EA201492123A1 - Композиции и способы для модулирования экспрессии семейства генов smn - Google Patents

Композиции и способы для модулирования экспрессии семейства генов smn

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Publication number
EA201492123A1
EA201492123A1 EA201492123A EA201492123A EA201492123A1 EA 201492123 A1 EA201492123 A1 EA 201492123A1 EA 201492123 A EA201492123 A EA 201492123A EA 201492123 A EA201492123 A EA 201492123A EA 201492123 A1 EA201492123 A1 EA 201492123A1
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EA
Eurasian Patent Office
Prior art keywords
expression
methods
modulating
compositions
smn2
Prior art date
Application number
EA201492123A
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English (en)
Russian (ru)
Inventor
Артур М. Криг
Ромеш Субраманиан
Джеймс Максвиджен
Джинни Т. Ли
Original Assignee
Рана Терапьютикс, Инк.
ДЗЕ ДЖЕНЕРАЛ ХОСПИТАЛ КОРПОРЕЙШН д/б/а МАССАЧУСЕТС ДЖЕНЕРАЛ ХОСПИТАЛ
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Application filed by Рана Терапьютикс, Инк., ДЗЕ ДЖЕНЕРАЛ ХОСПИТАЛ КОРПОРЕЙШН д/б/а МАССАЧУСЕТС ДЖЕНЕРАЛ ХОСПИТАЛ filed Critical Рана Терапьютикс, Инк.
Publication of EA201492123A1 publication Critical patent/EA201492123A1/ru

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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
    • C12N15/1131—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 against viruses
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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
    • C12N15/1131—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 against viruses
    • C12N15/1132—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 against viruses against retroviridae, e.g. HIV
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
    • C12N2310/3517—Marker; Tag

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
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  • Plant Pathology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EA201492123A 2012-05-16 2013-05-16 Композиции и способы для модулирования экспрессии семейства генов smn EA201492123A1 (ru)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201261647858P 2012-05-16 2012-05-16
US201261719394P 2012-10-27 2012-10-27
US201361785529P 2013-03-14 2013-03-14
PCT/US2013/041440 WO2013173638A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating smn gene family expression

Publications (1)

Publication Number Publication Date
EA201492123A1 true EA201492123A1 (ru) 2015-10-30

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EA201492123A EA201492123A1 (ru) 2012-05-16 2013-05-16 Композиции и способы для модулирования экспрессии семейства генов smn

Country Status (13)

Country Link
US (1) US10059941B2 (enExample)
EP (1) EP2850186B1 (enExample)
JP (1) JP2015523854A (enExample)
KR (1) KR20150030205A (enExample)
CN (1) CN104540947A (enExample)
AU (1) AU2013262649A1 (enExample)
CA (1) CA2873794A1 (enExample)
DK (1) DK2850186T3 (enExample)
EA (1) EA201492123A1 (enExample)
HK (1) HK1208700A1 (enExample)
SG (1) SG11201407483YA (enExample)
WO (1) WO2013173638A1 (enExample)
ZA (1) ZA201409228B (enExample)

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US20150252364A1 (en) 2015-09-10
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US10059941B2 (en) 2018-08-28
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JP2015523854A (ja) 2015-08-20
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