Chemistry: molecular biology and microbiology – Vector – per se
Patent
1997-11-28
2000-08-01
Hauda, Karen M.
Chemistry: molecular biology and microbiology
Vector, per se
4352351, 435325, C12N 1500, C12N 700, C12N 510
Patent
active
060965347
DESCRIPTION:
BRIEF SUMMARY
FIELD OF INVENTION
This invention relates to the fields of genetic engineering and gene transfer. More specifically, the invention relates to recombinant retrovirus vectors derived from avian sarcoma leukosis viruses (ASLVs) having an expanded host range. In particular, this invention relates to ASLV recombinant retrovirus vectors wherein a viral env gene derived from a virus capable of infecting both mammalian and avian cells is substituted for the ASLV env gene, allowing the vectors to efficiently infect a wide range of host cells, including mammalian and particularly human cells, in high titers. Additionally, this invention encompasses therapeutic applications employing these vectors.
BACKGROUND OF INVENTION
Retroviral vectors carrying and expressing nucleic acid sequences of interest are powerful tools for the transfer of genes into a broad range of mammalian cells and into animals, including humans. Indeed, retroviruses offer substantial advantages for use as vectors carrying and expressing desired nucleic acid sequences in both cultured cells and intact animals. (Weiss et al, RNA Tumor Viruses (1982)).
First, the retrovirus life cycle lends itself to the efficient transfer of genes into host cells. The infectious retroviral agent is called a viral particle or a virion. Virions consist of a capsid containing the viral genome and any inserted nucleic acid sequences and an envelope made up of glycoproteins. The envelope glycoproteins on the surface of the virion recognize receptors on the host cell that mediate entry of the RNA retroviral genome into the host cell. Once inside the host cell, a double stranded DNA copy of the virion RNA genome and any inserted nucleic acid sequences of interest is made by a viral enzyme, reverse transcriptase. This DNA copy integrates into the host genome at a precise point on the viral DNA molecule and at random, or nearly random sites on host chromosonal DNA. The integrated viral DNA copy is called a provirus. Since a DNA copy of the viral genome integrates into the host genome, the progeny of a single infected host cell are all infected, and the provirus is located in the same place in the genome of each of the progeny cells.
Second, in completing their replicative process, retroviruses usually do not lyse the host cell. Thus, the retroviruses constitute an efficient mechanism for the introduction and high level expression of genes in living host cells.
Third, retroviral genomes are small, making it relatively easy to manipulate a cloned DNA copy of the genome. Moreover, the viruses are efficient; in culture, essentially all of the cells can be infected.
The ability of the retroviral replication machinery to introduce genetic information into the genome of the target cell provided the inspiration for the development of recombinant retrovirus vectors containing a nucleic acid sequence of interest as a vehicle for the stable transfer of genes. Moreover, recombinant retroviral vectors have been used in a number of applications in addition to the expression of genes of interest, including insertional mutagenesis, cell lineage studies and the creation of transgenic animals.
A desirable property useful for the retroviral vector is the ability to replicate in certain easily manipulated host cells, (e.g., avian cells) allowing rapid replication in these cells without aid of a helper or packaging cell line. This permits generation of high titer virus stocks by simply passaging transfected cells and allowing the virus to spread.
Another useful property for a retroviral vector is the ability to infect a wide range of host cells, including mammalian, and particularly human, cells in high titers. Preferably, the retroviral vector is unable to replicate in mammalian cells. Thus, once the vector enters the mammalian host cell, it becomes a stable provirus, integrated in the host cell genome and incapable of further rounds of infection in either the present or subsequent generations.
A number of retroviral vector systems have been described, including systems based on both mammal
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Barsov Eugene
Hughes Stephen H.
Auth Dorothy R.
Beckerleg Anne Marie S.
Feiler William S.
Hauda Karen M.
The United States of America as represented by the Department of
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