Chemistry: molecular biology and microbiology – Vector – per se
Patent
1994-06-01
1998-04-07
Chambers, Jasemine C.
Chemistry: molecular biology and microbiology
Vector, per se
435325, 435 697, 435 695, 435 6951, 435 6952, 435 912, 435 6, 424 9321, 514 44, 5303871, 530350, 536 235, 536 2431, C12N 1563
Patent
active
057363870
DESCRIPTION:
BRIEF SUMMARY
TECHNICAL FIELD
The present invention relates to retroviral vectors useful for introducing polynucleotides into target cells and more specifically to retroviral particles having on their surface chimeric targeting proteins capable of both binding to specific target cells via cellular cytokine receptors and mediating vector entry into the targeted cells.
BACKGROUND OF THE INVENTION
Retroviruses are a family of RNA viruses that contain RNA-directed DNA polymerase activity. These viruses possess similar structural characteristics, being membranous, icosahedral particles that are 70-120 nM in diameter and contain RNA as well as the structural and catalytic proteins necessary for infection of a host cell and subsequent reverse transcription of the viral genome. Retroviruses that are secreted from cells can be categorized into B-, C-, and D-type, lentiviruses, spumaviruses, and others, based on criteria relating to the shape of the nucleocapsid core within the particle, the nature of the budding process and the complexity of the viral genome, see, e.g., Weiss, R., et al., (1984 and 1985) "RNA Tumor Viruses," Cold Spring Harbor, N.Y.
Retroviruses typically have three common open reading frames, gag, pol and env, which encode the structural proteins, enzymatic activities including reverse transcriptase, and envelope proteins, respectively. The product of the env gene is a polyprotein precursor (typically referred to herein as the "envelope protein" or the "env protein"), that is proteolytically cleaved during transport to yield two polypeptides: a glycosylated protein found on the external surface of the particle (the "SU" protein), and a membrane-spanning or transmembrane protein (the "TM" protein); see, e.g., Hunter, E. and R. Swanstrom, Curr. Topics Microbiol. Immunol. 157:187-253, 1990. The SU protein is involved in recognizing and binding to cellular receptors, while the TM protein is involved in mediating the fusion of viral and cellular membranes necessary for viral uptake, id.
A retroviral vector is an infectious viral particle useful in achieving stable and efficient transduction of a gene into mammalian cells, including some cells that are not readily transfectable by other methods, such as primary cells of various types and also cells in vivo. Retroviral vectors based on murine C-type retroviruses have been used successfully for a number of years to introduce and express genes in cultured mammalian cells. Typically, retroviral vectors are produced by "packaging cell lines" that provide the necessary retroviral gag, pol and env gene products in trans, see, e.g., Miller et al., Human Gene Therapy 1:5-14 (1990); and Ausubel, F. M. et al. (eds), Units 9.10-9.14 in "Current Protocols in Molecular Biology," John Wiley & Sons, New York, 1992. This approach results in the production of retroviral particles which are highly infectious for mammalian cells, but are incapable of further replication after they have integrated into the genome of the target cell. Gene therapy protocols using retroviral vectors generally rely on a cell line containing the gag and pol genes, along with the env genes from an "amphotropic" (i.e. broad host range) virus such as 4070A amphotropic virus, which yields amphotropic progeny virus (e.g., the "PA317" cell line described by Miller and Buttimore, Mol. Cell. Biol. 6:2895, 1986).
Gene therapy protocols typically are ex vivo in nature, in that they require harvesting cells from the patient, culturing them in vitro, transducing them with the retroviral vector and returning the transduced cells to the patient; see, e.g., Kasid et al., Proc. Natl. Acad. Sci. USA 87:473, 1990; and Rosenberg et al., N. Engl. J. Med. 323:570, 1990.
In many situations, it would be desirable to restrict viral infection to a specific sub-population of the exposed cells. In such circumstances, the broad host range typical of retroviruses presents a substantial problem. Even where it is possible to purify the desired target cells, either before or after transduction, this necessitates undesirable manipulation
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Overell Robert
Paul Ralph W.
Chambers Jasemine C.
Schmuck Jill D.
Targeted Genetics Corporation
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