MX2019015143A - Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso. - Google Patents

Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso.

Info

Publication number
MX2019015143A
MX2019015143A MX2019015143A MX2019015143A MX2019015143A MX 2019015143 A MX2019015143 A MX 2019015143A MX 2019015143 A MX2019015143 A MX 2019015143A MX 2019015143 A MX2019015143 A MX 2019015143A MX 2019015143 A MX2019015143 A MX 2019015143A
Authority
MX
Mexico
Prior art keywords
modified guide
guide rnas
methods
ribonucleotprotein
crispr
Prior art date
Application number
MX2019015143A
Other languages
English (en)
Inventor
Jared Matthew Carlson-Stevermer
Krishanu Saha
Amr Ashraf Adbeen
Lucille Katherine Kohlenberg
Original Assignee
Wisconsin Alumni Res Found
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 Wisconsin Alumni Res Found filed Critical Wisconsin Alumni Res Found
Publication of MX2019015143A publication Critical patent/MX2019015143A/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
    • C12N15/111—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
    • 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 RNAses, DNAses
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing 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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N2310/16—Aptamers
    • 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 [CRISPRs]
    • 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
    • C12N2310/30—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

En este documento se describen RNA guías modificados, tales como un RNA guía individual que incluye, de 5' a 3', una secuencia protoespaciadora de hebra sencilla, una primera hebra complementaria de una región de unión para el polipéptido de Cas9, un aptámero que se une a una molécula de unión a biotina, y una segunda hebra complementaria de la región de unión para el polipéptido de Cas9. También se describe un complejo de RNP que incluye el RNA guía modificado y un polipéptido de Cas9 o fragmento activo del mismo. También se incluyen métodos de modificación de genes objetivo en las células al utilizar los RNA guías modificados.
MX2019015143A 2017-06-14 2018-06-14 Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso. MX2019015143A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762519317P 2017-06-14 2017-06-14
PCT/US2018/037531 WO2018232114A1 (en) 2017-06-14 2018-06-14 Modified guide rnas, crispr-ribonucleotprotein complexes and methods of use

Publications (1)

Publication Number Publication Date
MX2019015143A true MX2019015143A (es) 2020-07-27

Family

ID=62842243

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019015143A MX2019015143A (es) 2017-06-14 2018-06-14 Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso.

Country Status (9)

Country Link
US (2) US10907150B2 (es)
EP (1) EP3638792A1 (es)
KR (1) KR102634727B1 (es)
CN (1) CN110753757B (es)
AU (1) AU2018283155A1 (es)
CA (1) CA3065326A1 (es)
IL (1) IL270785A (es)
MX (1) MX2019015143A (es)
WO (1) WO2018232114A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020530992A (ja) * 2017-07-31 2020-11-05 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ． ＬＬＣ Ｃｒｉｓｐｒ／ｃａｓアクチベーターシステムのための合成ガイドｒｎａ
US11739320B2 (en) 2018-11-05 2023-08-29 Wisconsin Alumni Research Foundation Gene correction of Pompe disease and other autosomal recessive disorders via RNA-guided nucleases
EP4095246A1 (en) * 2021-05-27 2022-11-30 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Novel crispr grnas
WO2023224352A1 (ko) * 2022-05-16 2023-11-23 주식회사 엔이에스바이오테크놀러지 나노 입자-crispr 결합체를 기반으로 하는 유전자 조작 및 이의 제조 방법

