MA39941A - Procédés et modifications permettant de produire des composés arni simple brin à activité, potentiel et durée d'effet améliorés - Google Patents

Procédés et modifications permettant de produire des composés arni simple brin à activité, potentiel et durée d'effet améliorés

Info

Publication number
MA39941A
MA39941A MA039941A MA39941A MA39941A MA 39941 A MA39941 A MA 39941A MA 039941 A MA039941 A MA 039941A MA 39941 A MA39941 A MA 39941A MA 39941 A MA39941 A MA 39941A
Authority
MA
Morocco
Prior art keywords
methods
potency
duration
produce
modifications
Prior art date
Application number
MA039941A
Other languages
English (en)
Inventor
Larry J Smith
Original Assignee
Larry J Smith
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 Larry J Smith filed Critical Larry J Smith
Publication of MA39941A publication Critical patent/MA39941A/fr

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N15/11—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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N15/11—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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

L'invention concerne des compositions et des procédés permettant de moduler à la baisse l'expression d'acides nucléiques cibles au moyen de composés arnsi d'oligoribonucléotides monocaténaires.
MA039941A 2014-05-01 2015-05-01 Procédés et modifications permettant de produire des composés arni simple brin à activité, potentiel et durée d'effet améliorés MA39941A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201461987448P 2014-05-01 2014-05-01

Publications (1)

Publication Number Publication Date
MA39941A true MA39941A (fr) 2017-03-08

Family

ID=54359412

Family Applications (1)

Application Number Title Priority Date Filing Date
MA039941A MA39941A (fr) 2014-05-01 2015-05-01 Procédés et modifications permettant de produire des composés arni simple brin à activité, potentiel et durée d'effet améliorés

Country Status (4)

Country Link
US (4) US20170051286A1 (fr)
EP (1) EP3137606A4 (fr)
MA (1) MA39941A (fr)
WO (1) WO2015168661A1 (fr)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
WO2018031933A2 (fr) 2016-08-12 2018-02-15 University Of Massachusetts Oligonucléotides conjugués
MX2020003596A (es) 2017-10-04 2020-07-22 Avidity Biosciences Inc Composiciones de acido nucleico-polipeptido y usos de las mismos.
US20210095283A1 (en) * 2018-01-04 2021-04-01 Avidity Biosciences, Inc. Heteroduplex nucleic acid molecules and uses thereof
EP3781677A4 (fr) 2018-04-16 2022-01-19 University of Massachusetts Compositions et méthodes pour l'édition génétique améliorée
KR20210093227A (ko) * 2018-08-10 2021-07-27 유니버시티 오브 매사추세츠 Snp를 표적화하는 변형된 올리고뉴클레오티드
JP2022528840A (ja) 2019-03-26 2022-06-16 ユニバーシティ・オブ・マサチューセッツ 安定性が増加した修飾オリゴヌクレオチド
WO2020247782A1 (fr) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Compositions d'acide nucléique-polypeptide et utilisations de celles-ci
EP3987024A4 (fr) 2019-06-20 2023-11-01 University Of Massachusetts Compositions et méthodes pour l'édition génomique améliorée
WO2021020412A1 (fr) 2019-07-30 2021-02-04 塩野義製薬株式会社 Médicament à base d'acide nucléique ciblant le murf1
CN112587663B (zh) * 2020-12-29 2022-01-07 浙江大学 长链非编码RNA-lncIVRL在防治甲型流感病毒感染中的应用
EP4359539A1 (fr) 2021-06-23 2024-05-01 University Of Massachusetts Composés oligonucléotidiques anti-flt1 optimisés pour le traitement de la prééclampsie et d'autres troubles angiogéniques

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5683987A (en) * 1994-07-12 1997-11-04 The Board Of Regents Of The University Of Nebraska Therapeutic oligonucleotides targeting the human MDR1 and MRP genes
US8093061B2 (en) * 2006-10-18 2012-01-10 Isis Pharmaceuticals, Inc. Methods of determining potency of chemically-synthesized oligonucleotides
US8461124B2 (en) * 2007-03-15 2013-06-11 Jyoti Chattopadhyaya Five- and six-membered conformationally locked 2′,4′-carbocyclic ribo-thymidines for the treatment of infections and cancer
US20150025122A1 (en) * 2009-10-12 2015-01-22 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
US9725479B2 (en) * 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
CN103154014B (zh) * 2010-04-28 2015-03-25 Isis制药公司 修饰核苷、其类似物以及由它们制备的寡聚化合物
RU2013151301A (ru) * 2011-04-20 2015-05-27 Ларри Дж. СМИТ Способы и композиции для модулирования экспрессии генов с использованием компонентов, которые обладают способностью к самосборке в клетках и обусловливают рнкi-активность
EP2751270B1 (fr) * 2011-08-29 2018-08-22 Ionis Pharmaceuticals, Inc. Complexes oligomère-conjugué et leur utilisation
WO2014066915A2 (fr) * 2012-10-26 2014-05-01 Smith Larry J Procédés et compositions pour produire une activité ss-arni ayant une puissance accrue

Also Published As

Publication number Publication date
US20170051286A1 (en) 2017-02-23
US20190300878A1 (en) 2019-10-03
US20200362343A1 (en) 2020-11-19
EP3137606A4 (fr) 2018-04-25
US20230220393A1 (en) 2023-07-13
EP3137606A1 (fr) 2017-03-08
WO2015168661A1 (fr) 2015-11-05

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