http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2020113868-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_552dfa0fd3a9f5b4339588aea8ad29a3 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_c5073a0063d6be9e25c14d2af0a1ee89 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_e4afb1530bd6dd6420ba72c97597ef00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_df02dc2eab1779ebc7dfb5accf94318b |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-28 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-357 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-28 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-357 |
| filingDate | 2017-12-19-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_c006b3e004ab9b34f2b3a35ddaf2ce27 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_4ef64aa37899aca29a0e3105264f3ef7 |
| publicationDate | 2020-04-16-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | US-2020113868-A1 |
| titleOfInvention | Methods and pharmaceutical compositions for the treatment of neurodegeneration with brain iron accumulation |
| abstract | The present invention relates to methods and pharmaceutical compositions for the treatment of neurodegeneration in with brain iron accumulation (NBIA). Studying two novel genes, namely, CRAT encoding the carnitine acetyltransferase and REPS1 involved in endocytosis and vesicle transport, and a series of known NBIA genes, the inventors reported on iron overload related to increased levels and abnormal recycling of transferrin receptor as a common feature in NBIA. They ascribe this anomaly, at least in part, to impaired palmitoylation of the receptor as a common consequence of the various disease causing mutations. Finally, the inventors show that Artesunate improved TfR1 palmitoylation in NBIA fibroblasts. In particular, the present invention relates to a method of treating neurodegeneration with brain iron accumulation in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a drug increasing TfR1 palmitoylation. |
| priorityDate | 2016-12-20-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 436.