Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_0a510f8260c48925c4883982d3db4a1b |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-755 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-22 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-102 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-907 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-00 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P7-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07K14-755 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-09 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N9-10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N9-16 |
filingDate |
2015-06-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_4b00bed74f0f922e18d2a72a1c1dc4d1 |
publicationDate |
2017-05-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
WO-2015191899-A9 |
titleOfInvention |
FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS |
abstract |
The present disclosure relates to methods, systems, and compositions to repair one or more mutations in a Factor VIII gene sequence of a subject by introducing into a cell of the subject one or more polynucleotides encoding a DNA scission enzyme (DNA-SE) and one or more repair vehicles (RVs) containing at least a cDNA-repair sequence (RS) such that insertion of the cDNA-RS through homologous recombination with the F8 gene of the subject (sF8) provides a repaired F8 gene (rF8), the repaired F8 gene (rF8) upon expression forming a functional FVIII conferring improved coagulation functionality to the FVIII protein encoded by the sF8. The present disclosure also relates to cells derived using the methods, systems and compositions described. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11083801-B2 |
priorityDate |
2014-06-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |