MX2022012265A - Inhibicion dirigida utilizando oligonucleotidos ingenieria. - Google Patents

Inhibicion dirigida utilizando oligonucleotidos ingenieria.

Info

Publication number
MX2022012265A
MX2022012265A MX2022012265A MX2022012265A MX2022012265A MX 2022012265 A MX2022012265 A MX 2022012265A MX 2022012265 A MX2022012265 A MX 2022012265A MX 2022012265 A MX2022012265 A MX 2022012265A MX 2022012265 A MX2022012265 A MX 2022012265A
Authority
MX
Mexico
Prior art keywords
engineered
targeted inhibition
engineered oligonucleotides
oligonucleotides
disclosed
Prior art date
Application number
MX2022012265A
Other languages
English (en)
Inventor
Robert Place
Anthony Saleh
Tishan Williams
Original Assignee
Mirecule 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 Mirecule Inc filed Critical Mirecule Inc
Publication of MX2022012265A publication Critical patent/MX2022012265A/es

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B15/00—ICT specially adapted for analysing two-dimensional or three-dimensional molecular structures, e.g. structural or functional relations or structure alignment
    • G16B15/30—Drug targeting using structural data; Docking or binding prediction
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
    • G16H50/00—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics
    • G16H50/20—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
    • 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
    • C12N2310/316—Phosphonothioates
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A90/00—Technologies having an indirect contribution to adaptation to climate change
    • Y02A90/10—Information and communication technologies [ICT] supporting adaptation to climate change, e.g. for weather forecasting or climate simulation

Abstract

Se divulgan en la presente oligonucleótidos genéticamente modificados para inhibición selectiva de la expresión y actividad del polipéptido. También se divulgan en la presente métodos para inhibir selectivamente la expresión y actividad del polipéptido poniendo en contacto un oligonucleótido genéticamente modificado con un polinucleótido que codifica el polipéptido.
MX2022012265A 2020-04-02 2021-04-02 Inhibicion dirigida utilizando oligonucleotidos ingenieria. MX2022012265A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063004045P 2020-04-02 2020-04-02
PCT/US2021/025639 WO2021203043A2 (en) 2020-04-02 2021-04-02 Targeted inhibition using engineered oligonucleotides

Publications (1)

Publication Number Publication Date
MX2022012265A true MX2022012265A (es) 2023-01-11

Family

ID=77927597

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022012265A MX2022012265A (es) 2020-04-02 2021-04-02 Inhibicion dirigida utilizando oligonucleotidos ingenieria.

Country Status (12)

Country Link
US (4) US20230132602A1 (es)
EP (1) EP4127175A2 (es)
JP (1) JP2023521042A (es)
KR (1) KR20230023612A (es)
CN (1) CN116096890A (es)
AU (1) AU2021246530A1 (es)
BR (1) BR112022019889A2 (es)
CA (1) CA3174286A1 (es)
CO (1) CO2022015141A2 (es)
IL (1) IL296957A (es)
MX (1) MX2022012265A (es)
WO (1) WO2021203043A2 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240032998A (ko) 2021-07-14 2024-03-12 마이레큘, 인크. 신경근육장애를 위한 올리고뉴클레오티드 및 이의 조성물
WO2023122347A2 (en) 2021-12-23 2023-06-29 Mirecule, Inc. Compositions for delivery of polynucleotides
KR20230102595A (ko) * 2021-12-30 2023-07-07 주식회사 이노제닉스 포스파티딜이노시톨 3-키나아제 촉매 소단위 알파 유전자의 3'비번역 부위에 특이적으로 결합하는 핵산 절편 및 그 응용
CN117402251B (zh) * 2023-12-15 2024-02-23 中国医学科学院基础医学研究所 一种抗小g蛋白rbj的抗体及其应用

