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MX2020004647A - Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna). - Google Patents

Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna).

Info

Publication number
MX2020004647A
MX2020004647A MX2020004647A MX2020004647A MX2020004647A MX 2020004647 A MX2020004647 A MX 2020004647A MX 2020004647 A MX2020004647 A MX 2020004647A MX 2020004647 A MX2020004647 A MX 2020004647A MX 2020004647 A MX2020004647 A MX 2020004647A
Authority
MX
Mexico
Prior art keywords
ace
methods
trna
reassignment
rescuing
Prior art date
Application number
MX2020004647A
Other languages
English (en)
Inventor
David B Weiner
Alfredo Perales Puchalt
John Lueck
Christopher A Ahern
Original Assignee
Wistar Inst
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 Wistar Inst filed Critical Wistar Inst
Publication of MX2020004647A publication Critical patent/MX2020004647A/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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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

Landscapes

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

Abstract

La presente invención provee composiciones y métodos para tratar enfermedades y trastornos asociados con codones de terminación prematura (PTCs), que comprenden la administración de moléculas de ácido nucleico que comprenden o codifican 5 para moléculas de ARNt editado anti-codones (ACE) (ACE-tRNAs); específicamente, los métodos comprenden administrar a un sujeto por lo menos un ACE-tRNA específico para UGA, UAA o UAG para tratar trastornos asociados; se describen además secuencias de ácido nucleico de moléculas de ACE-tRNA.
MX2020004647A 2017-11-02 2018-11-02 Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna). MX2020004647A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762580887P 2017-11-02 2017-11-02
US201862687015P 2018-06-19 2018-06-19
PCT/US2018/059085 WO2019090169A1 (en) 2017-11-02 2018-11-02 Methods of rescuing stop codons via genetic reassignment with ace-trna

Publications (1)

Publication Number Publication Date
MX2020004647A true MX2020004647A (es) 2021-03-25

Family

ID=66332719

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2020004647A MX2020004647A (es) 2017-11-02 2018-11-02 Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna).
MX2020004587A MX2020004587A (es) 2017-11-02 2018-11-02 Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2020004587A MX2020004587A (es) 2017-11-02 2018-11-02 Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt.

Country Status (12)

Country Link
US (4) US20230203482A1 (es)
EP (2) EP3703701A4 (es)
JP (4) JP7474511B2 (es)
KR (2) KR20200090171A (es)
CN (2) CN111712248B (es)
AU (2) AU2018360065A1 (es)
BR (2) BR112020008733A2 (es)
CA (2) CA3081737A1 (es)
IL (2) IL274356A (es)
MX (2) MX2020004647A (es)
RU (2) RU2020117928A (es)
WO (2) WO2019090154A1 (es)

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CN111712248B (zh) 2017-11-02 2024-07-26 衣阿华大学研究基金会 使用ACE-tRNA经由遗传重分配拯救终止密码子的方法
AU2019350869A1 (en) * 2018-09-26 2021-05-20 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
WO2021072201A1 (en) * 2019-10-11 2021-04-15 University Of Massachusetts Raav-mediated in vivo delivery of suppressor trnas
KR20220155981A (ko) * 2019-11-01 2022-11-24 테바드 바이오사이언시즈, 인크. 미성숙 종결 코돈-매개 장애를 치료하기 위한 방법 및 조성물
EP4069841A1 (en) * 2019-12-02 2022-10-12 Shape Therapeutics Inc. Targeted transfer rnas for treatment of diseases
TW202204620A (zh) * 2020-04-14 2022-02-01 美商旗艦先鋒創新有限責任公司 Trem組合物及其用途
WO2021252354A1 (en) 2020-06-12 2021-12-16 University Of Rochester ENCODING AND EXPRESSION OF ACE-tRNAs
KR20230127269A (ko) * 2020-12-31 2023-08-31 유니버시티 오브 아이오와 리써치 파운데이션 센스, 억제자 트랜스퍼 rna 조성물 및 관련된 용도와기능
WO2022182976A1 (en) * 2021-02-25 2022-09-01 Riboz, Llc Engineered expression constructs to increase protein expression from synthetic ribonucleic acid (rna)
KR20240025507A (ko) * 2021-05-05 2024-02-27 테바드 바이오사이언시즈, 인크. 미성숙 종결 코돈-매개 장애를 치료하기 위한 방법 및 조성물
CN113308463A (zh) * 2021-05-12 2021-08-27 中国农业科学院哈尔滨兽医研究所（中国动物卫生与流行病学中心哈尔滨分中心） 经过反密码子编辑的tRNA作为分子开关在精准控制蛋白表达及病毒复制中的应用
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AU2023210521A1 (en) 2022-01-21 2024-08-29 David H. Altreuter Modified transfer rna with improved activity
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TW202405173A (zh) 2022-04-18 2024-02-01 美商維泰克斯製藥公司 用於增強aav療法及降低aav向肝臟之趨性的組合物及方法
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WO2024178172A1 (en) 2023-02-23 2024-08-29 University Of Rochester Agents and methods for making closed-end dna thread molecules
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Also Published As

Publication number Publication date
CN111712248A (zh) 2020-09-25
EP3703762A1 (en) 2020-09-09
IL274356A (en) 2020-06-30
JP2024054361A (ja) 2024-04-16
EP3703701A4 (en) 2022-02-09
JP2021502078A (ja) 2021-01-28
BR112020008733A2 (pt) 2020-11-03
US20200291401A1 (en) 2020-09-17
US20230407300A1 (en) 2023-12-21
JP7474511B2 (ja) 2024-04-25
RU2020117928A3 (es) 2022-05-06
AU2018360055A1 (en) 2020-06-04
RU2020117928A (ru) 2021-12-03
WO2019090154A1 (en) 2019-05-09
JP2021508234A (ja) 2021-03-04
KR20200090171A (ko) 2020-07-28
AU2018360065A1 (en) 2020-06-18
US20240182891A1 (en) 2024-06-06
EP3703701A1 (en) 2020-09-09
KR20200083550A (ko) 2020-07-08
US11661600B2 (en) 2023-05-30
US20230203482A1 (en) 2023-06-29
JP2023106545A (ja) 2023-08-01
BR112020008725A2 (pt) 2021-03-30
IL274396A (en) 2020-06-30
JP7474532B2 (ja) 2024-04-25
WO2019090169A1 (en) 2019-05-09
EP3703762A4 (en) 2021-08-18
MX2020004587A (es) 2021-04-12
CA3081692A1 (en) 2019-05-09
CN111712248B (zh) 2024-07-26
CA3081737A1 (en) 2019-05-09
RU2020117787A (ru) 2021-12-02
CN111818945A (zh) 2020-10-23

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