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OwnerPierre Charneau, Veronique Zennou +3inventors
InventorsPierre Charneau, Veronique Zennou, Francoise Pflumio, Aude Sirven, Anne Dubart Kupperschmitt
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The present ivention provides nucleic acid, vectors, viruses, and recombinant cells comprising triple-stranded structures, such as those resulting from central initiation and termination of HIV-1 reverse transcription at the center of HIV-1 linear DNA genomes. These triplex structures can act as a cis-determinant of HIV-1 DNA nuclear import, allowing infection of non-dividing target cells. In one aspect, the presence of the DNA triplex sequence in an HIV vector strongly stimulates gene transfer in hematopoietic stem cells. The invention also provides methods of using these triplex structures for making recombinant cells, as well as methods of using the recombinant cells to express proteins of interest both in vitro and in vivo.
An isolated or purified nucleic acid comprising at least one copy of the cPPT and CTS cis-acting regions of a retrovirus, wherein the cPPT and CTS regions induce a three-stranded DNA structure.
A process for inserting a nucleic acid of interest into the nucleus of a target cell, said method comprising exposing an isolated or purified nucleic acid comprising at least one copy of the cPPT and CTS cis-acting regions of a retrovirus, wherein the cPPT and CTS regions induce a three-stranded DNA structure, to a target cell under conditions that permit uptake of the nucleic acid of interest into the target cell.
A process for expressing a gene of interest in vitro, said process comprising
A process for treating an individual suffering from, or having a high likelihood of developing, a disease or disorder having a genetic basis, said process comprising administering a retroviral vector comprising a) a nucleic acid encoding a therapeutic protein and b) at least one copy of the cPPT and CTS cis-acting regions of a retrovirus, wherein the cPPT and CTS regions induce a three-stranded DNA structure, to said individual in an amount sufficient to result in expression of said therapeutic protein in an amount sufficient to treat said disease or disorder.
A kit containing at least one container containing an isolated or purified nucleic acid comprising at least one copy of the cPPT and CTS cis-acting regions of a retrovirus, wherein the cPPT and CTS regions induce a three-stranded DNA structure.
Dependencies are inferred from the claim text.