| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_914ce1e64249e3dc0af1b375f3920f5d
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2de66e82adda02ef6bb18a5fd58febd7 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2267-0306
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2227-105
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0066
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2217-072
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0085
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N7-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0066
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-14
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4702
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4703
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0019
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-001 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07K14-47
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
| filingDate |
2018-03-23-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_665614aa4b51711e11b283bf599ed78e
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_9d9a167d1879625bc2d28b756027a028
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_e8b3af2b458e54a0593ef9fd26c4ebd9
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_585506ce98888475182ace66a3afe61a |
| publicationDate |
2020-04-22-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
BR-112019019857-A2 |
| titleOfInvention |
mecp2 expression cassettes |
| abstract |
the present invention provides nucleic acid molecules comprising a mecp2 expression cassette, the expression cassette comprising, in operable 5 'to 3' linkage: a 5 'transcriptional control region comprising a promoter capable of conducting transcription in neural cells; an open reading frame that encodes a mecp2 protein; translation control signals; a 3 '(3'utr) untranslated region comprising one or more of: (i) a mir-22 binding site; (ii) a link site for mir-19; (iii) a link site for mir-132; (iv) a link site for mir124; and (v) an element rich in au; and transcriptional termination signals; wherein the mecp2 expression cassette is no more than about 5 kb in length. the invention further provides viral vectors, especially vectors derived from the adeno-associated virus (aav), for use in the therapeutic administration of such expression cassettes. the nucleic acid molecules and viral vectors disclosed here provide new tools for expressing mecp2 and are of particular value in treating disorders associated with reduced mecp2 activity, including rett syndrome. |
| priorityDate |
2017-03-24-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |