http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3850094-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_43a72884e25aaecffebe0942132d704f http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_e4afb1530bd6dd6420ba72c97597ef00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_ed3c2f63a5b1c705b1b389a6f1f1f7c8 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_df02dc2eab1779ebc7dfb5accf94318b |
| classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-30 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2800-80 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-102 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-111 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-11 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-28 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-22 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-805 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-907 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-11 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-10 |
| filingDate | 2019-09-10-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_46e1b28ccab8d04d3311f5cb171b5038 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_7222c62d4686a6d313c4d5ad21b18aa3 |
| publicationDate | 2021-07-21-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | EP-3850094-A1 |
| titleOfInvention | Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies |
| abstract | The clinical severity of β-hemoglobinopathies is alleviated by the co-inheritance of genetic mutations causing a sustained fetal γ-globin chain production at adult age, a condition termed hereditary persistence of fetal hemoglobin (HPFH). Here, the inventors have compared the extent of fetal hemoglobin (HbF) de-repression following CRISPR/Cas9-mediated targeting of different regions of the HBG1 and HBG2 promoters in an adult erythroid cell line (HUDEP- 2). They achieved a potent and pancellular HbF re-activation upon disruption of binding sites for γ-globin repressors located in both HBG1 and HBG2 genes. They validated these findings in Red Blood Cells (RBCs) derived from genome edited Sickle Cell Disease (SCD) patient hematopoietic stem/progenitor cells. Overall, this study identified a binding site for an HbF repressor as a novel and potent target for the treatment of β-hemoglobinopathies. Accordingly, the present invention relates to a method for increasing fetal hemoglobin content in a eukaryotic cell comprising the step of disrupting the binding site for Leukemia/lymphoma-related factor (LRF) in the HBG1 or HBG2 promoter. |
| priorityDate | 2018-09-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 293.