Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_d94573ab2c57e8b193f3a22accddf9fe |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2800-107 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P9-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-47 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-28 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0008 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-02 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0058 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-864 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-28 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P9-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-02 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
filingDate |
2020-12-24-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_e45b883817b41a8e261ac2690c612a12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_622b824b4f129afcad637275c008abc6 |
publicationDate |
2021-05-14-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
CN-112795595-A |
titleOfInvention |
A gene therapy system for hereditary transthyretin amyloidosis |
abstract |
The invention provides a gene therapy system for hereditary transthyretin amyloidosis disease. The CRISPR/Cas9 system is used to treat hATTR, and Nme2Cas9 which can be applied to human transthyretin (TTR) gene therapy for the disease is selected. Applied to this system, high-efficiency gRNA was optimized and designed to knock out functional genes, showing a strong ability to inhibit the expression of mutant TTR, showing the application of this system in the treatment of hereditary transthyretin amyloidosis diseases prospect. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-115125316-A |
priorityDate |
2020-12-24-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |