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MX2021009750A - Moleculas del acido ribonucleico guia de cas12a y usos de las mismas. - Google Patents

Moleculas del acido ribonucleico guia de cas12a y usos de las mismas.

Info

Publication number
MX2021009750A
MX2021009750A MX2021009750A MX2021009750A MX2021009750A MX 2021009750 A MX2021009750 A MX 2021009750A MX 2021009750 A MX2021009750 A MX 2021009750A MX 2021009750 A MX2021009750 A MX 2021009750A MX 2021009750 A MX2021009750 A MX 2021009750A
Authority
MX
Mexico
Prior art keywords
guide rna
rna molecules
cas12a guide
cas12a
grna
Prior art date
Application number
MX2021009750A
Other languages
English (en)
Inventor
Anna Cereseto
Antonio Casini
Gianluca Petris
Giulia Maule
Marianne Carlon
Original Assignee
Univ Degli Studi Di Trento
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 Univ Degli Studi Di Trento filed Critical Univ Degli Studi Di Trento
Publication of MX2021009750A publication Critical patent/MX2021009750A/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
    • C12N15/1138—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 against receptors or cell surface 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
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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)
    • C12N9/16—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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • 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
    • C12N2320/34—Allele or polymorphism specific uses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

Moléculas de ARN guía (ARNg) de Cas12a diseñadas útiles, por ejemplo, para corregir el empalme de ARN aberrante resultante de mutaciones en una secuencia de ADN genómico y para prevenir la inclusión de exones en ARNm maduro.
MX2021009750A 2019-02-12 2020-02-11 Moleculas del acido ribonucleico guia de cas12a y usos de las mismas. MX2021009750A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962804591P 2019-02-12 2019-02-12
PCT/IB2020/051089 WO2020165768A1 (en) 2019-02-12 2020-02-11 Cas12a guide rna molecules and uses thereof

Publications (1)

Publication Number Publication Date
MX2021009750A true MX2021009750A (es) 2021-09-08

Family

ID=69726632

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021009750A MX2021009750A (es) 2019-02-12 2020-02-11 Moleculas del acido ribonucleico guia de cas12a y usos de las mismas.

Country Status (11)

Country Link
US (1) US20220145305A1 (es)
EP (1) EP3924494A1 (es)
JP (1) JP2022520783A (es)
KR (1) KR20210126012A (es)
CN (1) CN113614231A (es)
AU (1) AU2020222078A1 (es)
BR (1) BR112021015564A2 (es)
CA (1) CA3127527A1 (es)
EA (1) EA202192233A1 (es)
MX (1) MX2021009750A (es)
WO (1) WO2020165768A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4158020A4 (en) * 2020-05-29 2024-06-26 Arbor Biotechnologies, Inc. COMPOSITIONS COMPRISING A CAS12I2 POLYPEPTIDE AND THEIR USES
EP4320245A4 (en) * 2021-04-05 2025-08-06 Univ Texas COMPOSITIONS, METHODS AND USES FOR TREATING CYSTIC FIBROSIS AND RELATED DISORDERS
US20230193289A1 (en) * 2021-08-06 2023-06-22 Taipei Veterans General Hospital Compositions and methods for treating fabry disease
WO2023194359A1 (en) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions and methods for treatment of usher syndrome type 2a
WO2024249850A2 (en) * 2023-06-01 2024-12-05 Research Institute At Nationwide Children's Hospital Grna for targeting mutant allele and uses thereof
WO2025007937A1 (zh) * 2023-07-04 2025-01-09 广州瑞风生物科技有限公司 靶向USH2A pre-mRNA假外显子PE40的snRNA及其应用
KR102789465B1 (ko) * 2023-09-20 2025-04-04 주식회사 엔세이지 신규 CRISPR/Cas12a 시스템 및 그 유전자 편집 용도
US12428636B1 (en) 2024-07-08 2025-09-30 Pairwise Plants Services, Inc. Methods and compositions for modification of protospacer adjacent motif specificity of CAS12A

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US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
BRPI0513390A (pt) 2004-07-16 2008-05-06 Us Gov Health & Human Serv vacinas contra aids contendo construções de ácido nucléico cmv/r
AU2008346801A1 (en) 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのｃｒｉｓｐｒ−ｃａｓ系及び組成物の送達、使用及び治療適用
EP3114227B1 (en) * 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
KR102368920B1 (ko) * 2016-04-25 2022-02-28 프로큐알 테라퓨틱스 Ⅱ 비.브이. 안 질환 치료용 올리고뉴클레오타이드
JP7490211B2 (ja) * 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
US11168313B2 (en) 2016-07-26 2021-11-09 The General Hospital Corporation Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
EP3545090A1 (en) * 2016-11-28 2019-10-02 The Board of Regents of The University of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
CA3059793A1 (en) * 2017-05-03 2018-11-08 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene
EP3728594A1 (en) * 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a

Also Published As

Publication number Publication date
KR20210126012A (ko) 2021-10-19
EA202192233A1 (ru) 2021-11-11
CA3127527A1 (en) 2020-08-20
BR112021015564A2 (pt) 2021-11-03
AU2020222078A1 (en) 2021-07-15
US20220145305A1 (en) 2022-05-12
EP3924494A1 (en) 2021-12-22
CN113614231A (zh) 2021-11-05
WO2020165768A1 (en) 2020-08-20
JP2022520783A (ja) 2022-04-01

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