Pseudocomplementary oligonucleotides for targeted gene therapy
US8309356B2 · kind B2 · utility
Assignee
Inventor
Key dates
| Filing date | Apr 1, 2010 |
| Grant date | Nov 13, 2012 |
| Priority date | — |
| Expiry date | Sep 15, 2030 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61P43/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Compositions and methods for targeted gene therapy are disclosed. Compositions containing double duplex-forming pseudocomplementary oligonucleotides are administered in combination with a donor oligonucleotide that is homologous to a target sequence on a double-stranded DNA molecule in need of repair or replacement. By activating cellular mechanisms involved in DNA synthesis, repair and recombination, the double duplex-forming pseudocomplementary oligonucleotides can introduce one or more mutations at a site of interest by increasing the efficiency of targeted recombination of the donor oligonucleotide. The pseudocomplementary oligonucleotides/donor oligonucleotide compositions may be administered in combination with a second therapeutic agent that enhances access of the pseudocomplementary oligonucleotides and/or the donor oligonucleotide to the target site, an agent that enhances or increases DNA repair or recombination, or an agent that enhances uptake or delivery of the oligonucleotides.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.