MX2016002044A - Composiciones y metodos para modular el acido ribonucleico. - Google Patents

Composiciones y metodos para modular el acido ribonucleico.

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Publication number
MX2016002044A
MX2016002044A MX2016002044A MX2016002044A MX2016002044A MX 2016002044 A MX2016002044 A MX 2016002044A MX 2016002044 A MX2016002044 A MX 2016002044A MX 2016002044 A MX2016002044 A MX 2016002044A MX 2016002044 A MX2016002044 A MX 2016002044A
Authority
MX
Mexico
Prior art keywords
methods
compositions
aspects
useful
modulating rna
Prior art date
Application number
MX2016002044A
Other languages
English (en)
Inventor
OZSOLAK Fatih
Woo Caroline
Original Assignee
Rana Therapeutics 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 Rana Therapeutics Inc filed Critical Rana Therapeutics Inc
Publication of MX2016002044A publication Critical patent/MX2016002044A/es

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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
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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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
    • C12N2310/322—2'-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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Abstract

Los aspectos de la invención se refieren a métodos para incrementar la expresión de genes en una forma dirigida; en algunas modalidades, se proporcionan los métodos y las composiciones que son útiles para alterar de manera post-transcripcional la proteína y/o los niveles de ARN en una forma dirigida; los aspectos de la invención que se describen en la presente proporcionan métodos y composiciones que son útiles para proteger los ARN de la degradación (por ejemplo, degradación mediada por exonucleasa).
MX2016002044A 2013-08-16 2014-08-15 Composiciones y metodos para modular el acido ribonucleico. MX2016002044A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361866989P 2013-08-16 2013-08-16
US201361898461P 2013-10-31 2013-10-31
US201462010417P 2014-06-10 2014-06-10
PCT/US2014/051331 WO2015023975A1 (en) 2013-08-16 2014-08-15 Compositions and methods for modulating rna

Publications (1)

Publication Number Publication Date
MX2016002044A true MX2016002044A (es) 2016-08-17

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016002044A MX2016002044A (es) 2013-08-16 2014-08-15 Composiciones y metodos para modular el acido ribonucleico.

Country Status (13)

Country Link
US (8) US20150050738A1 (es)
EP (1) EP3033424A4 (es)
JP (1) JP2016528897A (es)
KR (1) KR20160036065A (es)
CN (1) CN105658797A (es)
AU (2) AU2014306416B2 (es)
BR (1) BR112016003127A2 (es)
CA (1) CA2921556A1 (es)
EA (1) EA201690403A1 (es)
IL (1) IL244081A0 (es)
MX (1) MX2016002044A (es)
SG (1) SG11201600987TA (es)
WO (1) WO2015023975A1 (es)

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