CL2019000025A1 - Composiciones y métodos que comprenden mejoras de los arn guías de crispr usando el promotor h1. - Google Patents

Composiciones y métodos que comprenden mejoras de los arn guías de crispr usando el promotor h1.

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Publication number
CL2019000025A1
CL2019000025A1 CL2019000025A CL2019000025A CL2019000025A1 CL 2019000025 A1 CL2019000025 A1 CL 2019000025A1 CL 2019000025 A CL2019000025 A CL 2019000025A CL 2019000025 A CL2019000025 A CL 2019000025A CL 2019000025 A1 CL2019000025 A1 CL 2019000025A1
Authority
CL
Chile
Prior art keywords
crispr
promoter
pol
cas9
addition
Prior art date
Application number
CL2019000025A
Other languages
English (en)
Inventor
Donald Zack
Vinod Jaskula-Ranga
Derek S Welsbie
Original Assignee
Univ Johns Hopkins
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Publication of CL2019000025A1 publication Critical patent/CL2019000025A1/es

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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0014—Skin, i.e. galenical aspects of topical compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • 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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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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
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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
    • C12N15/11—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
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    • 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
    • C12N15/11—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
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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
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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
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    • 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
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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)
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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 [CRISPRs]
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10344—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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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

Abstract

LA MATERIA AQUÍ DESCRITA PROPORCIONA COMPOSICIONES Y MÉTODOS QUE COMPRENDEN MEJORAS DE UN SISTEMA CRISPR (POR EJEMPLO, CRISPR-PROTEÍNA ASOCIADA (CAS) 9 (SISTEMA CRISPR-CAS9, SISTEMAS CRISPR SIN CAS9). TALES COMPOSICIONES PUEDEN COMPRENDER MODIFICACIONES EN LA REGIÓN PROMOTORA H1, ADEMÁS DE MODIFICACIONES EN UTR 5´, DIFERENTES SECUENCIAS ORTÓLOGAS DEL PROMOTOR H1, NUEVAS SECUENCIAS DEL PROMOTOR BIDIRECCIONAL COMPACTO CON ACTIVIDAD TANTO POL II COMO POL III, ADICIÓN DE SECUENCIAS DE CONSENSO DE KOZAK, SECUENCIAS DE TERMINACIÓN, ADICIÓN DE LA EXPRESIÓN CONDICIONAL DEL PROMOTOR BIDIRECCIONAL POL II/POL III, ADICIÓN DE UNA SECUENCIA MOLDE DONANTE PARA CORREGIR MUTACIONES, O COMBINACIONES DE LAS MISMAS. OTROS ASPECTOS DE LA INVENCIÓN SE RELACIONAN CON MODIFICACIONES DE CAS9 A TRAVÉS DE FUSIONES DE REGULACIÓN POSTRANSCRIPCIONAL DEL CICLO CELULAR, SITIOS DIANA PARCIALES DISEÑADOS POR INGENIERÍA GENÉTICA DE MANERA TAL QUE LA NUCLEASA PUEDE UNIRSE A ELLOS SIN ESCINDIR EL ADN, SITIOS DE AUTORREGULACIÓN, Y MODIFICACIONES N-TERMINALES PARA MODULAR LA VIDA MEDIA.
CL2019000025A 2016-07-05 2019-01-04 Composiciones y métodos que comprenden mejoras de los arn guías de crispr usando el promotor h1. CL2019000025A1 (es)

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US201662358335P 2016-07-05 2016-07-05

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CL2019000025A1 true CL2019000025A1 (es) 2019-06-21

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US (1) US11766488B2 (es)
EP (1) EP3481956A4 (es)
JP (2) JP2019520078A (es)
KR (1) KR20190039702A (es)
CN (1) CN109844116A (es)
AU (2) AU2017292667A1 (es)
BR (1) BR112019000055A2 (es)
CA (1) CA3029860A1 (es)
CL (1) CL2019000025A1 (es)
EA (1) EA201990213A1 (es)
IL (1) IL264020A (es)
MX (1) MX2019000251A (es)
SG (1) SG11201900038QA (es)
WO (1) WO2018009534A1 (es)

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EP3481956A4 (en) 2020-07-08
CN109844116A (zh) 2019-06-04
IL264020A (en) 2019-02-03
AU2017292667A1 (en) 2019-02-21
US20190314521A1 (en) 2019-10-17
CA3029860A1 (en) 2018-01-11
SG11201900038QA (en) 2019-02-27
AU2023266246A1 (en) 2023-12-07
EA201990213A1 (ru) 2019-11-29
KR20190039702A (ko) 2019-04-15
MX2019000251A (es) 2019-10-09
US11766488B2 (en) 2023-09-26
WO2018009534A1 (en) 2018-01-11
JP2019520078A (ja) 2019-07-18
BR112019000055A2 (pt) 2019-04-02
JP2022153576A (ja) 2022-10-12
EP3481956A1 (en) 2019-05-15

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