CA2238436A1 - Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant - Google Patents

Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant

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Publication number
CA2238436A1
CA2238436A1 CA002238436A CA2238436A CA2238436A1 CA 2238436 A1 CA2238436 A1 CA 2238436A1 CA 002238436 A CA002238436 A CA 002238436A CA 2238436 A CA2238436 A CA 2238436A CA 2238436 A1 CA2238436 A1 CA 2238436A1
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CA
Canada
Prior art keywords
cell
retroviral
env
gag
packaging
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Legal status (The legal status 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 status listed.)
Granted
Application number
CA002238436A
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French (fr)
Other versions
CA2238436C (en
Inventor
Richard J. Rigg
Jingyi Chen
Jonathan S. Dando
Ivan Plavec
Sean P. Forestell
Ernst Bohnlein
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Systemix Inc
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Individual
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Publication of CA2238436A1 publication Critical patent/CA2238436A1/en
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Publication of CA2238436C publication Critical patent/CA2238436C/en
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Expired - Lifetime legal-status Critical Current

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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
    • C12N15/63—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
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13051—Methods of production or purification of viral material
    • C12N2740/13052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Abstract

This invention provides a method for obtaining a recombinant retroviral packaging cell capable of producing retroviral vectors as well as the recombinant packaging cell obtained by the method. Also provided is a method of producing recombinant retroviral particles obtained by introducing into the packaging cells obtained according to the methods disclosed herein, a recombinant retroviral vector and propagating the resulting producer cells under conditions favorable for the production and secretion of retroviral vector supernatant. The retroviral supernatants produced by these methods also is claimed herein. This invention further provides a method for screening retroviral vector supernatant for high transduction efficiency and methods for producing retroviral vector supernatant for transducing cells with high efficiency in gene therapy applications.

Claims (41)

1. A method for obtaining a recombinant retroviral packaging cell capable of producing retroviral vectors comprising the steps:

