IL312762A - ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR) - Google Patents

ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR)

Info

Publication number
IL312762A
IL312762A IL312762A IL31276224A IL312762A IL 312762 A IL312762 A IL 312762A IL 312762 A IL312762 A IL 312762A IL 31276224 A IL31276224 A IL 31276224A IL 312762 A IL312762 A IL 312762A
Authority
IL
Israel
Prior art keywords
rna
adar
aso
efficient
antisense oligonucleotides
Prior art date
Application number
IL312762A
Other languages
English (en)
Hebrew (he)
Inventor
Ngadhnjim Latifi
Laura Sophia Pfeiffer
Thorsten Stafforst
Original Assignee
Eberhard Karls Universit?T T?Bingen
Ngadhnjim Latifi
Laura Sophia Pfeiffer
Thorsten Stafforst
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 Eberhard Karls Universit?T T?Bingen, Ngadhnjim Latifi, Laura Sophia Pfeiffer, Thorsten Stafforst filed Critical Eberhard Karls Universit?T T?Bingen
Publication of IL312762A publication Critical patent/IL312762A/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL312762A 2021-11-30 2022-11-29 ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR) IL312762A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP21211372 2021-11-30
PCT/EP2022/083711 WO2023099494A1 (en) 2021-11-30 2022-11-29 Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar)

Publications (1)

Publication Number Publication Date
IL312762A true IL312762A (en) 2024-07-01

Family

ID=78820190

Family Applications (1)

Application Number Title Priority Date Filing Date
IL312762A IL312762A (en) 2021-11-30 2022-11-29 ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR)

Country Status (9)

Country Link
US (1) US20230235329A1 (https=)
EP (1) EP4441216A1 (https=)
JP (1) JP2024544067A (https=)
KR (1) KR20240111752A (https=)
CN (1) CN118355119A (https=)
AU (1) AU2022399905A1 (https=)
CA (1) CA3238033A1 (https=)
IL (1) IL312762A (https=)
WO (1) WO2023099494A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024114908A1 (en) * 2022-11-30 2024-06-06 Eberhard Karls Universität Tübingen Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
AU2024325709A1 (en) * 2023-08-15 2026-01-29 Airna Corporation Chemically modified antisense oligonucleotides (asos) and compositions for rna editing
WO2025073902A1 (en) * 2023-10-06 2025-04-10 Eberhard Karls Universität Tübingen Chemical and sequence optimisation of antisense oligonucleotides for adar-mediated rna editing
WO2025172372A1 (en) 2024-02-12 2025-08-21 Airna Corporation Conjugate for targeted delivery of an antisense oligonucleotide
WO2025242018A1 (zh) * 2024-05-20 2025-11-27 时夕(广州)生物科技有限公司 一种经修饰的向导agRNA

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
WO2020001793A1 (en) 2018-06-29 2020-01-02 Eberhard-Karls-Universität Tübingen Artificial nucleic acids for rna editing
CA3158528A1 (en) * 2019-12-23 2021-07-01 Jim SWILDENS Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease

Also Published As

Publication number Publication date
JP2024544067A (ja) 2024-11-27
US20230235329A1 (en) 2023-07-27
WO2023099494A1 (en) 2023-06-08
CA3238033A1 (en) 2023-06-08
AU2022399905A1 (en) 2024-05-23
WO2023099494A9 (en) 2024-10-17
EP4441216A1 (en) 2024-10-09
KR20240111752A (ko) 2024-07-17
CN118355119A (zh) 2024-07-16

Similar Documents

Publication Publication Date Title
IL312762A (en) ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR)
PH12022550059A1 (en) Targeted rna editing by leveraging endogenous adar using engineered rnas
PH12021552613A1 (en) METHODS AND COMPOSITIONS FOR EDITING RNAs
PH12021550805A1 (en) Methods and Compositions for Editing RNAs
Zhang et al. Life without A tail: new formats of long noncoding RNAs
GB202011428D0 (en) Antisense oligonucleotides for RNA editing
MX2013009815A (es) Derivados de fosforoamidato de 5-fluoro-2'-desoxiuridina para uso en el tratamiento contra el cancer.
GB201901873D0 (en) Antisense oligonucleotides for nucleic acid editing
BRPI0418463A (pt) métodos de inibir expressão de stat3 humano, de induzir apoptose em células de carcinoma do pulmão, e de inibir crescimento de tumor de linfoma de células t, tumor alcl, e tumor mieloma múltiplo em um animal
BRPI0922355A2 (pt) ácidos nucléicos, métodos para reduzir a expressão de um gene em uma célula in vitro, uso de um ácido nucléico, complexo de rna e uso do complexo de rna
Yang et al. MicroRNAs and apoptosis: implications in the molecular therapy of human disease
RU2019124314A (ru) Сопряженные антисмысловые соединения и их применение
WO2003100017A3 (en) Oligonucleotides having modified nucleoside units
GB202304363D0 (en) Chemically modified antisense oligonucleotides for use in RNA editing
EP4277990A4 (en) TARGETED RNA EDITING BY USING ENDOGENOUS ADAR USING MANIPULATED RNAS
ATE442986T1 (de) Systeme zur nutzlastverkleidung bei luftfahrzeugen und entsprechende verfahren
EP3972571A4 (en) METHOD USING PHOTOTHERMAL NANOPARTICLES IN RAPID NUCLEIC ACID AMPLIFICATION AND PHOTOTHERMAL NANOPARTICLES
HK40117606A (en) Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar)
HK40109733A (en) Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar)
Lee et al. Selection of reference genes for quantitative real-time polymerase chain reaction in porcine embryos
Ge et al. Regulation of pre-mRNA splicing in Xenopus oocytes by targeted 2′-O-methylation
IL320984A (en) Chemically modified antisense oligonucleotides (ASOS) and compositions comprising them for RNA editing
BR112021020406A2 (pt) Sistemas de edição de genes para modificar um gene scn9a ou scn10a e métodos de uso dos mesmos
WO2004022782A3 (en) Compositions and methods for tissue specific or inducible inhibition of gene expression
Feng et al. A directed Monte Carlo solution of linear stochastic algebraic system of equations