| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2a819eda0adf22936a52362eeebb9fb4 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2740-16043 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-17
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-47
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/C12N9-22
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
| filingDate |
2021-03-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_3d634764e835ef7bc438a6515119afea |
| publicationDate |
2021-09-23-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2021188349-A2 |
| titleOfInvention |
Methods of treating mitochondrial disorders |
| abstract |
Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN. |
| priorityDate |
2020-03-16-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |