MX2023002246A - Sistema sintetico de crispr-cas en miniatura (casmini) para modificacion de genomas de eucariotas. - Google Patents

Sistema sintetico de crispr-cas en miniatura (casmini) para modificacion de genomas de eucariotas.

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Publication number
MX2023002246A
MX2023002246A MX2023002246A MX2023002246A MX2023002246A MX 2023002246 A MX2023002246 A MX 2023002246A MX 2023002246 A MX2023002246 A MX 2023002246A MX 2023002246 A MX2023002246 A MX 2023002246A MX 2023002246 A MX2023002246 A MX 2023002246A
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MX
Mexico
Prior art keywords
cas
rna molecules
casmini
cas proteins
genome engineering
Prior art date
Application number
MX2023002246A
Other languages
English (en)
Inventor
Lei S Qi
Xiaoshu Xu
Original Assignee
Univ Leland Stanford Junior
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Publication date
Application filed by Univ Leland Stanford Junior filed Critical Univ Leland Stanford Junior
Publication of MX2023002246A publication Critical patent/MX2023002246A/es

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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/305Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Micrococcaceae (F)
    • C07K14/31Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Micrococcaceae (F) from Staphylococcus (G)
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Tea And Coffee (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)

Abstract

En la presente se proporcionan proteínas Cas y moléculas de ARN guía modificadas para mostrar una actividad incrementada en células eucariotas. Las proteínas Cas y las moléculas de ARN guía, proporcionadas, son particularmente útiles para aplicaciones donde resulta ventajosa la modulación de ácidos nucleicos eucarióticos con moléculas relativamente pequeñas. También se proporcionan ácidos nucleicos y vectores que codifican para las proteínas Cas y las moléculas de ARN guía, divulgadas, composiciones farmacéuticas que incluyen las proteínas Cas y las moléculas de ARN guía, y métodos para utilizar los materiales divulgados.
MX2023002246A 2020-09-01 2021-08-31 Sistema sintetico de crispr-cas en miniatura (casmini) para modificacion de genomas de eucariotas. MX2023002246A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063073377P 2020-09-01 2020-09-01
US202163191611P 2021-05-21 2021-05-21
PCT/US2021/048362 WO2022051250A1 (en) 2020-09-01 2021-08-31 Synthetic miniature crispr-cas (casmini) system for eukaryotic genome engineering

Publications (1)

Publication Number Publication Date
MX2023002246A true MX2023002246A (es) 2023-05-09

Family

ID=80492161

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023002246A MX2023002246A (es) 2020-09-01 2021-08-31 Sistema sintetico de crispr-cas en miniatura (casmini) para modificacion de genomas de eucariotas.

Country Status (12)

Country Link
US (1) US20230340439A1 (es)
EP (1) EP4208545A1 (es)
JP (1) JP2023538964A (es)
KR (1) KR20230076820A (es)
CN (1) CN116438313A (es)
AU (1) AU2021337539A1 (es)
BR (1) BR112023003784A2 (es)
CA (1) CA3192654A1 (es)
GB (1) GB2615903A (es)
IL (1) IL301017A (es)
MX (1) MX2023002246A (es)
WO (1) WO2022051250A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3223009A1 (en) * 2021-06-17 2022-12-22 Kaiyi JIANG Systems, methods, and compositions comprising miniature crispr nucleases for gene editing and programmable gene activation and inhibition
WO2023168242A1 (en) * 2022-03-01 2023-09-07 Epicrispr Biotechnologies, Inc. Engineered nucleases, compositions, and methods of use thereof
WO2023173110A1 (en) * 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
WO2023240137A1 (en) * 2022-06-08 2023-12-14 The Board Institute, Inc. Evolved cas14a1 variants, compositions, and methods of making and using same in genome editing

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4219699A1 (en) * 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
CN111757889B (zh) * 2018-10-29 2021-05-25 中国农业大学 新型CRISPR/Cas12f酶和系统
US20200199555A1 (en) * 2018-12-05 2020-06-25 The Broad Institute, Inc. Cas proteins with reduced immunogenicity and methods of screening thereof

Also Published As

Publication number Publication date
AU2021337539A1 (en) 2023-05-04
GB2615903A (en) 2023-08-23
IL301017A (en) 2023-05-01
JP2023538964A (ja) 2023-09-12
EP4208545A1 (en) 2023-07-12
CN116438313A (zh) 2023-07-14
BR112023003784A2 (pt) 2023-03-28
KR20230076820A (ko) 2023-05-31
CA3192654A1 (en) 2022-03-10
WO2022051250A1 (en) 2022-03-10
GB202304773D0 (en) 2023-05-17
US20230340439A1 (en) 2023-10-26

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