MX2021012989A - Metodos y composiciones para modular el empalme y la traduccion. - Google Patents

Metodos y composiciones para modular el empalme y la traduccion.

Info

Publication number
MX2021012989A
MX2021012989A MX2021012989A MX2021012989A MX2021012989A MX 2021012989 A MX2021012989 A MX 2021012989A MX 2021012989 A MX2021012989 A MX 2021012989A MX 2021012989 A MX2021012989 A MX 2021012989A MX 2021012989 A MX2021012989 A MX 2021012989A
Authority
MX
Mexico
Prior art keywords
compositions
methods
modulating
mrna
translation
Prior art date
Application number
MX2021012989A
Other languages
English (en)
Inventor
Isabel Aznarez
Juergen Scharner
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 MX2021012989A publication Critical patent/MX2021012989A/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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Abstract

Los eventos de empalme alternativos en genes pueden conducir a transcripciones de ARNm no productivas o menos productivas y agentes terapéuticos que pueden dirigir los eventos de empalme alternativo en los genes pueden modular el nivel de expresión de las proteínas funcionales en pacientes y/o inhibir la expresión aberrante de proteínas. En el presente documento, se proporcionan composiciones y métodos para modular el nivel de expresión de una secuencia de péptidos diana mediante la modulación de los empalmes de un pre-ARNm. También se proporcionan en la presente composiciones y métodos para tratar una enfermedad o condición causada por una cantidad o actividad deficientes de una proteína diana funcional mediante la modulación de los empalmes de un pre-ARNm.
MX2021012989A 2019-04-24 2020-04-24 Metodos y composiciones para modular el empalme y la traduccion. MX2021012989A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962838010P 2019-04-24 2019-04-24
PCT/US2020/029897 WO2020219934A1 (en) 2019-04-24 2020-04-24 Methods and compositions for modulating splicing and translation

Publications (1)

Publication Number Publication Date
MX2021012989A true MX2021012989A (es) 2022-01-24

Family

ID=72941425

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021012989A MX2021012989A (es) 2019-04-24 2020-04-24 Metodos y composiciones para modular el empalme y la traduccion.

Country Status (15)

Country Link
US (1) US20220127612A1 (es)
EP (1) EP3958970A4 (es)
JP (1) JP2022529532A (es)
KR (1) KR20220012230A (es)
CN (1) CN114025848A (es)
AR (1) AR119722A1 (es)
AU (1) AU2020262435A1 (es)
BR (1) BR112021021047A2 (es)
CA (1) CA3134329A1 (es)
EA (1) EA202192755A1 (es)
IL (1) IL287398A (es)
MX (1) MX2021012989A (es)
SG (1) SG11202111597UA (es)
TW (1) TW202106877A (es)
WO (1) WO2020219934A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3873608A1 (en) 2018-10-31 2021-09-08 Gilead Sciences, Inc. Substituted 6-azabenzimidazole compounds having hpk1 inhibitory activity
CA3117556A1 (en) 2018-10-31 2020-05-07 Gilead Sciences, Inc. Substituted 6-azabenzimidazole compounds as hpk1 inhibitors
TWI826690B (zh) 2019-05-23 2023-12-21 美商基利科學股份有限公司 經取代之烯吲哚酮化物及其用途
CA3166000A1 (en) * 2020-01-28 2021-08-05 Stephen Wilton Antisense oligomers and methods for treating parkin-related pathologies
CN112553328B (zh) * 2020-12-30 2022-06-17 浙江大学 检测基因表达水平的产品及其在制备重度抑郁症诊断工具中的应用
WO2023239782A2 (en) * 2022-06-07 2023-12-14 Kicho Inc. Agents for modulating expression
WO2024009306A1 (en) * 2022-07-07 2024-01-11 Skip Therapeutics Ltd. Compositions and methods for treating primary ciliary dyskinesia

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE544473T1 (de) * 2000-11-09 2012-02-15 Cold Spring Harbor Lab Chimäre moleküle zur modulation der genexpression
JP2010507387A (ja) * 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のｓｉＲＮＡおよびその使用方法
CA2930859C (en) * 2013-09-04 2022-05-03 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
EP3265098A4 (en) * 2015-03-03 2019-02-13 Ionis Pharmaceuticals, Inc. COMPOSITIONS FOR MODULATING MECP2 EXPRESSION
JP7170656B2 (ja) * 2017-03-24 2022-11-14 ザ ユニバーシティ コート オブ ザ ユニバーシティ オブ エジンバラ Ｍｅｃｐ２ベースの治療

Also Published As

Publication number Publication date
CN114025848A (zh) 2022-02-08
JP2022529532A (ja) 2022-06-22
BR112021021047A2 (pt) 2022-03-22
CA3134329A1 (en) 2020-10-29
AR119722A1 (es) 2022-01-05
TW202106877A (zh) 2021-02-16
WO2020219934A1 (en) 2020-10-29
EP3958970A1 (en) 2022-03-02
EA202192755A1 (ru) 2022-03-23
KR20220012230A (ko) 2022-02-03
AU2020262435A1 (en) 2021-12-02
EP3958970A4 (en) 2023-05-31
US20220127612A1 (en) 2022-04-28
SG11202111597UA (en) 2021-11-29
IL287398A (en) 2021-12-01

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