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10022457B2 (en) * 2005-08-05 2018-07-17 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
CN102858985A (zh) * 2009-07-24 2013-01-02 西格马-奥尔德里奇有限责任公司 基因组编辑方法
EP3808844A1 (en) * 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
ES2786193T3 (es) 2012-12-12 2020-10-09 Broad Inst Inc Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
CN113563476A (zh) 2013-03-15 2021-10-29 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US11414695B2 (en) * 2013-05-29 2022-08-16 Agilent Technologies, Inc. Nucleic acid enrichment using Cas9
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
CN106604994B (zh) * 2014-06-23 2021-12-14 通用医疗公司 通过测序评估的DSBs的全基因组无偏鉴定(GUIDE-Seq)
EP3172321B2 (en) * 2014-07-21 2023-01-04 Illumina, Inc. Polynucleotide enrichment using crispr-cas systems
EP3633032A3 (en) 2014-08-28 2020-07-29 North Carolina State University Novel cas9 proteins and guiding features for dna targeting and genome editing
WO2016065364A1 (en) * 2014-10-24 2016-04-28 Life Technologies Corporation Compositions and methods for enhancing homologous recombination
BR112017016080B1 (pt) 2015-01-28 2024-02-20 Caribou Biosciences, Inc Polinucleotídeo crispr da classe 2 único, sistema crispr da classe 2 e método in vitro de modificação de uma molécula de ácido nucleico alvo em um organismo não-humano, ou em uma célula
WO2016183402A2 (en) 2015-05-13 2016-11-17 President And Fellows Of Harvard College Methods of making and using guide rna for use with cas9 systems
CN108138176B (zh) * 2015-08-19 2022-05-24 阿克生物公司 使用基于核酸引导的核酸酶的系统捕获核酸
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
IL260532B2 (en) 2016-01-11 2023-12-01 Univ Leland Stanford Junior Systems containing chaperone proteins and their uses for controlling gene expression
WO2019046211A1 (en) * 2017-08-28 2019-03-07 Wisconsin Alumni Research Foundation NANOCAPSULES FOR THE ADMINISTRATION OF RIBONUCLEOPROTEINS
US11739320B2 (en) 2018-11-05 2023-08-29 Wisconsin Alumni Research Foundation Gene correction of Pompe disease and other autosomal recessive disorders via RNA-guided nucleases

Also Published As

Publication number Publication date
US20210139891A1 (en) 2021-05-13
IL270785A (en) 2020-01-30
CA3065326A1 (en) 2018-12-20
KR102634727B1 (ko) 2024-02-07
EP3638792A1 (en) 2020-04-22
WO2018232114A1 (en) 2018-12-20
KR20200017485A (ko) 2020-02-18
US10907150B2 (en) 2021-02-02
US20180362971A1 (en) 2018-12-20
CN110753757B (zh) 2024-02-20
AU2018283155A1 (en) 2019-12-19
CN110753757A (zh) 2020-02-04

Similar Documents

Publication Publication Date Title
MX2020004578A (es) Composiciones de casz y metodos de uso.
MX2019015143A (es) Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso.
WO2019126762A3 (en) Cas12a systems, methods, and compositions for targeted rna base editing
GB2569733A (en) RNA-guided nucleic acid modifying enzymes and methods of use thereof
ZA202002094B (en) Trispecific proteins and methods of use
UA118014C2 (uk) Спосіб модифікації днк-мішені
WO2015200555A3 (en) Rna modification to engineer cas9 activity
EP4241784A3 (en) Artificial nucleic acid molecules for improved protein expression
EP4286012A3 (en) Compounds and compositions for intracellular delivery of therapeutic agents
WO2020198509A3 (en) Modified oligonucleotides with increased stability
NZ742040A (en) Engineered nucleic-acid targeting nucleic acids
WO2017156311A3 (en) Combination vectors and methods for treating cancer
SG10201805815YA (en) Rna-guided gene drives
MX2017006624A (es) Anticuerpos contra cd73 y sus usos.
WO2015051199A3 (en) Modified pseudomonas exotoxin a
PH12019500626A1 (en) Aav treatment of huntington's disease
NZ739133A (en) Cellular targeted active ingredient delivery system
MX2022006948A (es) Composiciones y metodos para programar celulas terapeuticas utilizando nanoportadores de acidos nucleicos dirigidos.
JP2013523181A5 (es)
CR20220317A (es) Anticuerpos anti-cd73 y usos de estos
EA201792103A1 (ru) Композиции и способы для ингибирования экспрессии генов hif2альфа
MX2020000154A (es) Composiciones y métodos para inhibir la expresión de hmgb1.
RU2022103641A (ru) Искусственная модификация генома для регуляции экспрессии гена
WO2016164743A3 (en) Cd38 ligand-drug conjugates for targeted cancer therapy
PH12021550256A1 (en) Novel crispr-associated protein and use thereof