CA3125299A1 - Systems and methods for modulating rna - Google Patents

Systems and methods for modulating rna Download PDF

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Publication number
CA3125299A1
CA3125299A1 CA3125299A CA3125299A CA3125299A1 CA 3125299 A1 CA3125299 A1 CA 3125299A1 CA 3125299 A CA3125299 A CA 3125299A CA 3125299 A CA3125299 A CA 3125299A CA 3125299 A1 CA3125299 A1 CA 3125299A1
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Prior art keywords
rna
domain
acc
source
seq
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CA3125299A
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English (en)
French (fr)
Inventor
Bryan C. Dickinson
Simone RAUCH
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University of Chicago
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University of Chicago
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C12N15/62—DNA sequences coding for fusion 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/63—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/35—Fusion polypeptide containing a fusion for enhanced stability/folding during expression, e.g. fusions with chaperones or thioredoxin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/50—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
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Epidemiology (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)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
CA3125299A 2019-01-04 2020-01-03 Systems and methods for modulating rna Pending CA3125299A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962788571P 2019-01-04 2019-01-04
US62/788,571 2019-01-04
US201962831342P 2019-04-09 2019-04-09
US62/831,342 2019-04-09
US201962903080P 2019-09-20 2019-09-20
US62/903,080 2019-09-20
US201962929339P 2019-11-01 2019-11-01
US62/929,339 2019-11-01
PCT/US2020/012169 WO2020142676A1 (en) 2019-01-04 2020-01-03 Systems and methods for modulating rna

Publications (1)

Publication Number Publication Date
CA3125299A1 true CA3125299A1 (en) 2020-07-09

Family

ID=71407144

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3125299A Pending CA3125299A1 (en) 2019-01-04 2020-01-03 Systems and methods for modulating rna

Country Status (12)

Country Link
US (1) US20220048962A1 (https=)
EP (1) EP3906042A4 (https=)
JP (2) JP2022516919A (https=)
KR (1) KR20210124238A (https=)
CN (1) CN113543797A (https=)
AU (2) AU2020204917A1 (https=)
BR (1) BR112021013173A2 (https=)
CA (1) CA3125299A1 (https=)
IL (1) IL284516A (https=)
MX (1) MX2021008153A (https=)
SG (1) SG11202107209WA (https=)
WO (1) WO2020142676A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114685685A (zh) * 2022-04-20 2022-07-01 上海科技大学 编辑rna的融合蛋白及其应用

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210095296A1 (en) * 2019-09-29 2021-04-01 Technion Research & Development Foundation Limited Synthetic non-coding rnas
EP4110918A4 (en) * 2020-02-28 2024-04-03 The University of Chicago METHODS AND COMPOSITIONS COMPRISING TRANS-ACTING TRANSLATION ACTIVATORS
CN113583136A (zh) * 2021-04-25 2021-11-02 南方科技大学 一种植物激素脱落酸荧光分子探针及其用途
WO2022251264A1 (en) * 2021-05-24 2022-12-01 Donald Danforth Plant Science Center Compositions for rna-protein tethering and methods of using
JP2023040984A (ja) * 2021-09-10 2023-03-23 国立大学法人信州大学 Ｒｎａの編集方法及びｒｎａを編集するためのキット
KR20240082405A (ko) * 2021-10-08 2024-06-10 펜슬 바이오사이언시스 리미티드 합성 게놈 편집 시스템
CN114085837B (zh) * 2021-11-19 2024-04-12 中山大学 一种敲除基因ythdf1的细胞系及其构建方法
WO2023150131A1 (en) * 2022-02-01 2023-08-10 The Regents Of The University Of California Method of regulating alternative polyadenylation in rna
US20250154508A1 (en) * 2022-02-25 2025-05-15 The University Of Chicago Methods and compositions for activating translation
US20250283062A1 (en) * 2022-05-02 2025-09-11 The University Of Chicago Systems and methods for modulating rna
EP4689118A1 (en) * 2023-03-31 2026-02-11 Duke University Compositions for and methods of engineering the transcriptome
GB202401827D0 (en) * 2024-02-09 2024-03-27 Ucl Business Ltd Therapy for dravet syndrome
CN119899820B (zh) * 2025-01-23 2026-04-03 深圳开悦生命科技有限公司 Dhx8融合蛋白及其制备方法

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US20040077082A1 (en) * 2002-10-18 2004-04-22 Koehn Richard K. RNA-based inhibitory oligonucleotides
PL3360964T3 (pl) * 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
EP4286517A3 (en) * 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
EP3341410B1 (en) * 2015-08-24 2021-06-02 Cellectis Chimeric antigen receptors with integrated controllable functions
US20200291369A1 (en) * 2016-03-11 2020-09-17 Erasmus University Medical Center Rotterdam Improved CRISPR-Cas9 Genome Editing Tool
CA3025523A1 (en) * 2016-05-27 2017-11-30 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of genome-editing molecules
CN110612353A (zh) * 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
AU2018251801B2 (en) * 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
CN108559738A (zh) * 2018-02-12 2018-09-21 南昌大学 一种植物rna修饰和编辑的系统及方法

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114685685A (zh) * 2022-04-20 2022-07-01 上海科技大学 编辑rna的融合蛋白及其应用
CN114685685B (zh) * 2022-04-20 2023-11-07 上海科技大学 编辑rna的融合蛋白及其应用

Also Published As

Publication number Publication date
SG11202107209WA (en) 2021-07-29
JP2025084877A (ja) 2025-06-03
KR20210124238A (ko) 2021-10-14
WO2020142676A1 (en) 2020-07-09
JP2022516919A (ja) 2022-03-03
EP3906042A4 (en) 2023-03-15
EP3906042A1 (en) 2021-11-10
IL284516A (en) 2021-08-31
CN113543797A (zh) 2021-10-22
MX2021008153A (es) 2021-08-11
US20220048962A1 (en) 2022-02-17
AU2026200853A1 (en) 2026-04-02
BR112021013173A2 (pt) 2021-09-28
AU2020204917A1 (en) 2021-08-19

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