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MX2021013081A - Métodos y composiciones para modular el corte y empalme de intrones alternativos. - Google Patents

Métodos y composiciones para modular el corte y empalme de intrones alternativos.

Info

Publication number
MX2021013081A
MX2021013081A MX2021013081A MX2021013081A MX2021013081A MX 2021013081 A MX2021013081 A MX 2021013081A MX 2021013081 A MX2021013081 A MX 2021013081A MX 2021013081 A MX2021013081 A MX 2021013081A MX 2021013081 A MX2021013081 A MX 2021013081A
Authority
MX
Mexico
Prior art keywords
compositions
methods
splicing
modulate
introns
Prior art date
Application number
MX2021013081A
Other languages
English (en)
Inventor
Isabel Aznarez
Original Assignee
Stoke 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 Stoke Therapeutics Inc filed Critical Stoke Therapeutics Inc
Publication of MX2021013081A publication Critical patent/MX2021013081A/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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12N15/11—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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • C12N2310/11—Antisense
    • 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]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Ophthalmology & Optometry (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

En el presente documento se proporcionan métodos y composiciones para modular la expresión de una proteína diana o un ARN diana modulando el corte y empalme del pre-ARNm y para tratar enfermedades o afecciones asociadas con la concentración de expresión de la proteína diana o el ARN diana.
MX2021013081A 2019-04-26 2020-04-24 Métodos y composiciones para modular el corte y empalme de intrones alternativos. MX2021013081A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962839572P 2019-04-26 2019-04-26
PCT/US2020/029953 WO2020219977A1 (en) 2019-04-26 2020-04-24 Methods and compositions for modulating splicing of alternative introns

Publications (1)

Publication Number Publication Date
MX2021013081A true MX2021013081A (es) 2021-12-10

Family

ID=72941393

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021013081A MX2021013081A (es) 2019-04-26 2020-04-24 Métodos y composiciones para modular el corte y empalme de intrones alternativos.

Country Status (10)

Country Link
US (1) US20220162599A1 (es)
EP (1) EP3959313A4 (es)
JP (2) JP2022532998A (es)
KR (1) KR20220019676A (es)
CN (1) CN114127286A (es)
AU (1) AU2020263581A1 (es)
BR (1) BR112021021384A2 (es)
CA (1) CA3134947A1 (es)
MX (1) MX2021013081A (es)
WO (1) WO2020219977A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023348346A1 (en) * 2022-09-30 2025-03-27 Contera Pharma A/S Antisense oligonucleotides for the treatment of canavan disease

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1529105B1 (en) * 2002-08-12 2012-10-03 Société de commercialisation des produits de la recherche appliquée SOCPRA Sciences Santé et Humaines s.e.c. Methods to reprogram splice site selection in pre-messenger rnas
JP2013523162A (ja) * 2010-04-06 2013-06-17 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｃｄ２７４／ｐｄ−ｌ１遺伝子の発現を阻害するための組成物および方法
CA2935804A1 (en) * 2014-01-14 2015-07-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of melanoma using pd-l1 isoforms
KR102620328B1 (ko) * 2014-10-03 2024-01-02 콜드스프링하버러보러토리 핵 유전자 산출량의 표적화 증강
WO2017100587A1 (en) * 2015-12-09 2017-06-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting programmed cell death 1 ligand 1 (pd-l1) and methods of use thereof
CA3005256A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
CN108779465B (zh) * 2016-03-14 2022-05-13 豪夫迈·罗氏有限公司 用于减少pd-l1表达的寡核苷酸
SG10201705285SA (en) * 2017-06-27 2019-01-30 Agency Science Tech & Res Antisense oligonucleotides

Also Published As

Publication number Publication date
EP3959313A1 (en) 2022-03-02
AU2020263581A1 (en) 2021-12-16
US20220162599A1 (en) 2022-05-26
JP2022532998A (ja) 2022-07-21
CA3134947A1 (en) 2020-10-29
EP3959313A4 (en) 2023-09-27
CN114127286A (zh) 2022-03-01
KR20220019676A (ko) 2022-02-17
BR112021021384A2 (pt) 2021-12-21
WO2020219977A1 (en) 2020-10-29
JP2025128180A (ja) 2025-09-02

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