DK2847335T3 - Nukleasemedieret målretning med store målretningsvektorer - Google Patents
Nukleasemedieret målretning med store målretningsvektorer Download PDFInfo
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- DK2847335T3 DK2847335T3 DK13720718.9T DK13720718T DK2847335T3 DK 2847335 T3 DK2847335 T3 DK 2847335T3 DK 13720718 T DK13720718 T DK 13720718T DK 2847335 T3 DK2847335 T3 DK 2847335T3
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- 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
- C12N15/63—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
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
- C07K14/7155—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for interleukins [IL]
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- 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
- C12N15/63—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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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- 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
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Claims (13)
1. Fremgangsmåde til modificering af et genomisk mållocus i en muse-embryostam- (ES) celle, der omfatter: (a) introduktion i muse-ES-cellen af: (i) en zinkfingemuklease (ZFN), der frembringer et dobbeltstrenget brud ved eller i nærheden af et genomisk mållocus; og (ii) en stor målretningsvektor (LTVEC), der omfatter et indsætningsmolekyle flankeret af en opstrømshomologiarm og en nedstrømshomologiarm, hvor indsætningsnukleinsyren ligger inden for intervallet fra 5 kb til 30 kb i længden, hvor den totale sum af opstrøms- og nedstrømshomologiarmene er mindst 10 kb i længden, hvor opstrøms- og nedstrømshomologiarmene er mellem 5 kb og 200 kb i længden, og hvor LTVEC’en ligger inden for intervallet fra 50 kb til 300 kb i længden; og (b) udvælgelse af en målrettet muse-ES-celle, der omfatter indsætningsnukleinsyren i det genomiske mållocus, hvor integration af indsætningsnukleinsyren i det genomiske mållocus resulterer i udskiftning af en endogen nukleinsyresekvens ved det genomiske mållocus med indsætningsnukleinsyren.
2. Fremgangsmåde ifølge krav 1, hvor indsætningsnukleinsyren omfatter et reporter-gen operativt bundet til en exogen reporter.
3. Fremgangsmåde ifølge krav 1, hvor kombineret anvendelse af LTVEC’en med ZFN’en resulterer i øget målretningseffektivitet sammenlignet med anvendelse af LTVEC’en alene, eventuelt hvor målretningseffektiviteten øges mindst to gange sammenlignet med anvendelse af LTVEC’en alene.
4. Fremgangsmåde ifølge krav 1, hvor: (I) ZFN’en er et ekspressionskonstrukt, der omfatter en nukleinsyresekvens, der koder for et ZFN-protein, og hvor nukleinsyren er operativt bundet til en promoter, der er aktiv i cellen; eller (II) ZFN’en er et mRNA, der koder for et ZFN-protein.
5. Fremgangsmåde ifølge krav 1, hvor en målsekvens for ZFN’en er placeret i en intron, en exon, en promoter, en promoter-regulatorisk region eller en enhancer-region i det genomiske mållocus.
6. Fremgangsmåde ifølge krav 1, hvor: (I) indsætningsnukleinsyren omfatter en udvælgelseskassette; (II) indsætningsnukleinsyren omfatter en human nukleinsyresekvens; eller (III) indsætningsnukleinsyren omfatter en konditionel allel.
7. Fremgangsmåde ifølge krav 1, hvor: (I) indsætningsnukleinsyren omfatter en nukleinsyresekvens, der er homolog med den udskiftede nukleinsyresekvens ved muse-ES-cellens genomiske mållocus; (II) indsætningsnukleinsyren omfatter et nukleinsyresekvens, der er ortolog med den udskiftede nukleinsyresekvens ved muse-ES-cellens genomiske mållocus; eller (III) indsætningsnukleinsyren omfatter en nukleinsyresekvens af en anden art.
8. Fremgangsmåde ifølge krav 1, hvor indsætningsnukleinsyren omfatter en ikke-omarrangeret human nukleinsyresekvens af en variabel tungkæderegion operativt bundet til en immunoglobulinnukleinsyresekvens af en konstant tungkæderegion, eventuelt hvor immunoglobulinnukleinsyresekvensen af den konstante tungkæderegion er en muse-immunoglobulinsekvens af en konstant tungkæderegion, en human immunoglobulinsekvens af en konstant tungkæderegion, eller en kombination deraf, og eventuelt hvor immunoglobulinnukleinsyresekvensen af en konstant tungkæderegion er valgt fra et ChI, et hængsel, et Ch2, et Ch3 og en kombination deraf.
9. Fremgangsmåde ifølge krav 1, hvor indsætningsnukleinsyren omfatter en ikke-omarrangeret human λ- eller κ-nukleinsyresekvens af en variabel letkæderegion operativt bundet til en muse- eller human immunoglobulinnukleinsyresekvens af en konstant letkæderegion valgt fra en λ-nukleinsyresekvens af en konstant letkæderegion og en k-nukleinsyresekvens af en konstant letkæderegion.
10. Fremgangsmåde ifølge krav 1, hvor indsætningsnukleinsyren omfatter en omarrangeret human λ- eller κ-nukleinsyresekvens af en variabel letkæderegion operativt bundet til en museeller human immunoglobulin nukleinsyresekvens af en konstant letkæderegion valgt fra en λ-nukleinsyresekvens af en konstant letkæderegion og en κ-nukleinsyresekvens af en konstant letkæderegion.
11. Fremgangsmåde ifølge krav 1, hvor integration af indsætningsnukleinsyren i det genomiske mållocus resulterer i et knockout, et knock-in, en punktmutation, et domæne-swap, et exon-swap, et intron-swap, et regulatorisk sekvens-swap, et gen-swap eller en kombination deraf.
12. Fremgangsmåde ifølge krav 1, hvor valg af trin (b) udføres via en modifikation af allel-(MOA) assay.
13. Fremgangsmåde ifølge krav 1, der endvidere omfatter: (c) introduction af den modificerede muse-ES-celle i et embryo i præmorulastadiet; og (d) inkubation af embryoet indtil blastocyststadiet, eventuelt hvor fremgangsmåden omfatter frembringelse af en FO-mus ud fra embryoet implanteret i en surrogatmor; og (e) identificering af en mus, der bærer det genetisk modificerede genomiske locus.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261638267P | 2012-04-25 | 2012-04-25 | |
| PCT/US2013/038165 WO2013163394A1 (en) | 2012-04-25 | 2013-04-25 | Nuclease-mediated targeting with large targeting vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2847335T3 true DK2847335T3 (da) | 2018-08-13 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK13720718.9T DK2847335T3 (da) | 2012-04-25 | 2013-04-25 | Nukleasemedieret målretning med store målretningsvektorer |
Country Status (19)
| Country | Link |
|---|---|
| US (2) | US9834786B2 (da) |
| EP (1) | EP2847335B1 (da) |
| JP (1) | JP6275120B2 (da) |
| KR (1) | KR101904508B1 (da) |
| CN (2) | CN104364380B (da) |
| AU (1) | AU2013251558B2 (da) |
| BR (1) | BR112014026294B1 (da) |
| CA (1) | CA2869016C (da) |
| CY (1) | CY1120572T1 (da) |
| DK (1) | DK2847335T3 (da) |
| ES (1) | ES2683071T3 (da) |
| IL (1) | IL234905B (da) |
| IN (1) | IN2014DN09261A (da) |
| MX (1) | MX362523B (da) |
| PL (1) | PL2847335T3 (da) |
| PT (1) | PT2847335T (da) |
| RU (1) | RU2645475C2 (da) |
| SG (2) | SG11201406547YA (da) |
| WO (1) | WO2013163394A1 (da) |
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