Chemistry: molecular biology and microbiology – Process of mutation – cell fusion – or genetic modification – Introduction of a polynucleotide molecule into or...
Patent
1997-04-15
2000-01-25
Railey, II, Johnny F.
Chemistry: molecular biology and microbiology
Process of mutation, cell fusion, or genetic modification
Introduction of a polynucleotide molecule into or...
4353201, 435325, 435350, 435357, 435363, 435364, 435366, 435369, 435371, 536 2372, C12N 510, C12N 1509, C12N 1548, C12N 1586
Patent
active
060177616
DESCRIPTION:
BRIEF SUMMARY
TECHNICAL FIELD OF THE INVENTION
This invention generally relates to the derivation and use of packaging cell lines for the production of retroviral transducing supernatant.
BACKGROUND OF THE INVENTION
Human gene transfer involves the transfer of one or more therapeutic genes and the sequences controlling their expression to appropriate target cells. A number of vector systems have been developed for the transfer of the therapeutic genes for various clinical indications. In vivo gene transfer involves the direct administration of vector to the target cells within a patient. Ex vivo gene transfer entails removing target cells from an individual, modifying them ex vivo and returning the modified cells to the patient.
The majority of gene therapy protocols approved for clinical trials by the NIH Recombinant DNA Advisory Committee (RAC) have used arnphotropic retroviral vectors (ORDA Reports Recombinant DNA Advisory Committee (RAC) Data Management Report, June 1994, (1994) Human Gene Therapy 5:1295-1302). Retroviral vectors are the vehicle of choice primarily due to the generally high rate of gene transfer obtained in experiments with cell lines and the ability to obtain stable integration of the genetic material, ensuring that the progeny of the modified cell will contain the transferred genetic material. For a review of retroviral vectors and their use in the transfer and expression of foreign genes, see Gilboa (1988) Adv. Exp. Med. Biol. 241:29; Luskey et al. (1990) Ann. N.Y. Acad. Sci. 612:398; and Smith (1992) J. Hematother. 1:155-166.
Many retroviral vectors currently in use are derived from the Moloney murine leukemia virus (MMLV). In most cases, the viral gag, pol and env sequences are removed from the virus, allowing for insertion of foreign DNA sequences. Genes encoded by the foreign DNA are often expressed under the control of the strong viral promoter in the LTR. Such a construct can be packaged into vector particles efficiently if the gag, pol and env functions are provided in trans by a packaging cell line. Thus, when the vector construct is introduced into the packaging cell, the Gag-Pol and Env proteins produced by the cell assemble with the vector RNA to produce replication-defective or transducing virions that are secreted into the culture medium. The vector particles thus produced can infect and integrate into the DNA of the target cell, but generally will not produce infectious virus since it is lacking essential viral sequences.
Most of the packaging cell lines currently in use have been transfected with separate plasmids encoding Gag-Pol and Env, so that multiple recombination events are necessary before a replication-competent retrovirus (RCR) can be produced. Commonly used retroviral vector packaging cell lines are based on the murine NIH/3T3 cell line and include PA317 (Miller & Buttimore (1986) Mol. Cell Biol. 6:2895; Miller & Rosman (1989) BioTechniques 7:980), CRIP (Danos & Mulligan (1988) Proc. Natl Acad Sci USA 85:6460), and gp+am12 (Markowitz et al. (1988) Virology 167:400). Although splitting the gag-pol and env genes within the packaging cell genome decreases the incidence of RCR, RCR is occasionally observed in clinical-scale productions of retroviral vector preparations and is a major safety concern. This is likely due, at least in part, to the fact that NIH/3T3 cells contain endogenous MLV sequences (Irving et al. (1993) Bio/Technol. 11:1042-1046) which could participate in recombination to form RCR (Cosset et al. (1993) Virology 193:385-395 and Vanin et al. (1994) J Virology 68:4241-4250), particularly in mass culture during large-scale clinical vector production.
The range of host cells that may be infected by a retrovirus or transduced by a retroviral vector is determined by the viral Env protein. The recombinant virus can be used to infect virtually any cell type recognized by the Env protein provided by the packaging cell, resulting in the integration of the viral genome in the transduced cell and the stable production of the foreign gene product. The efficiency of
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Bohnlein Ernst
Chen Jingyi
Dando Jonathan S.
Forestell Sean P.
Plavec Ivan
Railey II Johnny F.
Shaw Melissa A.
Systemix, Inc.
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