http://rdf.ncbi.nlm.nih.gov/pubchem/patent/JP-2005528911-A
Outgoing Links
| Predicate | Object |
|---|---|
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P35-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-04 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-755 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-63 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-67 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-10 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N1-19 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N1-15 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-09 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P7-04 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P35-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07K14-755 |
| filingDate | 2003-06-05-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationDate | 2005-09-29-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | JP-2005528911-A |
| titleOfInvention | Spliceosome-mediated RNA trans-splicing and correction of genetic defects in factor VIII using spliceosome-mediated RNA trans-splicing |
| abstract | The composition of the present invention is designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction that results in the generation of a novel chimeric RNA molecule (chimeric RNA). Contains molecules (PTM). In particular, the PTM of the present invention is genetically engineered to interact with the factor VIII (FVIII) target pre-mRNA, thereby correcting the genetic defect of coagulation factor FVIII that causes hemophilia A including. The composition of the present invention further comprises a recombinant vector system capable of expressing the PTM of the present invention, and a cell expressing the PTM. The method of the invention comprises contacting the PTM of the invention and the FVIII target pre-mRNA under conditions such that a portion of the PTM is trans-spliced to a portion of the target pre-mRNA to form an mRNA molecule. This method corrects a genetic defect in the FVIII gene. The methods and compositions of the present invention can be used in gene therapy for the correction of FVIII disorders such as hemophilia A. |
| priorityDate | 2002-06-05-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 1652.