a. selecting a retrovirus;
b. obtaining a eukaryotic cell free of endogenous retroviral nucleic acid of the same type as the retrovirus of step (a);
c. preparing a minimal gag-pol open reading frame (ORF) insert from the retrovirus or its equivalent, wherein the minimal gag-pol ORF is defined by the sequences from the initiation through the termination codon of the gag-pol gene with no flanking retroviral sequences;
d. inserting the minimal gag-pol ORF prepared from step (c) into an appropriate expression plasmid;
e. preparing a minimal env open reading frame (ORF) insert from the retrovirus or its equivalent, wherein the minimal env ORF
is defined by the sequences from the initiation through the termination codon of the env gene with no flanking retroviral sequences;
f. inserting the minimal env orf prepared from step (e) into an appropriate expression plasmid;
g. inserting the expression plasmids of steps (d) and (f) into the cell of step (b);
h. propagating the cell obtained from step (g) under conditions favorable for expression of the minimal retroviral gag-pol and env orf; and i. screening the cell of step (h) for retroviral Gag, Pol and Env production, thereby obtaining a retroviral packaging cell capable of packaging recombinant retroviral vector sequences to produce recombinant, transducing retroviral vector particles.
2. The method of claim 1, wherein the retrovirus is a murine leukemia virus.
3. The method of claim 1, wherein the cell is a non-murine cell.
4. The method of claim 3, wherein the non-murine cell is a primate cell.
5. The method of claim 4, wherein the primate cell is a human cell.
6. The method of claim 3, wherein the non-murine cell is selected from the group consisting of Vero, HT-1080, D17 MRC-5, FS-4, TE671, human embryonic kidney (293), and HeLa.
7. The method of claim 6, wherein the human embryonic kidney cells are 293 cells (ATCC CRL 1573).
8. The method of claim 1, wherein the gag-pol ORF is a moloney murine leukemia virus gag-pol gene.
9. The method of claim 1, wherein the env ORF is a murine leukemia virus env gene.
10. The method of claim 1, wherein the plasmid of steps (d) or (f) comprises a selectable or detectable marker gene.
11. The method of claim 1, wherein in step (i), Gag, Pol and Env production is screened by a sandwich ELISA assay.
12. The method of claim 11, wherein Env is detected using a primary antibody from hybridoma 83A25 followed by antisera 79S-834, enzyme-conjugated antispecies antibody and enzyme substrate; and Gag is detected separately using a primary antibody from hybridoma R187 (ATCC Accession No. CRL 1912) followed by antisera 77S-227, enzyme-conjugated antispecies antibody and enzyme substrate.
13. The method of claim 1, further comprising screening the cell after step (i) for the ability to produce a supernatant having high transduction efficiency, comprising measuring the ability of the supernatant to transduce a target cell population.
14. The method of claim 13, wherein the target cell population is human 293 cells.
15. The method of claim 1 wherein the cell is propagated in a sparged packed-bed bioreactor.
16. The method of claim 8 wherein the gag-pol gene is expressed from the MMLV-LTR promoter.
17. The method of claim 8, wherein the gag-pol gene is expressed from the CMV-IE promoter or the RSV-LTR promoter.
18. The method of claim 1, wherein both the gag-pol open reading frame insert and the env open reading frame insert are prepared by polymerase chain reaction.
19. The recombinant retroviral packaging cell obtained by the method of claim 1.
20. The recombinant retroviral packaging cell obtained by the method of claim 18 wherein the cell is derived from human 293 cells (ATCC CRL
1573).
21. The retroviral packaging cell of claim 19, wherein the cell produces an amphotropic env.
22. The retroviral packaging cell of claim 19, wherein the cell produces a xenotropic env.
23. The retroviral packaging cell of claim 19, wherein the cell produces a chimeric amphotropic/xenotropic env.
24. A retroviral vector producer cell produced by the method of claim 13.
25. The retroviral packaging cell line of claim 24 wherein the cell line is designated ProPak-A.6 and has ATCC Accession No. CRL 12006.
26. The retroviral packaging cell line of claim 24 wherein the cell line is designated ProPak A.52.
27. The retroviral packaging cell line of claim 24 wherein the cell line is designated ProPak-X.36 and has ATCC Accession No. CRL 12007.
28. A method of producing a retroviral vector producer cell which comprises transducing the cells of claims 19 or 20 with a retroviral-based vector and subsequently propagating the cell under conditions favorable for the production and secretion of retroviral vector supernatant.
29. The method of claim 26, wherein the retroviral-based vector is produced in human cells.
30. The retroviral vector producer cell produced by the method of claim 28.
31. A method of increasing the gene transduction efficiency of a cell, comprising transducing the cell with a retroviral vector supernatant produced from the culture of at least one retroviral vector producer cell of claim 30.
32. The method of claim 28 wherein the vector supernatant is produced from a stable vector producer cell culture.
33. The method of claim 31 wherein the packaging cell is selected from group consisting of ProPak-A.6 (ATCC Accession No. CRL 12006), ProPak-A.52 or ProPak-X.36 (ATCC Accession No. 12007).
34. The method of claim 31 wherein the retroviral vector supernatant is produced from the co-culture of a first and a second complementary vector producer cell.
35. The method of claim 34 wherein the first vector producer cell is derived from an amphotropic packaging cell and the second vector producer cell is derived from a xenotropic packaging cell.
36. The method of claim 34 wherein the first packaging cell is ProPak-A.6 designated by ATCC Accession No. CRL 12006, and the second packaging cell is ProPak-X.36 designated by ATCC Accession No. CRL

12007.
37. The method of claim 35 wherein both the amphotropic and xenotropic packaging cells are produced from human 293 cells (ATCC CRL
1573).
38. The method of claim 33 wherein the cell is a primary human hematopoietic cell.
39. The method of claim 33 wherein the cell is a human hematopoietic stem cell.
40. The method of claim 38 wherein the hematopoietic cell is a CD34+Thyl+ cell from mobilized peripheral blood or a CD4+PBL.
41. The method of claim 40 wherein the packaging cell is propagated in a sparged packed-bed bioreactor and the hematopoietic cell is transduced by two or more rounds of spinoculation with retroviral vector supernatant in the presence of IL3, IL6 and either LIF or SCF.
CA2238436A 1995-12-15 1996-12-13 Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant Expired - Lifetime CA2238436C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US08/572,959 1995-12-15
US08/572,959 US5910434A (en) 1995-12-15 1995-12-15 Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
PCT/US1996/020777 WO1997021825A1 (en) 1995-12-15 1996-12-13 Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant

Publications (2)

Publication Number Publication Date
CA2238436A1 true CA2238436A1 (en) 1997-06-19
CA2238436C CA2238436C (en) 2011-08-23

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Country Status (11)

Country Link
US (2) US5910434A (en)
EP (1) EP0871754B1 (en)
JP (1) JP2000501944A (en)
AT (1) ATE498689T1 (en)
AU (1) AU706543B2 (en)
CA (1) CA2238436C (en)
DE (1) DE69638334D1 (en)
DK (1) DK0871754T3 (en)
ES (1) ES2357736T3 (en)
PT (1) PT871754E (en)
WO (1) WO1997021825A1 (en)

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