| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_1721d75f5bf603447ec5a26b5c1c0be0 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2330-51
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-34
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P27-02
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-102
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0019
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-907
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P27-02
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K9-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
| filingDate |
2016-05-02-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_e5d1e81f7617fd5c73242d4cfe067b68
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_5ddc628c45359ed0057a0d80562876fc
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_0fe709f1f2244bd97fe91a6bc7abc7af
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_39453c049e8067f15fc564e903c10ec0 |
| publicationDate |
2019-09-12-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
US-2019275168-A1 |
| titleOfInvention |
Gene therapy for autosomal dominant diseases |
| abstract |
The present disclosure provides methods for treating autosomal dominant diseases in a subject. In some aspects, the methods involve the use of a gene editing enzyme with a pair of unique guide RNA sequences that targets both mutant and wildtype forms of autosomal dominant disease-related gene for destruction in cells, and then supplying the cells with wildtype autosomal dominant disease-related gene cDNA which is codon modified to evade recognition by the guide RNAs. These methods are broadly applicable to any autosomal dominant disease. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021174173-A1 |
| priorityDate |
2015-04-30-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |