antisense
antisense oligomer
oligomer
exon
dystrophin
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리차드 케이. 베스트윅
다이앤 엘리자베스 프랭크
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사렙타 쎄러퓨틱스 인코퍼레이티드
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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
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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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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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
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    • C12N2310/30—Chemical structure
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    • C12N2310/3513—Protein; Peptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • 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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  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
KR1020157028188A 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 Ceased KR20150133752A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361782706P 2013-03-14 2013-03-14
US61/782,706 2013-03-14
PCT/US2014/029766 WO2014153240A2 (en) 2013-03-14 2014-03-14 Exon skipping compositions for treating muscular dystrophy

Related Child Applications (1)

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KR1020217000648A Division KR20210006516A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

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KR20150133752A true KR20150133752A (ko) 2015-11-30

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KR1020247018517A Ceased KR20240094032A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020217000648A Ceased KR20210006516A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020237016315A Ceased KR20230074606A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020227014526A Ceased KR20220061280A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020157028188A Ceased KR20150133752A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

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KR1020247018517A Ceased KR20240094032A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020217000648A Ceased KR20210006516A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020237016315A Ceased KR20230074606A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020227014526A Ceased KR20220061280A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

Country Status (26)

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US (5) US20160040162A1 (enExample)
EP (3) EP3760720A1 (enExample)
JP (4) JP2016521119A (enExample)
KR (5) KR20240094032A (enExample)
CN (2) CN105392884B (enExample)
AU (3) AU2014236160A1 (enExample)
BR (1) BR112015023038A2 (enExample)
CA (1) CA2903872A1 (enExample)
CY (1) CY1121876T1 (enExample)
DK (1) DK2970964T3 (enExample)
EA (1) EA201591694A1 (enExample)
ES (1) ES2714290T3 (enExample)
HR (1) HRP20190382T1 (enExample)
HU (1) HUE042218T2 (enExample)
IL (2) IL241047B (enExample)
LT (1) LT2970964T (enExample)
MX (2) MX366274B (enExample)
NZ (1) NZ631132A (enExample)
PL (1) PL2970964T3 (enExample)
PT (1) PT2970964T (enExample)
RS (1) RS58529B1 (enExample)
SA (1) SA515361121B1 (enExample)
SI (1) SI2970964T1 (enExample)
SM (1) SMT201900139T1 (enExample)
TR (1) TR201903009T4 (enExample)
WO (1) WO2014153240A2 (enExample)

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KR20190015311A (ko) * 2016-05-24 2019-02-13 사렙타 쎄러퓨틱스 인코퍼레이티드 올리고머의 제조 방법

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