Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_68c213ba9952360f439567152e047b18 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_9cfe5308b6e8e901fbb62d4c726c1647 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a33a756f2fbeb706009421ba25af95c8 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3513 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3511 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3181 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-34 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-331 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-30 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-152 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3183 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P43-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-06 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-111 |
classificationIPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P7-06 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P43-00 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-7125 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
filingDate |
2010-04-21-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_878b55e977a3b973d8bc8437a73f4134 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_3431250680c54279de30cccc28fc1ecb |
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. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020257779-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020257776-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11136597-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-109476706-A http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2017143042-A3 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2017143061-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2017143042-A2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020047353-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2018187493-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021050568-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020047344-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11739124-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2019022434-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020033951-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020112195-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2011053989-A3 |
priorityDate |
2009-04-21-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |