ZA201800026B - Crispr/cas9 complex for genomic editing - Google Patents

Crispr/cas9 complex for genomic editing

Info

Publication number
ZA201800026B
ZA201800026B ZA2018/00026A ZA201800026A ZA201800026B ZA 201800026 B ZA201800026 B ZA 201800026B ZA 2018/00026 A ZA2018/00026 A ZA 2018/00026A ZA 201800026 A ZA201800026 A ZA 201800026A ZA 201800026 B ZA201800026 B ZA 201800026B
Authority
ZA
South Africa
Prior art keywords
crispr
genomic editing
cas9 complex
cas9
complex
Prior art date
Application number
ZA2018/00026A
Other languages
English (en)
Inventor
Townes Tim
Ding Lei
Chang Chia-Wei
Original Assignee
Uab 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 Uab Res Found filed Critical Uab Res Found
Publication of ZA201800026B publication Critical patent/ZA201800026B/en

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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
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
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    • C12N5/0602Vertebrate cells
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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 [RNase]; Deoxyribonucleases [DNase]
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    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
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    • C07K2319/00Fusion polypeptide
    • C07K2319/60Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
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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 [CRISPR]
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    • C12N2800/00Nucleic acids vectors
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    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers

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ZA2018/00026A 2015-06-17 2018-01-03 Crispr/cas9 complex for genomic editing ZA201800026B (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562181138P 2015-06-17 2015-06-17
US201562266316P 2015-12-11 2015-12-11
PCT/US2016/038161 WO2016205703A1 (en) 2015-06-17 2016-06-17 Crispr/cas9 complex for genomic editing

Publications (1)

Publication Number Publication Date
ZA201800026B true ZA201800026B (en) 2019-04-24

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ZA2018/00026A ZA201800026B (en) 2015-06-17 2018-01-03 Crispr/cas9 complex for genomic editing

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US (2) US11643668B2 (enExample)
EP (1) EP3310932B1 (enExample)
JP (1) JP2018518182A (enExample)
CN (1) CN107949641A (enExample)
AU (1) AU2016278982A1 (enExample)
CA (1) CA2989831A1 (enExample)
WO (1) WO2016205703A1 (enExample)
ZA (1) ZA201800026B (enExample)

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US20240117380A1 (en) 2024-04-11
EP3310932A4 (en) 2019-01-02
US11643668B2 (en) 2023-05-09
AU2016278982A1 (en) 2018-01-18
JP2018518182A (ja) 2018-07-12
WO2016205703A1 (en) 2016-12-22
CN107949641A (zh) 2018-04-20

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