| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_6beb217a9d9a1f28e4bfec27cf7bad03
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_15d6137a95f111623f1a58dd6b47ffd6 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-907
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-85 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-10
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-85
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-90
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
| filingDate |
2020-02-26-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d2eb96ef94d2a76adb4da857cade95ec
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_28d58db772ec0e42418bbd2bcc754888
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_a711ee0b773ba9c16ed0d2eef4ef49ad
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_38d8323d898afcc0bcbbcae8c8fee55f |
| publicationDate |
2020-05-19-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
CN-111172163-A |
| titleOfInvention |
Editing of βIVS-2-654 splice mutant sgRNA based on CRISPR/Cas9 technology |
| abstract |
The invention discloses an sgRNA, a carrier and an application for editing βIVS-2-654 splicing mutation based on CRISPR/Cas9 technology. Its nucleotide sequence table is shown in SEQ ID No: 1, SEQ ID No: 2 or SEQ ID No: 3. By microinjecting sgRNA and Cas9 mRNA into the fertilized eggs of β 654 thalassemia mice, gene-edited thalassemia mice were obtained, the abnormal splicing of β-globin mRNA was corrected, and the expression of β-globin was restored. The hematological and pathological phenotypes of the gene-edited β 654 -thalassemia mice were significantly improved. Therefore, the CRISPR repairing abnormal splicing system of the present invention has important clinical application value for the precise treatment of gene editing based on splicing abnormal single gene disease. |
| priorityDate |
2020-02-26-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |