Lentiviral nucleic acids and uses thereof
AssigneeThe Regents of the University of California
InventorsEric M. Poeschla, David J. Looney, Flossie Wong-Staal
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Abstract
The invention provides non-primate lentiviral vectors, packaging cells and packaging plasmids based, for example, on feline and ungulate retroviruses. In particular, the packaging plasmids are designed for expression in human cells (which are also used as packaging cells). The vectors of the invention transduce human cells, including difficult to target non-dividing cells of the hematopoietic and nervous system, in vitro and in vivo. The vectors are suitable for general gene transfer to these cells and for gene therapy to treat conditions mediated by these non-dividing cells including cancer and HIV infection.
Claims46
A packageable nucleic acid comprising a FIV packaging site and a FIV 5′ LTR, wherein:
- the U3 region of the 5′ LTR or a portion thereof is replaced by an expression control sequence which functions in human cells;
- and
- the packageable nucleic acid is not capable of self packaging.
A method for transfecting a cell with a packageable nucleic acid or transducing a cell with a viral particle comprising a packageable nucleic acid, which comprises contacting the cell with the packageable nucleic acid or the viral particle, wherein:
- the packageable nucleic acid comprises a FIV packaging site, a FIV 5′ LTR, and a heterologous target nucleic acid operably linked to an expression control sequence;
- the U3 region of the 5′ LTR or a portion thereof is replaced by the expression control sequence;
- and
- the packagable nucleic acid is not capable of self packaging.
Dependencies are inferred from the claim text.