Family Cites Families (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
ES2465574T3 (es) 2002-05-03 2014-06-06 Duke University Un método para regular la expresión génica
US8106180B2 (en) * 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
WO2005017145A1 (ja) 2003-08-13 2005-02-24 Japan Biological Informatics Consortium 機能性ｒｎａが制御する被制御遺伝子の同定・予測方法及びその利用方法
US7795419B2 (en) * 2004-05-26 2010-09-14 Rosetta Genomics Ltd. Viral and viral associated miRNAs and uses thereof
EP1838870A2 (en) 2004-12-29 2007-10-03 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAs
JP2006248978A (ja) 2005-03-10 2006-09-21 Mebiopharm Co Ltd 新規なリポソーム製剤
US20090209621A1 (en) 2005-06-03 2009-08-20 The Johns Hopkins University Compositions and methods for decreasing microrna expression for the treatment of neoplasia
ITMI20051248A1 (it) 2005-07-01 2007-01-02 Vimar Spa Modem per bus per impianti elettrici civili ed industriali
CN101296702B (zh) 2005-09-12 2012-11-28 俄亥俄州立大学研究基金会 用于诊断或治疗bcl2相关癌症的组合物和方法
US8603991B2 (en) 2005-11-18 2013-12-10 Gradalis, Inc. Individualized cancer therapy
ES2387250T3 (es) 2005-12-12 2012-09-19 The University Of North Carolina At Chapel Hill Microarn que regulan la proliferación y diferenciación de células musculares
ES2557411T3 (es) 2006-01-05 2016-01-25 The Ohio State University Research Foundation Métodos y composiciones basados en microARN para el diagnóstico y el tratamiento de cánceres sólidos
WO2007143752A2 (en) 2006-06-09 2007-12-13 The Regents Of The University Of California Targets in breast cancer for prognosis or therapy
WO2007149521A2 (en) 2006-06-20 2007-12-27 The Johns Hopkins University Nucleotide motifs providing localization elements and methods of use
WO2008069940A2 (en) 2006-12-06 2008-06-12 Albert Einstein College Of Medicine Of Yeshiva University Multi-microrna methods and compositions
WO2008088858A2 (en) 2007-01-17 2008-07-24 The Johns Hopkins University Compositions and methods featuring micronas for treating neoplasia
WO2009012263A2 (en) * 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
JP5654347B2 (ja) 2007-07-31 2015-01-14 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム ミオシンの発現及び筋線維の同一性を制御するマイクロｒｎａ
WO2009075817A1 (en) 2007-12-06 2009-06-18 Minerva Biotechnologies Corporation Method for treating cancer using interference rna
JP2010094122A (ja) 2008-06-12 2010-04-30 Keio Gijuku バイオマーカーとしてのマイクロｒｎａを用いた頭頸部腫瘍の診断・治療選択
US8729041B2 (en) 2008-12-03 2014-05-20 The Johns Hopkins University Compositions and methods for treating hepatic neoplasia
CN102803284B (zh) 2009-06-08 2015-11-25 米拉根医疗公司 用于miRNA抑制剂和模拟物的化学修饰基序
WO2011034811A1 (en) 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics
US20120302626A1 (en) * 2009-12-04 2012-11-29 Sandeep Dave Microrna and use thereof in identification of b cell malignancies
US8768630B2 (en) * 2010-02-19 2014-07-01 The Regents Of The University Of Michigan miRNA target prediction
AU2011252799B2 (en) 2010-05-14 2015-05-14 Cold Spring Harbor Laboratory Compositions and methods for treating leukemia
DK2426203T3 (en) 2010-09-02 2016-11-07 Université de Mons Agents that are useful in the treatment of muscular dystrophy, facioscapulohumeral
MY165507A (en) 2011-02-03 2018-03-28 Mirna Therapeutics Inc Synthetic mimics of mir-34
CN103459598B (zh) 2011-02-03 2016-08-10 米尔纳医疗股份有限公司 Mir-124的合成模拟物
WO2013016352A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
CA2843636A1 (en) 2011-07-29 2013-02-07 Fred Hutchinson Cancer Research Center Methods and compositions for modulating the innate immune response and/or myogenesis in a mammalian subject
US9428749B2 (en) 2011-10-06 2016-08-30 The Board Of Regents, The University Of Texas System Control of whole body energy homeostasis by microRNA regulation
TWI516267B (zh) 2011-11-07 2016-01-11 臺北榮民總醫院 抑制癌症或腫瘤細胞之類癌幹細胞特性及抗化學及放射治療特性的醫藥組合物
WO2013120038A2 (en) 2012-02-10 2013-08-15 Regents Of The University Of Minnesota Morpholino targeting dux4 for treating fshd
US11293064B2 (en) * 2013-05-02 2022-04-05 Thomas Jefferson University Human miRNAs for use in diagnosis, prognosis, and therapy of human conditions and diseases
US9518300B2 (en) 2014-04-14 2016-12-13 Dana-Farber Cancer Institute, Inc. Composition and method for treating a hematological malignancy
JP6514717B2 (ja) 2014-04-25 2019-05-15 ザ チルドレンズ メディカル センター コーポレーション 異常ヘモグロビン症を治療するための組成物および方法
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
US20180305689A1 (en) 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
WO2017007886A2 (en) 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof
CN117757794A (zh) * 2016-03-07 2024-03-26 美国政府(由卫生和人类服务部的部长所代表) 微小rna及其使用方法
US11518995B2 (en) 2017-09-19 2022-12-06 Children's National Medical Center Gapmers and methods of using the same for the treatment of muscular dystrophy
EP3668997A4 (en) * 2017-10-02 2021-05-19 Research Institute at Nationwide Children's Hospital MIARN SCREENING SYSTEM FOR FABRIC SPECIFIC INTERFERENCE
US10676744B2 (en) * 2017-10-13 2020-06-09 University Of South Carolina Modulation of adipose tissue macrophage phenotype by use of MicroRNA-30
US11268305B2 (en) 2018-05-17 2022-03-08 Kiekert Ag Latching system for a motor vehicle
MX2021001284A (es) 2018-08-02 2021-07-15 Dyne Therapeutics Inc Complejos dirigidos al musculo y sus usos para el tratamiento de distrofia muscular facioescapulohumeral.
CN113710283A (zh) 2019-03-29 2021-11-26 田边三菱制药株式会社 用于调节dux4的表达的化合物、方法和医药组合物
TW202117015A (zh) 2019-07-12 2021-05-01 日商第一三共股份有限公司 使DUX4 pre-mRNA之剪接變化之反義寡核苷酸
BR112022013603A2 (pt) 2020-01-10 2022-09-13 Dyne Therapeutics Inc Complexos de direcionamento a músculo e usos dos mesmos para tratar distrofia muscular facioescapuloumeral
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
BR112022020945A2 (pt) 2020-04-17 2022-12-27 Nevada Res & Innovation Corporation Inibição por crispr para distrofia muscular fácioescápulo- umeral
GB202009618D0 (en) 2020-06-24 2020-08-05 Ucl Business Ltd Therapeutics for the treatment of FSHD
US20230287108A1 (en) 2020-07-23 2023-09-14 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
CA3186746A1 (en) 2020-07-23 2022-01-27 Romesh R. Subramanian Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2022043566A1 (en) 2020-08-31 2022-03-03 Merck Patent Gmbh Method and device for manufacturing of solid oral dosage forms
WO2022051332A1 (en) 2020-09-01 2022-03-10 Ultragenyx Pharmaceutical Inc. Dux4 inhibitors and methods of use thereof
MX2023002853A (es) 2020-09-11 2023-03-31 Arrowhead Pharmaceuticals Inc Agentes de arni para inhibir la expresion de dux4, composiciones de dichos agentes, y metodos de uso.
CA3189071A1 (en) 2020-09-11 2022-03-17 Xiaokai Li Skeletal muscle delivery platforms and methods of use
JP2022055361A (ja) 2020-09-28 2022-04-07 田辺三菱製薬株式会社 Ｄｕｘ４の発現を調節するための医薬組成物

