http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2010123983-A1

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filingDate 2010-04-21-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_878b55e977a3b973d8bc8437a73f4134
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publicationDate 2010-10-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2010123983-A1
titleOfInvention Compostions and methods for targeted gene therapy
abstract Compositions containing molecules that bind to duplex DNA in a sequence-specific manner to form a triple-stranded structure are disclosed. Triplex-forming molecules facilitate strand displacement and triplex formation, referred to as a "clamp," in which one molecule binds to the target strand by Hoogsteen binding and the other molecule binds to the target strand by Watson-Crick binding in a sequence specific manner. A Watson- Crick binding "tail" added to the end of the Watson-Crick binding portion of the clamp increases the stringency of binding to the target duplex, improves the frequency of modification at the target site, and reduces the requirement for a polypurine:polypyrimidine stretch compared to triplex forming oligonucleotides (TFOs) or peptide nucleic acids (PNAs). When administered in combination with a donor oligonucleotide, triplex-forming molecules can enhance recombination of a donor oligonucleotide at the target site. Ex vivo and in vivo methods of gene correction in patients using the disclosed compositions are also disclosed.
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