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US 9,732,326 B2Granted
Recombinant vectors
AssigneeTocagen Inc.
InventorsHarry E. Gruber, Douglas J. Jolly, Omar D. Perez, Christopher R. Logg
Life of this patent
Estimating the term…
Abstract
This disclosure provides modified cytosine deaminases (CDs). The disclosure further relates to cells and vector expressing or comprising such modified CDs and methods of using such modified CDs in the treatment of disease and disorders.
Claims24
A recombinant replication competent retrovirus comprising:
- a retroviral GAG protein;
- a retroviral POL protein;
- a retroviral envelope;
- a retroviral RNA polynucleotide comprising 3′ untranslated region (U3) and repeat region (R) sequences at the 3′ end of the retroviral polynucleotide sequence, an R and 5′ untranslated region (U5) sequence at the 5′ end of the retroviral polynucleotide, a gag nucleic acid domain, a pol nucleic acid domain and an env nucleic acid domain located between the U5 and U3 regions;
- a cassette comprising a pol III promoter upstream and operably linked to a heterologous polynucleotide, wherein the cassette is positioned 5′ to the U3 region and 3′ to the env nucleic acid domain;
- and
- cis-acting sequences necessary for reverse transcription, packaging and integration in a target cell;
- wherein the retroviral RNA polynucleotide provides a proviral DNA sequence comprising a gag nucleic acid domain comprising a sequence from about nucleotide number 1203 to about nucleotide 2819 of SEQ ID NO:
- 19 or 22, the pol nucleic acid domain comprising the sequence from about nucleotide number 2820 to about nucleotide 6358 of SEQ ID NO:19 or 22, the env domain comprising the sequence from about nucleotide number 6359 to about nucleotide 8323 of SEQ ID NO:19 or 22;
- and the cassette, and
- wherein the virus can infect a target cell multiple times resulting in an average number of copies/diploid genome of 5 or greater.
8 more elements
A recombinant replication competent retrovirus (RCR) comprising:
- a retroviral GAG protein;
- a retroviral POL protein;
- a retroviral envelope;
- a retroviral RNA polynucleotide comprising a 3′ untranslated region (U3) and repeat region (R) sequences at the 3′ end of the retroviral polynucleotide sequence, an R and 5′ untranslated region (U5) sequence at the 5′ end of the retroviral polynucleotide, a gag nucleic acid domain, a pol nucleic acid domain and an env nucleic acid domain located between the U5 and U3 regions;
- a cassette comprising an primary precursor miRNA (pri-miRNA) for an miRNA or siRNA sequence operably linked to a pol III promoter, wherein the cassette is positioned 5′ to the U3 region and 3′ to the env nucleic acid domain;
- wherein the retroviral RNA polynucleotide provides a proviral DNA sequence consisting of a gag nucleic acid domain comprising a sequence from about nucleotide number 1203 to about nucleotide 2819 of SEQ ID NO:
- 19 or 22, a pol nucleic acid domain comprising the sequence from about nucleotide number 2820 to about nucleotide 6358 of SEQ ID NO:19 or 22, an env domain comprising the sequence from about nucleotide number 6359 to about nucleotide 8323 of SEQ ID NO:19 or 22, and the cassette;
- and
- cis-acting sequences necessary for reverse transcription, packaging and integration in a target cell.
6 more elements
Dependencies are inferred from the claim text.