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 nucleic acids [NA]
    • 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 nucleic acids [NA]
    • 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 (5) US20170051286A1 (fr)
EP (1) EP3137606A4 (fr)
MA (1) MA39941A (fr)
WO (1) WO2015168661A1 (fr)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (fr) 2015-08-14 2017-02-23 University Of Massachusetts Conjugués bioactifs pour l'administration d'oligonucléotides
AU2016366229A1 (en) * 2015-12-09 2018-05-17 Excision Biotherapeutics, Inc. Gene editing methods and compositions for eliminating risk of JC virus activation and PML (progressive multifocal leukoencephalopathy) during immunosuppresive therapy
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
CA3033368A1 (fr) 2016-08-12 2018-02-15 University Of Massachusetts Oligonucleotides conjugues
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
EP3691657A4 (fr) 2017-10-04 2021-07-21 Avidity Biosciences, Inc. Compositions d'acide nucléique-polypeptide et utilisations de celles-ci
EP3735252A4 (fr) * 2018-01-04 2021-10-06 Avidity Biosciences, Inc. Molécules d'acide nucléique hétéroduplex et leurs utilisations
EP3781677A4 (fr) 2018-04-16 2022-01-19 University of Massachusetts Compositions et méthodes pour l'édition génétique améliorée
WO2020033899A1 (fr) * 2018-08-10 2020-02-13 University Of Massachusetts Oligonucléotides modifiés ciblant des snp
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
EP3880820A4 (fr) * 2018-11-01 2022-11-30 The National Institute for Biotechnology in the Negev Ltd. Molécules de silençage vdac1 et leur utilisation
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
US11820985B2 (en) 2019-03-26 2023-11-21 University Of Massachusetts Modified oligonucleotides with increased stability
CN114375296A (zh) 2019-06-06 2022-04-19 艾维迪提生物科学公司 核酸多肽组合物及其用途
US12006499B2 (en) 2019-06-06 2024-06-11 Avidity Biosciences, Inc. Una amidites and uses thereof
US20220348912A1 (en) 2019-06-20 2022-11-03 University Of Massachusetts Compositions and methods for improved gene editing
JP7692829B2 (ja) 2019-07-30 2025-06-16 塩野義製薬株式会社 Ｍｕｒｆ１を標的とする核酸医薬
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
JP7837560B2 (ja) 2020-03-26 2026-03-31 ユニバーシティー オブ マサチューセッツ 安定性の向上を有する修飾オリゴヌクレオチドの合成
WO2021242883A1 (fr) 2020-05-26 2021-12-02 University Of Massachusetts Oligonucléotides synthétiques ayant des régions de séquences et de modifications de groupe
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
AU2022245292A1 (en) * 2021-03-26 2023-10-12 Neumirna Therapeutics Aps Microrna-134 inhibitors
WO2022200633A1 (fr) * 2021-03-26 2022-09-29 Neumirna Therapeutics Aps Inhibiteurs de microarn-27 b
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
EP4372085A4 (fr) * 2021-07-16 2026-01-21 Suzhou Ribo Life Science Co Ltd Acide nucléique, composition et conjugué le contenant, procédé de préparation et utilisation

Family Cites Families (9)

* Cited by examiner, † Cited by third party
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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
WO2008111908A1 (fr) * 2007-03-15 2008-09-18 Jyoti Chattopadhyaya Ribothymidines carbocycliques 2', 4' à conformation verrouillée à 5 et 6 éléments destinées à traiter des infections et le 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'
EP2601204B1 (fr) * 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Nucléosides modifiés et composés oligomères préparés à partir de ceux-ci
CA2871089A1 (fr) * 2011-04-20 2012-10-26 Smith Holdings, Llc Procedes et compositions de modulation de l'expression des genes a l'aide de composants qui sont auto-assembles dans des cellules et qui produisent une activite d'arni
WO2013033230A1 (fr) * 2011-08-29 2013-03-07 Isis 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
WO2015168661A1 (fr) 2015-11-05
US20230220393A1 (en) 2023-07-13
US20250101422A1 (en) 2025-03-27
US20200362343A1 (en) 2020-11-19
US20170051286A1 (en) 2017-02-23
US20190300878A1 (en) 2019-10-03
EP3137606A1 (fr) 2017-03-08
EP3137606A4 (fr) 2018-04-25

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