Also Published As

Publication number Publication date
US20220213481A1 (en) 2022-07-07
US20220213480A1 (en) 2022-07-07
BR112022019889A2 (pt) 2023-03-07
EP4127175A2 (en) 2023-02-08
IL296957A (en) 2022-12-01
CO2022015141A2 (es) 2022-12-30
AU2021246530A1 (en) 2022-11-03
US20230132602A1 (en) 2023-05-04
CN116096890A (zh) 2023-05-09
WO2021203043A3 (en) 2021-11-11
US20230295626A1 (en) 2023-09-21
KR20230023612A (ko) 2023-02-17
US11795459B2 (en) 2023-10-24
WO2021203043A2 (en) 2021-10-07
US11597930B2 (en) 2023-03-07
JP2023521042A (ja) 2023-05-23
CA3174286A1 (en) 2021-10-07

Similar Documents

Publication Publication Date Title
MX2022012265A (es) Inhibicion dirigida utilizando oligonucleotidos ingenieria.
NZ733882A (en) Compositions for modulating c9orf72 expression
MX2021012126A (es) Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central.
MX2019002004A (es) Metodos para editar la metilacion del adn.
MX2018011243A (es) Metodos para promover la salud de las plantas usando enzimas libres y microorganismos que sobreexpresan enzimas.
MX2022004101A (es) Composiciones de oligonucleotidos y metodos de uso de las mismas.
EP2032686A4 (en) DEVICES AND SYSTEMS FOR THE CREATION OF ORDINATED DNA AMASES
MX2018008266A (es) Dinucleotidos ciclicos para tratar condiciones asociadas con actividad del estimulador de genes del interferon (sting) tales como cancer.
MY151551A (en) Oxadiazole- and oxazole-substituted benzimidazole- and indole-derivatives as dgat1 inhibitors
JO2872B1 (en) New vehicles
BR112021024463A2 (pt) Oligonucleotídeos e métodos de uso para o tratamento de doenças neurológicas
MX2015015564A (es) Moduladores de oligonucleotido de linfoma linfocitico cronico/linfoma de celulas b 11a (bcl11a) y sus usos.
MX2021014206A (es) Oligonucleotidos gapmer modificados y metodos de uso.
MX2017009427A (es) Inhibidores de mir-92 y usos de los mismos.
GB201202561D0 (en) Treatment of skin disorders
MX2020006297A (es) Variantes de cd19.
JOP20200044A1 (ar) مثبط انتقائي egfr طافر exon 18 و/أو exon 21
PH12021550736A1 (en) Double-stranded ribonucleic acid inhibiting expression of complement c5
MX2019007947A (es) Hoja de acero para estampacion en caliente.
SA517390005B1 (ar) حفاز لهدرجة هيدروكربونات أروماتية، وطريقة معالجة بالهدرجة واستخداماتها فيها
SG11202111575WA (en) Engineered microorganisms and methods for improved aldehyde dehydrogenase activity
MX2018001126A (es) Oligonucleotidos terapeuticos.
MX2017001927A (es) Plantas de tomate que permiten el establecimiento de los acaros.
GB202305377D0 (en) Systems and methods for targeted neuromodulation
MX2017014855A (es) Oligonucleotidos atisentido para tratar epidermolisis bullosa distrofica.