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

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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 (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3334499A4 (fr) 2015-08-14 2019-04-17 University of Massachusetts Conjugués bioactifs pour l'administration d'oligonucléotides
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
CA3033368A1 (fr) 2016-08-12 2018-02-15 University Of Massachusetts Oligonucleotides conjugues
US10844377B2 (en) 2017-06-23 2020-11-24 University Of Massachusetts Two-tailed self-delivering siRNA
JP2021500862A (ja) 2017-10-04 2021-01-14 アビディティー バイオサイエンシーズ，インク． 核酸ポリペプチド組成物およびその使用
EP3735252A4 (fr) * 2018-01-04 2021-10-06 Avidity Biosciences, Inc. Molécules d'acide nucléique hétéroduplex et leurs utilisations
WO2019204226A1 (fr) 2018-04-16 2019-10-24 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
WO2020089906A1 (fr) * 2018-11-01 2020-05-07 National Institute For Biotechnology In The Negev Ltd. Molécules de silençage vdac1 et leur utilisation
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
US12006499B2 (en) 2019-06-06 2024-06-11 Avidity Biosciences, Inc. Una amidites and uses thereof
US11578090B2 (en) 2019-06-06 2023-02-14 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
US20220348912A1 (en) 2019-06-20 2022-11-03 University Of Massachusetts Compositions and methods for improved gene editing
JPWO2021020412A1 (fr) 2019-07-30 2021-02-04
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
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
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物

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
WO2008049089A2 (fr) * 2006-10-18 2008-04-24 Isis Pharmaceuticals, Inc. Procédés de détermination de la puissance d'oligonucléotides synthétisés chimiquement
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
WO2011133871A2 (fr) * 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Dérivés d'extrémité 5'
US8993738B2 (en) * 2010-04-28 2015-03-31 Isis Pharmaceuticals, Inc. Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
BR112013027070A2 (pt) * 2011-04-20 2019-09-24 J Smith Larry métodos e composições para diminuir expressão de gene utilizando componentes que auto montam em células e produzem atividade de rnai
DK2751270T3 (en) * 2011-08-29 2018-10-29 Ionis Pharmaceuticals Inc OLIGOMER-CONJUGATE COMPLEXES AND THEIR USE
US20150291957A1 (en) * 2012-10-26 2015-10-15 Larry J. Smith METHODS AND COMPOSITIONS TO PRODUCE ss-RNAi ACTIVITY WITH ENHANCED POTENCY

Also Published As

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

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