Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_4021f8d8a53258a5a3615b4139dd1eee http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_8dde94dce60e96fd9e7426f319f4d158 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_8f2b74fc00e630d7a65419ad81f84236 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_b19c3d18e0c4fdfa84fadefdacf4b74e |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2506-45 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-91 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-727 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-415 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-26 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-22 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-16 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-145 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-125 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-115 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-105 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2500-25 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2510-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2506-11 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-602 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-14 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-2303 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-155 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-165 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-113 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-2311 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-606 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-604 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2501-603 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N5-0647 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N5-0696 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P3-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-06 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-0789 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-074 |
filingDate |
2016-11-07^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_c1913635e3df269615862f89d1381b3c http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_a73f9f73c5fec03068b0f7ac4d449b10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_0202133392855db2c73f60ce340666b1 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_2e22365d44cd45611c7f377e02c1f547 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_2e22ceb6e00183d7be166801fdf5bcde http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_3dbe8e62864a357e9269c8de555f6ffb http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_138bb54f1c952c451cf93dc8736c77d2 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_3312c3e6fd6da9678323301a514d1eef |
publicationDate |
2018-09-12^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
EP-3371300-A1 |
titleOfInvention |
Process of gene-editing of cells isolated from a subject suffering from a metabolic disease affecting the erythroid lineage, cells obtained by said process and uses thereof |
abstract |
The present invention relates to the medical field, in particular to gene editing as a therapeutic approach for the treatment of metabolic diseases affecting the erythroid lineage in a mammalian subject. In invention particular embodiment it refers to the combination of cell reprograming and gene editing for PKD correction as a first example of the potential application of these advanced technologies to metabolic diseases affecting the erythroid lineage. In this sense, PKD patient-specific iPSCs were efficiently generated from PB-MNCs (peripheral blood mononuclear cells) by a SeV non-integrative system and efficiently use to treat pyruvate kinase deficiency. The gene editing strategy for PKLR gene correction was also successfully applied directly to hematopoietic progenitors. |
priorityDate |
2015-11-05^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |