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US 8,652,460 B2Granted
Gene delivery system and method of use
AssigneeUniversity of Southern California
InventorsNoriyuki Kasahara, Christopher R. Logg, W. French Anderson
Life of this patent
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Abstract
A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.
Claims13
A replication competent mammalian oncoretrovirus comprising:
- a nucleic acid sequence encoding a retroviral GAG protein;
- a nucleic acid sequence encoding a retroviral POL protein;
- a nucleic acid sequence encoding a retroviral envelope;
- a mammalian oncoretroviral polynucleotide sequence comprising Long-Terminal Repeat (LTR) sequences at the 5′ and 3′ end of the mammalian oncoretroviral polynucleotide sequence;
- a cassette comprising an internal ribosome entry site (IRES) operably linked to a heterologous nucleic acid sequence, wherein the cassette is positioned 5′ to the U3 region of the 3′ LTR, and just downstream and 3′ to the sequence encoding the retroviral envelope, wherein the heterologous nucleic acid sequence is expressed;
- and
- cis-acting sequences for reverse transcription, packaging and integration in a naturally occurring target mammalian cell.
4 more elements
A recombinant replication competent mammalian oncoretrovirus comprising:
- a retroviral GAG protein;
- a retroviral POL protein;
- a retroviral envelope (ENV);
- a mammalian oncoretroviral polynucleotide sequence comprising Long-Terminal Repeat (LTR) sequences at the 5′ and 3′ end of the retroviral polynucleotide sequence, wherein a mammalian promoter sequence is contained within the LTR sequences at the 5′ and/or 3′ end of the retroviral polynucleotide sequence, wherein the mammalian oncoretroviral polynucleotide sequence contains a coding sequence for GAG, POL and ENV;
- a cassette comprising an internal ribosome entry site (IRES) operably linked to a heterologous polynucleotide, wherein the cassette is positioned just downstream and 3′ to the sequence encoding the retroviral envelope and 5′ to the 3′ LTR, wherein the heterologous polynucleotide is expressed;
- and
- cis-acting sequences necessary for reverse transcription, packaging and integration in a target cell.
4 more elements
Dependencies are inferred from the claim text.