SG11201900038QA - Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter - Google Patents

Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter

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Publication number
SG11201900038QA
SG11201900038QA SG11201900038QA SG11201900038QA SG11201900038QA SG 11201900038Q A SG11201900038Q A SG 11201900038QA SG 11201900038Q A SG11201900038Q A SG 11201900038QA SG 11201900038Q A SG11201900038Q A SG 11201900038QA SG 11201900038Q A SG11201900038Q A SG 11201900038QA
Authority
SG
Singapore
Prior art keywords
international
promoter
crispr
baltimore
compositions
Prior art date
Application number
SG11201900038QA
Other languages
English (en)
Inventor
Vinod Jaskula-Ranga
Donald Zack
Derek Welsbie
Original Assignee
Univ Johns Hopkins
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 Johns Hopkins filed Critical Univ Johns Hopkins
Publication of SG11201900038QA publication Critical patent/SG11201900038QA/en

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0014Skin, i.e. galenical aspects of topical compositions
    • 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)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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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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    • 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
    • C12N15/111General methods applicable to biologically active non-coding 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
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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
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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
    • C12N15/63Introduction 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
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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
    • C12N15/63Introduction 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
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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)
    • 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]
    • CCHEMISTRY; METALLURGY
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    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10344Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
SG11201900038QA 2016-07-05 2017-07-05 Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter SG11201900038QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662358335P 2016-07-05 2016-07-05
PCT/US2017/040707 WO2018009534A1 (en) 2016-07-05 2017-07-05 Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter

Publications (1)

Publication Number Publication Date
SG11201900038QA true SG11201900038QA (en) 2019-02-27

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SG11201900038QA SG11201900038QA (en) 2016-07-05 2017-07-05 Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter

Country Status (14)

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

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GB2589246A (en) 2018-05-16 2021-05-26 Synthego Corp Methods and systems for guide RNA design and use
WO2019225638A1 (ja) * 2018-05-25 2019-11-28 国立大学法人筑波大学 融合タンパク質、核酸、細胞及び動物の製造方法
JPWO2020059708A1 (ja) * 2018-09-17 2021-09-02 国立大学法人 東京医科歯科大学 Casタンパク質の活性調節法
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US20200140893A1 (en) * 2018-11-01 2020-05-07 Blueallele, Llc Methods for altering gene expression for genetic disorders
JPWO2020096049A1 (ja) * 2018-11-08 2021-11-25 国立大学法人東海国立大学機構 単一のaavベクターによるゲノム編集を用いた遺伝子治療
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CN110607302A (zh) * 2019-10-09 2019-12-24 广州医科大学附属第二医院 检测花生四烯酸脂氧合酶aloxe3基因启动子突变的方法及其应用
CN111718933B (zh) * 2020-06-28 2022-01-28 暨南大学 一种rrbp1基因敲除热带爪蛙模型的制备方法与应用
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Publication number Publication date
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
CL2019000025A1 (es) 2019-06-21
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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