| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_6e29526f284c0155fd79806c9dd5a1c7 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2267-0306
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2800-80
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14171
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-127
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-705
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y301-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-907
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0058
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0075
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0019
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-465
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-76
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-763
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0048
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-177 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07K14-705
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-17
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-90
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K9-00 |
| filingDate |
2016-04-15-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_cb1c9d7c3c6e3743d2758136f3deb68c
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_bd640722bc665ecd0ceccc352f6ded2e
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_3fd15a1f2fdc70396862e47722416d03
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d140038fd13242c22376d49486a6cc85 |
| publicationDate |
2016-11-10-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
US-2016324987-A1 |
| titleOfInvention |
Use of crispr/cas9 as in vivo gene therapy to generate targeted genomic disruptions in genes bearing dominant mutations for retinitis pigmentosa |
| abstract |
Described herein are methods and compositions for genomic editing. Clustered regularly interspaced short palindromic (CRISPR) allows for highly selective targeting and alteration of genetic loci. Here, the Inventors demonstrate CRISPR as capable of being used in living animals to prophylactically prevent a genetic disease from manifesting. Targeting and disruption of mutated rhodopsin gene prevents progression of retinitis pigmentosa in the retinal cells of a transgenic rat model. Such techniques allow for treatment methods in subjects with dominant genetic mutations, often associated with lack of a gene product, or a toxic gene product. The described technology effectively abrogates deleterious effects due to the presence of a mutated gene copy allowing the normal function of the wild-type protein to prevent cell and vision loss. The efficacy of these in vivo mechanisms are widely extensible to similar dominant negative gene mutations causing disease, or other types of genetic disease. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11730828-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2019102381-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11242525-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11339437-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2023086889-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11535835-B1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020176552-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11566263-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10662425-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11268086-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11141493-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2023203481-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11512311-B2
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2019212594-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11180793-B2 |
| priorityDate |
2015-04-15-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |