LTC2548560I2 - Kompozicijos ir būdai, skirti SMN2 splaisingo moduliavimui - Google Patents

Kompozicijos ir būdai, skirti SMN2 splaisingo moduliavimui

Info

Publication number
LTC2548560I2
LTC2548560I2 LTPA2017037C LTPA2017037C LTC2548560I2 LT C2548560 I2 LTC2548560 I2 LT C2548560I2 LT PA2017037 C LTPA2017037 C LT PA2017037C LT PA2017037 C LTPA2017037 C LT PA2017037C LT C2548560 I2 LTC2548560 I2 LT C2548560I2
Authority
LT
Lithuania
Prior art keywords
compositions
techniques
smn2 splicing
modulating smn2
modulating
Prior art date
Application number
LTPA2017037C
Other languages
English (en)
Original Assignee
Cold Spring Harbor Laboratory
Biogen Ma Inc.
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 Cold Spring Harbor Laboratory, Biogen Ma Inc. filed Critical Cold Spring Harbor Laboratory
Publication of LTPA2017037I1 publication Critical patent/LTPA2017037I1/lt
Publication of LTC2548560I2 publication Critical patent/LTC2548560I2/lt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
LTPA2017037C 2005-06-23 2017-11-10 Kompozicijos ir būdai, skirti SMN2 splaisingo moduliavimui LTC2548560I2 (lt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US69354205P 2005-06-23 2005-06-23
EP06773838A EP1910395B1 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of SMN2 splicing

Publications (2)

Publication Number Publication Date
LTPA2017037I1 LTPA2017037I1 (lt) 2017-11-27
LTC2548560I2 true LTC2548560I2 (lt) 2018-11-12

Family

ID=37595872

Family Applications (2)

Application Number Title Priority Date Filing Date
LTEP17185196.7T LT3308788T (lt) 2005-06-23 2006-06-23 Kompozicijos ir būdai, skirti smn2 splaisingo moduliavimui
LTPA2017037C LTC2548560I2 (lt) 2005-06-23 2017-11-10 Kompozicijos ir būdai, skirti SMN2 splaisingo moduliavimui

Family Applications Before (1)

Application Number Title Priority Date Filing Date
LTEP17185196.7T LT3308788T (lt) 2005-06-23 2006-06-23 Kompozicijos ir būdai, skirti smn2 splaisingo moduliavimui

Country Status (13)

Country Link
US (6) US8361977B2 (lt)
EP (5) EP1910395B1 (lt)
CY (2) CY2017040I1 (lt)
DK (3) DK1910395T3 (lt)
ES (3) ES2702531T3 (lt)
HK (2) HK1181307A1 (lt)
HU (2) HUE027486T2 (lt)
LT (2) LT3308788T (lt)
LU (1) LUC00046I2 (lt)
PL (3) PL2548560T3 (lt)
PT (3) PT1910395E (lt)
SI (2) SI2548560T1 (lt)
WO (1) WO2007002390A2 (lt)

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